How to Spot Autoimmune Disease Early
28 Mar 2025
8 Mins
How to Identify and Manage Autoimmune DiseasesAutoimmune diseases are sneaky, often developing slowly but impacting various parts of the body. When your immune system attacks its own cells, it can lead to frustrating symptoms and long-term complications. The objective of this blog is to guide you in recognising the signs of autoimmune disorders [https://www.mrmed.in/health-library/immune-disorders/fatal-autoimmune-diseases], diagnosing them accurately, and offering strategies for managing them through treatments and lifestyle adjustments.    Learn how to stay ahead of these conditions and take proactive steps toward better health and well-being.   WHAT IS AUTOIMMUNE DISEASE?   Autoimmune diseases [https://www.mrmed.in/health-library/immune-disorders/most-common-types-of-autoimmune-diseases] occur when the immune system mistakenly attacks the body’s healthy cells instead of protecting it from harmful invaders. This can lead to inflammation, tissue damage, and organ dysfunction.    A 2024 NIH study [https://pmc.ncbi.nlm.nih.gov/articles/PMC9918670/] found a 12.5% annual increase in the global prevalence of autoimmune diseases. This rise is consistent with global trends, including a 3–4% annual increase in type 1 diabetes incidence over the last three decades and a growing recognition of additional autoimmune and chronic inflammatory conditions.   There are over 80 types of autoimmune diseases affecting various organs and systems in the body.    * Immune System Malfunction: The body’s immune system, designed to protect against harmful pathogens, mistakenly targets healthy cells, leading to immune system disorders.   * Inflammation and Tissue Damage: The immune system’s attack causes inflammation, which can result in long-term tissue damage and dysfunction in affected organs.   * Over 80 Types: Autoimmune diseases can affect different parts of the body, and there are over 80 recognised conditions.   WHAT ARE THE MOST COMMON AUTOIMMUNE DISORDERS?   Autoimmune diseases can affect various parts of the body, leading to a wide range of symptoms and complications. Here are some of the most common autoimmune disorders:   1. Lupus [https://www.mrmed.in/health-library/skin-care/lupus-symptoms-causes-treatment-guide]: It is a chronic autoimmune disease where the immune system attacks the skin, joints, kidneys, heart, and other organs. The symptoms can vary widely, making it difficult to diagnose. Key symptoms include skin rashes (especially a butterfly-shaped rash across the face), joint pain, fatigue, and organ involvement.   2. Rheumatoid Arthritis [https://www.mrmed.in/health-library/rheumatology/rheumatoid-arthritis-causes-treatment]: This autoimmune disease primarily affects the joints, leading to pain, stiffness, and swelling. It can cause long-term damage to the joints if not properly managed. Rheumatoid arthritis often starts in the smaller joints and can progress to larger joints over time.   3. Multiple Sclerosis [https://www.mrmed.in/health-library/immune-disorders/world-multiple-sclerosis-day-2023]: Multiple sclerosis (MS) affects the central nervous system, causing symptoms such as numbness, difficulty walking, vision problems, and fatigue. The immune system attacks the protective covering of nerve fibres, leading to nerve damage and interference with nerve signals.   4. Hashimoto's Disease [https://www.mrmed.in/health-library/health-care/hashimoto-symptoms-treatment]: Hashimoto's disease is an autoimmune disorder that affects the thyroid gland, leading to hypothyroidism [https://www.mrmed.in/health-library/health-care/thyroid-and-weight-loss]. Symptoms include weight gain, cold intolerance, constipation [https://www.mrmed.in/health-library/gastroenterology/types-of-constipation], and unexplained fatigue. It is one of the most common causes of an underactive thyroid.   WHAT ARE THE MAIN CAUSES OF AUTOIMMUNE DISEASE?   The exact autoimmune causes are not fully understood, but it is believed that a combination of genetic and environmental factors plays a role. Some potential triggers include:   * Genetic predisposition: A family history of autoimmune diseases increases the likelihood of developing one.   * Infections: Viral or bacterial infections may trigger the immune system to attack the body’s own tissues.   * Environmental factors: Exposure to certain chemicals, toxins, or even sunlight (in the case of lupus) may trigger an autoimmune response.   * Hormonal factors: Women are more likely to develop autoimmune diseases, suggesting that hormones may influence their development.   WHAT ARE THE EARLY SIGNS OF AUTOIMMUNE DISEASE?   The symptoms of autoimmune diseases can be varied and often mimic those of other health conditions, making diagnosis challenging. Common autoimmune symptoms that may point to an underlying autoimmune disease include:   * Unexplained fatigue: Unexplained fatigue and joint pain autoimmune and feeling excessively tired even after a good night’s sleep.   * Joint pain and swelling: Persistent pain and swelling in the joints without any clear injury.   * Skin rashes: Unexplained rashes, especially those that appear on the face, hands, or other areas.   * Muscle weakness: A general feeling of weakness or difficulty lifting objects or performing normal tasks.   * Fever [https://www.mrmed.in/health-library/viral-infections/home-remedies-fever]: A low-grade fever that doesn’t go away may indicate inflammation in the body.   * Digestive issues: Bloating, gas, diarrhoea, and constipation can be symptoms of autoimmune diseases that affect the digestive system.   * Hair loss: Some autoimmune diseases, like lupus, can cause hair thinning or loss.   * Numbness or tingling: Autoimmune diseases like multiple sclerosis can cause these sensations, particularly in the limbs.   HOW DO YOU DIAGNOSE AN AUTOIMMUNE DISEASE?   An autoimmune diagnosis can be complex due to the variety of symptoms that overlap with other health conditions. If you are experiencing unexplained symptoms such as fatigue, joint pain, or skin rashes, it’s important to consult with a healthcare professional.    They may order a combination of blood tests, imaging tests, and physical examinations to help diagnose the condition. Common diagnostic tools for diagnosing autoimmune diseases include:   * Blood Tests: Blood tests for autoimmune diseases can check for antibodies, which are often elevated in autoimmune diseases. Specific tests like the ANA (antinuclear antibody) test and rheumatoid factor test can provide clues.   * Imaging Tests: X-rays, MRI scans, or ultrasounds may be used to assess joint damage, inflammation, or organ involvement.   * Biopsy: In some cases, a biopsy may be necessary to examine tissues from the affected organ, such as skin or kidney tissue, to confirm the diagnosis.   HOW TO TAKE CARE OF YOURSELF IF YOU HAVE AN AUTOIMMUNE DISEASE?   While there is currently no cure for autoimmune diseases, autoimmune treatment options are available to help manage symptoms, prevent flare-ups, and improve quality of life. Managing an autoimmune disease typically involves a combination of medications, lifestyle changes, and regular monitoring.    Below are some common and easy strategies to manage autoimmune diseases:   1. Medications for Autoimmune Disorders: There are several types of medications for autoimmune disorders that can help control the immune system and reduce inflammation:   * Non-steroidal anti-inflammatory drugs (NSAIDs): These drugs, such as ibuprofen, help reduce pain and inflammation in conditions like rheumatoid arthritis and lupus.   * Disease-modifying antirheumatic drugs (DMARDs): These medications, such as methotrexate [https://www.mrmed.in/molecule/methotrexate], slow the progression of diseases like rheumatoid arthritis and lupus.   * Biologic drugs: Biologics, like adalimumab [https://www.mrmed.in/molecule/adalimumab] (Humira), target specific parts of the immune system and are used for conditions like rheumatoid arthritis and psoriatic arthritis.   * Steroids: Corticosteroids are used to reduce inflammation quickly during disease flare-ups, but they come with potential long-term side effects.   * Immunosuppressants: Drugs like azathioprine [https://www.mrmed.in/molecule/azathioprine] suppress the immune system, preventing it from attacking healthy cells.   2. Autoimmune Therapy: Autoimmune therapy focuses on modulating the immune system to prevent it from attacking the body. Some therapies include:   * Plasmapheresis: This procedure removes harmful antibodies from the blood and is often used in severe cases of autoimmune diseases like lupus or myasthenia gravis.   * Intravenous immunoglobulin (IVIg): This treatment provides the body with normal antibodies and can help regulate immune function in autoimmune diseases.   3. Diet and Lifestyle Changes for Autoimmune Disorders: Certain diet and lifestyle changes for autoimmune disorders can help manage symptoms and prevent flare-ups:   * Anti-inflammatory diet: A diet rich in anti-inflammatory foods, such as fruits, vegetables, fatty fish (like salmon), nuts, and seeds, can help reduce inflammation.   * Gluten-free or low-carb diets: Some people with autoimmune diseases, especially those with conditions like Hashimoto's disease, may benefit from reducing gluten or refined carbohydrates in their diet.   * Regular exercise: Regular physical activity helps maintain joint health, reduce inflammation, and improve overall well-being.   * Stress management [https://www.mrmed.in/health-library/mental-wellness/relieve-mental-stress]: Chronic stress can trigger flare-ups, so practising relaxation techniques like yoga, meditation, and deep breathing exercises can be beneficial.   * Adequate sleep: Good quality sleep is crucial for managing autoimmune diseases and reducing fatigue.   4. Autoimmune Disease Flare-Up Prevention Tips: To avoid flare-ups and manage symptoms, consider these autoimmune disease flare-up prevention tips:   * Track your symptoms: Keep a journal to track your symptoms and triggers, which can help you identify patterns and avoid potential flare-ups.   * Avoid infections: Since infections can trigger flare-ups, it’s important to take steps to avoid illness, such as washing hands regularly and getting vaccinations as recommended by your doctor.   * Follow your treatment plan: Stick to the treatment prescribed by your healthcare provider, including taking medications as directed and attending follow-up appointments.   Nahid Khilji [https://www.linkedin.com/in/nahidkhilji/], Nutritionist and Co-founder of Health Hatch, a wellness company, said, “Lack of physical activity or a sedentary lifestyle is linked to autoimmune diseases such as rheumatoid arthritis, SLE, Sjogren syndrome, gout and others. Physical activity, in fact, boosts immunity and anti-inflammatory responses.”   POPULAR CELEBRITIES WITH AUTOIMMUNE DISORDERS IN INDIA:   Several popular celebrities in India have openly shared their experiences with autoimmune disorders, raising awareness and inspiring others to manage their conditions. Here are some notable figures:   * Kangana Ranaut: The actress has been open about her battle with Hashimoto’s disease, a condition where the thyroid is attacked by the immune system.   * VJ Dhivyasarshini: VJ Dhivyasarshini has shared her experience with autoimmune thyroiditis, a condition that affects the thyroid gland and its functioning.   * Samantha Ruth Prabhu: Samantha has been vocal about her struggle with myositis, a rare autoimmune disease that causes muscle weakness and inflammation.   * Arjun Kapoor: Arjun has openly discussed his battle with Hashimoto’s disease.   FREQUENTLY ASKED QUESTIONS   Q1. What are the early signs of autoimmune disease?   Early signs of autoimmune diseases include persistent fatigue, joint pain or swelling, and skin rashes. These symptoms often appear gradually and may come and go.   Q2. How to diagnose autoimmune disorders?   To diagnose autoimmune disorders, doctors perform blood tests to check for specific antibodies. They may also review medical history, conduct physical exams, and use imaging tests to assess organ or tissue damage.   Q3. What are some common autoimmune disease symptoms in women?   Common autoimmune disease symptoms in women include fatigue, joint pain, swelling, and muscle weakness. Skin rashes, hair loss, and digestive issues may also occur. These symptoms can vary depending on the specific autoimmune condition.   Q4. What are the first symptoms of lupus?   The first symptoms of lupus often include fatigue, joint pain, and a butterfly-shaped rash across the cheeks and nose. You may also experience unexplained fever or hair loss. Early diagnosis is crucial for effective management.   Q5. Can autoimmune diseases be cured?   Autoimmune diseases cannot be cured, but they can be managed with treatment. Medications and lifestyle changes help control symptoms and reduce flare-ups. Early diagnosis and consistent care improve outcomes.   WRAPPING UP   Autoimmune diseases are complex and can affect quality of life. Early recognition, timely diagnosis, and appropriate treatment are essential for managing conditions like Lupus, Rheumatoid arthritis, and Multiple sclerosis. With proper care, symptoms can be controlled, reducing the risk of complications.   Lifestyle changes also play a key role in improving overall well-being. The best supplements for autoimmune disease management include Vitamin D, Omega-3 fatty acids [https://www.mrmed.in/molecule/omega-3-fatty-acids], Turmeric, and Probiotics, which can support immune function and reduce inflammation.    Consult a healthcare professional for accurate diagnosis and treatment to manage symptoms and live a fulfilling life.   ALSO, READ OUR OTHER BLOGS ON:   1. The Rising Tide of Autoimmune Disorders: Why Now? [https://www.mrmed.in/health-library/immune-disorders/autoimmune-disorders] 2. Autoimmune Myths vs. Facts: What’s the Truth? [https://www.mrmed.in/health-library/immune-disorders/myths-facts-autoimmune-disorder] 3. Top Foods Packed with Vitamin D You Need [https://www.mrmed.in/health-library/health-care/foods-high-in-vitamin-d]   REFERENCES   1. Kumar U, Kanjilal M, Ramakrishnan L, Thangavelu M. Prevalence of pre-clinical autoimmunity in the normal adult population residing in a metropolitan city of India: A cross-sectional study. https://pmc.ncbi.nlm.nih.gov/articles/PMC8133893/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC8133893/] 2. Rosenblum MD, Remedios KA, Abbas AK. Mechanisms of human autoimmunity. https://pmc.ncbi.nlm.nih.gov/articles/PMC4518692/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC4518692/] 3. Goris A, Liston A. The immunogenetic architecture of autoimmune disease. https://pmc.ncbi.nlm.nih.gov/articles/PMC3282406/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC3282406/] 4. https://www.who.int/publications/i/item/9241572361 [https://www.who.int/publications/i/item/9241572361]
Dr. Vijayalakshmi
Is Mounjaro Worth The Hype, Or Just Another Fad?
25 Mar 2025
7 mins
Is Mounjaro Worth The Hype, Or Just Another Fad?Disclaimer: The information in this blog is based on news reports about Mounjaro's approval in India. MrMed or related parties do not have any confirmation of the drug's launch or availability in India. We encourage readers not to make any changes to their lifestyle or medicine consumption patterns based only on this article.   “Wait, another weight loss injection? How is this one any different?”   If you’re wondering the same thing, you’re not alone! The buzz around Mounjaro has skyrocketed, and for good reason. Whether you’ve heard about it from your fitness-obsessed friend, seen it trending online, or stumbled across a headline screaming “miracle weight loss drug,” you’re probably curious about what makes this injectable prescription drug a game-changer.   We’re here to break it down for you—no jargon, no complicated science, just real talk. Let’s dive into what Mounjaro is, how it works, and why it’s taking the weight loss world by storm.   WHAT EXACTLY IS MOUNJARO AND HOW DOES IT WORK?   Mounjaro, also known by its scientific name, tirzepatide injection [https://www.mrmed.in/health-library/health-care/weight-loss-drug-tirzepatide-coming-to-india], is a once-weekly injectable medication approved by the U.S. FDA in May 2022. Originally developed to manage type 2 diabetes [https://www.mrmed.in/health-library/diabetes/prevention-type-2-diabetes], this drug has now gained popularity for its impressive ability to promote weight loss.   Here’s the cool part: Mounjaro mimics not one but two important hormones—GLP-1 (Glucagon-Like Peptide-1) and GIP (Glucose-Dependent Insulinotropic Polypeptide). These hormones regulate blood sugar and appetite, making you feel full for longer and reducing those sneaky cravings.   Unlike traditional GLP-1 receptor agonists, Mounjaro’s dual-action mechanism makes it more effective in promoting weight loss while improving insulin [https://www.mrmed.in/molecule/insulin] sensitivity. This is why it's often considered more powerful than other medications in the same category.   IS MOUNJARO EFFECTIVE FOR WEIGHT LOSS?   Short answer? YES! But let’s back that up with some real numbers.   In a 2022 clinical trial (SURMOUNT-1), [https://investor.lilly.com/news-releases/news-release-details/lillys-surmount-1-results-published-new-england-journal-medicine] participants who took Mounjaro lost an average of 15-22% of their body weight over 72 weeks. That’s nearly 52 pounds for someone weighing 230 pounds! To put this in perspective, most other weight loss medications help people lose around 5-10% of their body weight.   Penn Medicine experts have also weighed in, stating that tirzepatide (Mounjaro) can be highly effective in weight management and diabetes control but should be administered under professional supervision, considering individual responses to dosage levels.   HOW IS MOUNJARO DIFFERENT FROM OTHER WEIGHT LOSS INJECTIONS?   You might be wondering—how does Mounjaro stack up against other popular weight loss drugs like Ozempic or Wegovy?   1. Dual Hormone Action: Mounjaro’s ability to target both GLP-1 and GIP receptors sets it apart, making it more effective at reducing hunger and slowing down digestion.   2. Higher Efficacy: Clinical studies show that tirzepatide Mounjaro outperforms semaglutide [https://www.mrmed.in/molecule/semaglutide] (found in Ozempic and Wegovy) in terms of weight loss results.   3. Improved Blood Sugar Control: Since Mounjaro was originally designed for type 2 diabetes, it offers an added bonus of helping manage blood sugar levels alongside weight loss.   HOW MUCH DOES MOUNJARO COST?   Let’s talk money. Mounjaro isn’t cheap, but it’s gaining traction due to its transformative results.   * In the U.S., the average monthly price is around $1,023 for a one-month supply (without insurance). * Mounjaro Injection Price in India: The estimated price can vary between ₹45,000 to ₹60,000 per month, depending on location and pharmacy availability.   If you’re considering Mounjaro, check with your insurance provider to see if the cost is covered under your health plan. Many providers may offer partial or full coverage for diabetic patients.   HOW DO YOU TAKE MOUNJARO?   Mounjaro is a once-weekly injection that you administer yourself. The starting dose is usually Mounjaro 2.5 mg injected under the skin. Depending on how your body responds, the dose may gradually be increased over the weeks.   * Inject into the stomach, thigh, or upper arm. * Rotate injection sites to avoid skin irritation. * Set a reminder so you don’t miss your weekly shot.   WHAT ARE THE SIDE EFFECTS OF MOUNJARO?   Let’s be real—every medication has side effects, and Mounjaro is no exception. Most people experience mild discomfort, but some may face more intense side effects.   Common Side Effects Include: * Nausea and vomiting * Diarrhea or constipation * Fatigue or dizziness * Reduced appetite   Less Common but Serious Side Effects: * Pancreatitis (inflammation of the pancreas) * Gallbladder problems * Low blood sugar (especially if taken with other diabetic medications [https://www.mrmed.in/condition/diabetes]) If you experience persistent nausea, try taking Mounjaro at night or with food. It often reduces discomfort.   WHO SHOULD CONSIDER USING MOUNJARO?   Mounjaro isn’t for everyone. While it’s highly effective for weight loss, it’s primarily approved for:   * People with Type 2 Diabetes who need better blood sugar control. * Individuals with a BMI of 30 or higher looking for effective weight management. * Those with a BMI of 27 or higher with at least one weight-related condition (like high blood pressure).   Avoid Mounjaro if: * You have a history of pancreatitis. * You have a personal or family history of medullary thyroid carcinoma [https://www.mrmed.in/health-library/cancer-care/thyroid-cancer-symptom-causes-treatment]. * You’re pregnant or planning to become pregnant.   HOW FAST CAN YOU LOSE WEIGHT WITH MOUNJARO?   Patience pays off. While some people notice a drop in weight within the first month, significant changes typically occur around 12-16 weeks. The speed of weight loss depends on factors like:   * Dosage and duration * Diet and lifestyle * Body’s response to the medication   On average, people lose around 1.5 to 2 pounds per week on Mounjaro when combined with a healthy diet and regular physical activity.   WHAT’S THE BUZZ ABOUT MOUNJARO IN INDIA?   Mounjaro has generated immense interest in India, especially among people seeking effective weight loss solutions. However, Mounjaro injection in India is not yet widely available and may only be accessible through select importers or specialised pharmacies.   Dr. Anjali Hooda, a leading metabolic specialist in New Delhi, stressed the importance of using Mounjaro responsibly. In a social media post, she warned: "Are you planning to take Ozempic or Mounjaro? Be responsible... Lifestyle changes must accompany these medications for sustainable results."   This highlights the need for long-term lifestyle adjustments rather than relying solely on medication.   CAN YOU GET MOUNJARO WITHOUT A PRESCRIPTION?   Nope, and for good reason. Mounjaro is an injectable prescription drug that requires medical supervision. Self-medicating or sourcing it without a valid prescription can lead to serious health complications.   Always consult a healthcare provider before starting Mounjaro to ensure it’s the right fit for your health goals.   FREQUENTLY ASKED QUESTIONS (FAQS)   1. IS MOUNJARO APPROVED FOR WEIGHT LOSS?   Mounjaro is FDA-approved for managing type 2 diabetes but is widely prescribed off-label for weight loss due to its remarkable efficacy. For weight management, the FDA has approved Zepbound [https://www.healthline.com/health/drugs/zepbound], which contains the same active ingredient, tirzepatide. If your primary goal is weight loss, your doctor might suggest Zepbound instead of Mounjaro.      2. HOW LONG DOES IT TAKE FOR MOUNJARO TO SHOW RESULTS?   Noticeable weight loss typically begins after 12-16 weeks, with optimal results observed around 6 months when combined with a healthy diet and regular exercise. Some users may experience gradual improvements even earlier.     3. CAN MOUNJARO BE USED FOR NON-DIABETIC WEIGHT LOSS?   Yes, Mounjaro is frequently prescribed off-label for non-diabetics, particularly for individuals with obesity [https://www.mrmed.in/health-library/cancer-care/obesity-symptoms-side-effects] or weight-related conditions. Studies have shown significant weight reduction even in non-diabetic individuals using tirzepatide.     4. IS MOUNJARO SAFE FOR LONG-TERM USE?   Early clinical data suggests that Mounjaro has a favourable safety profile, but long-term safety is still under review. Regular follow-ups with your healthcare provider are recommended to monitor potential side effects and ensure ongoing safety.     5. WHAT HAPPENS IF I STOP TAKING MOUNJARO?   Discontinuing Mounjaro may lead to appetite return and potential weight regain. To maintain weight loss, it’s essential to develop sustainable lifestyle habits and consider a long-term maintenance plan with your doctor.   FINAL THOUGHTS   If you’re tired of fad diets and exhausting workout routines that lead nowhere, Mounjaro might just be the solution you’ve been waiting for. With its promising results and unique dual-action mechanism, it’s no surprise that Mounjaro is gaining popularity across the globe.   But remember—it’s not magic. Long-term success still depends on maintaining a healthy lifestyle and staying consistent. So, if you’re considering this journey, talk to your doctor, weigh the pros and cons, and get ready to embrace a healthier, happier you.   READ ALSO   10 Ways To Lose Weight Naturally [https://www.mrmed.in/health-library/health-care/lose-weight-naturally] World Obesity Day [https://www.mrmed.in/health-library/health-care/world-obesity-day] Side Effects Of Semaglutide [https://www.mrmed.in/health-library/diabetes/side-effects-of-semaglutide] Semaglutide Diet Plan [https://www.mrmed.in/health-library/diabetes/semaglutide-diet-plan]   REFERENCES   1. https://www.fda.gov/news-events/press-announcements/fda-approves-new-medication-chronic-weight-management [https://www.fda.gov/news-events/press-announcements/fda-approves-new-medication-chronic-weight-management] 2. https://www.nejm.org/doi/full/10.1056/NEJMoa2206038 [https://www.nejm.org/doi/full/10.1056/NEJMoa2206038] 3. https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/fdas-concerns-unapproved-glp-1-drugs-used-weight-loss [https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/fdas-concerns-unapproved-glp-1-drugs-used-weight-loss] 4. https://www.drugs.com/history/zepbound.html [https://www.drugs.com/history/zepbound.html] 5. https://investor.lilly.com/news-releases/news-release-details/fda-approves-zepboundr-tirzepatide-first-and-only-prescription [https://investor.lilly.com/news-releases/news-release-details/fda-approves-zepboundr-tirzepatide-first-and-only-prescription] 
Dr. Vijayalakshmi
Disability Rights A Path to Equal Healthcare
24 Mar 2025
8 Mins
Disability Rights & Healthcare: Ensuring Access“Healthcare is a right, not a privilege” The healthcare system is meant to serve everyone equally, yet individuals with disabilities often face obstacles that undermine their access to essential care. From physical barriers to societal attitudes, healthcare disparities are a real concern. These inequities not only affect the quality of care but also perpetuate stigma and inequality.    The objective of this blog is to highlight the importance of disability rights in healthcare, address ongoing challenges, and examine the laws protecting disabled individuals' rights in healthcare settings.   WHAT IS THE SIGNIFICANCE OF DISABILITY RIGHTS IN HEALTHCARE?   Disability rights in healthcare are crucial to ensure that individuals with disabilities receive equitable treatment and access to necessary care.    A 2023 NIH study [https://pmc.ncbi.nlm.nih.gov/articles/PMC10569625/] found that the prevalence of disability in India is 22 per 1,000 people, with hearing impairment [https://www.mrmed.in/health-library/health-care/world-hearing-day] being the most common (2.2 per 1,000), followed by visual and speech disabilities. Additionally, 12.4 per 1,000 individuals were found to have locomotor disabilities.   These rights aim to eliminate discrimination and promote inclusivity within the healthcare system. Here are five important aspects to think about:   * Equal Access and Dignity: People with disabilities [https://www.mrmed.in/health-library/health-care/international-day-of-people-with-disabilities] should have the same access to healthcare services as those without, ensuring they receive proper care with dignity and respect.   * Tailored Medical Care: Healthcare services should be adapted to meet the specific needs of individuals with disabilities, acknowledging their unique requirements for treatment.   * Physical and Procedural Accessibility: Beyond physical accessibility, healthcare providers must ensure that their facilities, communication methods, and procedures are inclusive of people with disabilities.   * Training and Awareness: Healthcare professionals should receive adequate training on how to address the needs of patients with disabilities to ensure competent and compassionate care.   * Policy Gaps: In many regions, healthcare policies still fail to fully address the needs of disabled individuals, creating additional barriers to care.   WHAT ARE DISABILITY HEALTHCARE DISPARITIES? If you wonder, How do they affect disabled individuals? Disability healthcare disparities refer to the inequalities that disabled people face when trying to access proper medical care.    These disparities are often caused by a mix of social, physical, and systemic issues within healthcare systems.    * Lack of Specialised Care: People with intellectual disabilities often require specific treatments and ongoing support, but many healthcare facilities are not equipped to provide them.   * Communication and Mobility Challenges: Disabled individuals may face difficulties with communication, mobility, or understanding medical instructions, which means healthcare providers must be specially trained to meet their needs.   * Inadequate Healthcare Laws: Many regions have laws that don't fully address the unique needs of people with intellectual or developmental disabilities, leaving these patients underserved.   * Extra Time and Assistance Needed: Disabled patients may need more time for consultations or help with filling prescriptions, yet these needs are often ignored or inadequately addressed.   * Worse Health Outcomes: Without proper care and accommodations, disabled individuals are more likely to experience poor health outcomes compared to others.   WHAT ARE THE RIGHTS OF DISABLED PERSONS?   In India, the legal rights of disabled patients are protected by several laws aimed at preventing disability discrimination and ensuring equal access to healthcare. These laws are designed to guarantee that individuals with disabilities receive the necessary support and accommodations to access medical care.    Below are some important legislations and provisions that protect the rights of disabled patients in India:   Law/Policy  Key Provisions The Rights of Persons with Disabilities Act, 2016This is one of the major disability healthcare laws. It ensures that people with disabilities have the right to access healthcare services without discrimination, requiring healthcare providers to make reasonable accommodations.National Health Policy, 2017Emphasises the importance of inclusive healthcare, ensuring that public healthcare institutions make their services accessible to individuals with physical, sensory, or intellectual disabilities.The Mental Healthcare Act, 2017Ensures that individuals with mental disabilities receive appropriate healthcare and treatment without discrimination, providing access to mental health [https://www.mrmed.in/health-library/mental-wellness] services and support for their specific needs.Accessibility Standards under the Ministry of HealthRequires healthcare facilities to ensure physical accessibility, including ramps, elevators, and accessible toilets, for people with disabilities. Also mandates providing appropriate assistance for patients with special needs.Inconsistent EnforcementWhile these laws exist, their enforcement often remains inconsistent, and many disabled individuals still encounter significant barriers in accessing healthcare due to a lack of infrastructure and awareness among healthcare providers.   WHAT IS DISABILITY DISCRIMINATION IN HEALTHCARE?   Disability discrimination in healthcare occurs when disabled individuals face barriers to accessing proper care due to physical obstacles or negative attitudes from healthcare providers.    This can result in substandard care or outright denial of services. Here are the main ways this happens:   * Physical Barriers: Inaccessible medical equipment or healthcare facilities that prevent disabled individuals from receiving care.   * Lack of Training: Healthcare professionals may not be trained to properly interact with or accommodate disabled patients, leading to poor or inadequate care.   * Communication Challenges: Healthcare providers may not offer accessible communication methods for individuals with hearing or speech impairments.   * Failure to Modify Procedures: Medical procedures may not be adapted for patients who use mobility aids, leaving them unable to receive proper treatment.   * Advocacy and Policy Change: Disability rights groups play a key role in raising awareness, providing support, and pushing for changes in laws to create more inclusive healthcare systems.   WHAT IS THE HEALTH INSURANCE SCHEME FOR PERSONS WITH DISABILITIES?   In India, several health insurance schemes provide coverage and financial support for persons with disabilities, ensuring access to quality healthcare. Key options include healthcare policy disability initiatives such as:   1. Ayushman Bharat Pradhan Mantri Jan Arogya Yojana [https://nha.gov.in/PM-JAY] (PMJAY)   This government scheme provides health insurance coverage for economically vulnerable populations, including persons with disabilities. It offers free access to hospitalisation and treatment at empanelled hospitals.   2. Rashtriya Vayoshri Yojana [https://www.india.gov.in/spotlight/rashtriya-vayoshri-yojana] (RVY) While primarily aimed at senior citizens, this scheme also provides assistance for the elderly with disabilities, offering support for the purchase of assistive devices to improve their quality of life.   3. National Disability Insurance Scheme (NDIS)   Aimed specifically at individuals with disabilities, the NDIS provides funding for necessary healthcare and rehabilitation services, including medical treatment, therapy, and support services.   4. The ADIP Scheme [https://depwd.gov.in/adip/] (Assistance to Disabled Persons for Purchase/Fitting of Aids and Appliances)   The ADIP Scheme, launched by the Government of India, provides financial assistance for persons with disabilities to acquire aids and appliances such as wheelchairs, hearing aids, and prosthetics. This scheme aims to enhance the mobility and quality of life for disabled individuals.   5. State-Specific Health Schemes   Several states in India have developed their own schemes to assist persons with disabilities. For example, the Karnataka State Disability Welfare Scheme provides health insurance coverage to persons with disabilities for surgeries, medical treatment, and hospitalisation.   6. Private Insurance Providers   Some private insurance companies in India offer specialised health insurance policies tailored for persons with disabilities. These plans may cover medical expenses, rehabilitation costs, and assistive devices, but premiums may vary depending on the disability and coverage required.   HOW TO IMPROVE HEALTHCARE ACCESSIBILITY FOR A DISABLED PERSON?   To ensure that healthcare access for disabled individuals becomes a reality, systemic changes must be implemented at all levels of healthcare. Here are some key areas where change is needed:   * Improving Physical Accessibility: Healthcare facilities must be fully accessible, including ramps, elevators, accessible toilets, and medical equipment that accommodates mobility aids. This ensures equal healthcare access for disabled individuals.   * Training Healthcare Providers: Healthcare professionals need training in understanding disabilities, communication strategies for intellectual or sensory impairments, and respecting patient rights disability to improve patient interactions.   * Policy Reforms: Disability rights and healthcare access require reforms to close gaps in care, incentivise specialised services, and ensure inclusive healthcare policies. Governments must support these changes to make healthcare settings both physically and service-wise accessible.   * Insurance and Financial Support: Financial barriers often hinder disabled individuals' healthcare access. Governments should provide adequate insurance coverage that includes assistive technologies, therapy, and long-term care.   * Inclusive Healthcare Technology: Healthcare providers should adopt assistive technologies, such as accessible medical records and telemedicine, to improve diagnosis, treatment, and communication for disabled patients. This is vital for advancing disability rights advocacy and ensuring effective healthcare delivery.   FREQUENTLY ASKED QUESTIONS   Q1. What are some government programs for disabled healthcare?   In India, the Rights of Persons with Disabilities Act of 2016 ensures equal healthcare access for disabled individuals. It mandates that public and private healthcare providers make reasonable accommodations and offers various government schemes like Ayushman Bharat to support medical expenses for disabled people.   Q2. What are the challenges in accessing healthcare for disabled people?   Challenges in accessing healthcare for disabled people include physical barriers in medical facilities, lack of trained healthcare providers, and insufficient disability healthcare support programs tailored to specific needs.   Q3. How to find a wheelchair accessible doctor?   To find a wheelchair-accessible doctor, check online directories, contact local healthcare providers, or ask for recommendations from disability support organisations to ensure the practice meets accessibility needs. In India, the Rights of Persons with Disabilities Act of 2016 mandates that public spaces, including healthcare facilities like hospitals and clinics, be made accessible to persons with disabilities.   Q4. How to ensure healthcare access for people with disabilities?   To ensure healthcare access for people with disabilities, it's crucial to implement Disability healthcare support programs and provide specialised care. Additionally, focusing on Healthcare for people with intellectual disabilities and ensuring facilities are accessible can significantly improve healthcare outcomes.   Q5. What are the health care rights of persons with disabilities?   In India, persons with disabilities have the right to accessible healthcare, non-discriminatory treatment, and reasonable accommodations. Additionally, the National Trust for Welfare of Persons with Autism, Cerebral Palsy [https://www.mrmed.in/health-library/nerve-disorders/world-cerebral-palsy-day], Mental Retardation, and Multiple Disabilities Act, 1999 also supports healthcare rights, ensuring provisions for their medical needs and welfare.   THE PATH AHEAD   Ensuring healthcare access for people with disabilities requires systemic changes, legal protections, and continued advocacy. Disability discrimination and access barriers remain challenges, but legal rights and advocacy offer hope for an inclusive future. Evolving healthcare systems must become more accessible, equitable, and responsive.    By advocating for disability rights, we can build a healthcare system that serves all patients without discrimination.   ALSO, READ OUR OTHER BLOGS:   1. Achieve Work-Life Balance: Essential Mental Wellness Tips for You [https://www.mrmed.in/health-library/mental-wellness/tips-for-working-professionals] 2. International Day Of People With Disabilities [https://www.mrmed.in/health-library/health-care/international-day-of-people-with-disabilities] 3. Mental Stress Symptoms And Effects On Your Body [https://www.mrmed.in/health-library/mental-wellness/mental-stress-symptoms-effects] 4. Healthy Ways To Relieve Mental Stress [https://www.mrmed.in/health-library/mental-wellness/relieve-mental-stress]   REFERENCES   1. Series L. Disability and human rights. In: Watson N, Vehmas S, editors. Routledge Handbook of Disability Studies. 2nd edition. New York : Routledge; 2019 Oct 21. Chapter 6. Available from: https://www.ncbi.nlm.nih.gov/books/NBK558160/ [https://www.ncbi.nlm.nih.gov/books/NBK558160/] 2. Pattnaik S, Murmu J, Agrawal R, Rehman T, Kanungo S, Pati S. Prevalence, pattern and determinants of disabilities in India: Insights from NFHS-5 (2019-21). Front Public Health. https://pmc.ncbi.nlm.nih.gov/articles/PMC10009251/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC10009251/] 3. https://nhrc.nic.in/sites/default/files/DisabilityRights.pdf [https://nhrc.nic.in/sites/default/files/DisabilityRights.pdf]
Dr. Vijayalakshmi
The Importance of Clean Water for Health
21 Mar 2025
8 mins
The Importance of Clean Water for HealthWater is life. Yet, for millions of people, access to clean and safe drinking water remains a luxury. In a country like India, where water scarcity and contamination are pressing concerns, the conversation about clean water and health is not just a matter of choice—it's a matter of survival. But what makes clean water so essential for our health? And how does a lack of it impact entire communities?   Whether you’re sipping on a glass of water after a long day or using it to cook meals, the quality of that water matters more than you might think. Let’s dive into why safe drinking water is a necessity and explore the effects of dirty water on our well-being.   WHY IS CLEAN WATER IMPORTANT FOR HEALTH?   Water does more than just quench thirst. It plays a vital role in almost every bodily function—regulating temperature, flushing out toxins, aiding digestion, and keeping our organs functioning. But when the water we consume is contaminated, it can do more harm than good.   In India, 37.7 million people are affected by waterborne diseases annually, according to a report by the National Institution for Transforming India (NITI Aayog [https://www.niti.gov.in/sites/default/files/2023-08/COMPENDIUM-OF-BEST-PRACTICES-IN-WATER-MANAGEMENT-3.0_Water-Resources-Vertical_2_8_23.pdf]). Contaminated water can carry bacteria, viruses, and harmful chemicals that lead to severe illnesses, often proving fatal for young children and the elderly.   HOW DOES CLEAN WATER PREVENT DISEASES?   When we drink clean, treated water, we’re shielding ourselves from a host of waterborne diseases that plague communities with poor sanitation.   1. Prevents Diarrhoea and Cholera According to a 2023 report by the Ministry of Health and Family Welfare (MoHFW) [https://mohfw.gov.in/?q=%2Fpress-info%2F7535], India reports over 1.5 million cases of diarrhoea from contaminated water every year, leading to around 300,000 child deaths [https://www.sciencedirect.com/science/article/abs/pii/S0277953616304853#:~:text=Introduction,year%20(UNICEF%2C%202013).]. Clean water prevents the spread of bacterial infections [https://www.mrmed.in/health-library/health-care/bacterial-infections] like cholera and typhoid, which thrive in unhygienic conditions.   2. Reduces Heavy Metal Poisoning High levels of arsenic and fluoride in groundwater are a serious concern in India, particularly in states like West Bengal, Uttar Pradesh, and Bihar. Long-term exposure to heavy metals [https://www.who.int/news-room/fact-sheets/detail/arsenic] in drinking water can lead to cancer, kidney damage, and developmental disorders.   3. Prevention of the Spread of Waterborne Illnesses Clean water coupled with proper sanitation can reduce the transmission of diseases like hepatitis A and E, leptospirosis, and parasitic infections, keeping entire communities safer.   WHAT ARE THE BENEFITS OF DRINKING CLEAN WATER?   Staying hydrated is essential, but when the water you drink is clean and free of contaminants, the benefits go beyond hydration.   1. Improved Digestion and Metabolism   Clean water aids in the breakdown of food and absorption of nutrients, promoting better digestion and a healthy gut.     2. Boosts Immunity Proper hydration flushes out toxins, supports the immune system, and helps the body fight off infections more effectively.     3. Promotes Healthy Skin Drinking clean water keeps the skin hydrated, preventing dryness and reducing the risk of skin infections [https://www.mrmed.in/health-library/fungal-infections/fungal-skin-infection] caused by bacteria in unclean water.     4. Prevents Dehydration and Fatigue Dehydration health effects include dizziness, fatigue, and kidney problems. Clean water replenishes lost fluids and keeps energy levels stable.   WHAT DISEASES ARE CAUSED BY CONTAMINATED WATER?   Contaminated water is a silent killer responsible for a wide range of health risks that disproportionately affect vulnerable populations.   1. Cholera and Dysentery: Caused by bacteria in contaminated water, these diseases lead to severe diarrhoea and dehydration, often requiring urgent medical intervention.   2. Hepatitis A and E: Viral infections [https://www.mrmed.in/health-library/viral-infections] transmitted through contaminated food and water can cause liver inflammation, leading to jaundice and severe fatigue.   3. Typhoid and Paratyphoid Fever: These bacterial infections thrive in unsanitary conditions and are transmitted through ingestion of contaminated water.   4. Arsenic Poisoning: Prolonged exposure to arsenic in drinking water leads to skin lesions, cancer [https://www.mrmed.in/condition/anti-cancer], and developmental issues.   HOW DOES THE LACK OF CLEAN WATER AFFECT COMMUNITIES?   When communities lack access to safe drinking water, the consequences extend far beyond individual health. It creates a ripple effect that disrupts education, livelihoods, and overall well-being.   1. Increased Healthcare Burden   According to a 2023 report by the Central Pollution Control Board (CPCB [https://pib.gov.in/PressReleseDetailm.aspx?PRID=1941065&reg=3&lang=1]), around 46% of India’s surface water is contaminated. This leads to a spike in healthcare costs as communities grapple with the aftermath of waterborne diseases.     2. Impact on Child Mortality   UNICEF India reports that poor water quality and sanitation are responsible for 21% of communicable diseases in India, disproportionately affecting children under five [5].     3. Economic Losses and Productivity Decline   When people fall sick due to contaminated water, they miss work and lose wages, impacting the overall economic output of the community.   WHAT IS THE ROLE OF CLEAN WATER IN CHILD DEVELOPMENT?   For children, clean water is not just about preventing disease—it’s about enabling proper growth and development.   1. Improved Cognitive Development   Studies by the Indian Council of Medical Research (ICMR) highlight that chronic exposure to contaminated water can impair cognitive development in children, affecting their ability to learn and perform in school.     2. Reduced Risk of Malnutrition   Contaminated water often leads to recurrent diarrhoea, which inhibits nutrient absorption, making children more susceptible to malnutrition and stunted growth.   HOW DOES SAFE DRINKING WATER IMPROVE LIFE EXPECTANCY?   Access to safe drinking water can add years to life. By preventing infections, reducing malnutrition, and promoting overall health, clean water plays a direct role in increasing life expectancy.   1. Lower Child Mortality Rates   According to a 2022 World Bank report [https://www.worldbank.org/en/country/india/brief/world-water-day-2022-how-india-is-addressing-its-water-needs], ensuring access to safe water and improved sanitation could reduce child mortality rates by up to 50% in India.     2. Reduced Incidence of Chronic Illnesses   Long-term exposure to contaminants in drinking water has been linked to chronic diseases [https://www.mrmed.in/health-library/health-care/chronic-illness-rise] like kidney failure [https://www.mrmed.in/health-library/nephrology/causes-kidney-failure], liver damage, and cancer. Clean water reduces these risks significantly.   HOW DOES WATER POLLUTION IMPACT HUMAN HEALTH?   India’s water bodies are under constant threat from industrial discharge, agricultural runoff, and untreated sewage. This water pollution health affects millions across the country.   1. Toxic Chemicals in Groundwater   In 2023, the Central Ground Water Board (CGWB) reported that 19 states in India had dangerously high levels of fluoride and arsenic in groundwater, affecting over 60 million people [7].     2. Microbial Contamination   Bacteria and viruses, often found in untreated sewage, lead to widespread outbreaks of waterborne illnesses, especially in urban slums and rural areas.   EXCLUSIVE TIPS: HOW TO ENSURE CLEAN WATER AT HOME   * Boil Water Before Use: Boiling kills most bacteria and viruses, making water safe to drink. * Use Water Purifiers: Investing in a reliable water purifier can remove heavy metals and other contaminants. * Regularly Clean Water Storage Tanks: Unclean storage containers can reintroduce bacteria into treated water. * Practice Rainwater Harvesting: Collecting and treating rainwater can provide a sustainable source of clean water.   FREQUENTLY ASKED QUESTIONS (FAQS)    1. WHAT ARE THE FIRST SIGNS OF WATERBORNE ILLNESS?   The most common signs include diarrhoea, nausea, vomiting, fever, and stomach cramps. In severe cases, dehydration can set in quickly, requiring medical attention.     2. HOW CAN I TEST IF MY DRINKING WATER IS SAFE?   You can get your water tested through government-certified laboratories like the Central Pollution Control Board (CPCB) or local health authorities to detect contaminants. You can also purchase home water testing kits.     3. IS BOILING WATER ENOUGH TO KILL ALL BACTERIA?   Yes, boiling water for at least 1-3 minutes kills most bacteria, viruses, and parasites. However, it does not remove heavy metals or chemical contaminants.     4. CAN CONTAMINATED WATER AFFECT LONG-TERM HEALTH?   Absolutely. Prolonged exposure to contaminants can lead to chronic illnesses such as kidney damage, cancer, and developmental disorders, especially in children.     5. HOW CAN COMMUNITIES ENSURE BETTER WATER SANITATION?   By investing in proper sewage treatment, promoting awareness about hygiene practices, and installing community water purification systems, communities can significantly reduce cholera and water contamination risks.   FINAL THOUGHTS   Clean water isn’t a luxury—it’s a basic human right. While progress is being made, millions in India still face the consequences of unsafe water. By ensuring access to safe drinking water and improving water sanitation, we can prevent disease, promote better health outcomes, and enhance life expectancy. Let’s work towards making clean water accessible to every household—because good health starts with safe water.   RELATED ARTICLES   1. 10 Must-know Pros & Cons of Fluoridated Drinking Water [https://www.mrmed.in/health-library/health-care/pros-and-cons-of-fluoridated-drinking-water] 2. All You Need To Know About Viral Hepatitis [https://www.mrmed.in/health-library/viral-infections/all-about-viral-hepatitis] 3. A Guide To Common Seasonal Diseases in India [https://www.mrmed.in/health-library/viral-infections/seasonal-diseases-in-india]   REFERENCES   1. https://pib.gov.in/PressReleaseIframePage.aspx?PRID=1807831 [https://pib.gov.in/PressReleaseIframePage.aspx?PRID=1807831] 2. https://www.nitiforstates.gov.in/sector?sector=Water+%26+WASH [https://www.nitiforstates.gov.in/sector?sector=Water+%26+WASH] 3. https://nhm.gov.in/index1.php?lang=1&level=2&lid=219&sublinkid=819 [https://nhm.gov.in/index1.php?lang=1&level=2&lid=219&sublinkid=819] 4. https://pmc.ncbi.nlm.nih.gov/articles/PMC1151007/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC1151007/]
Dr. Vijayalakshmi
Understanding Orphan Drug Treatments For Rare Diseases
19 Mar 2025
7 min
Understanding Orphan Drug Treatments For Rare DiseasesWhat if Your Disease Had No Cure? Imagine walking into a doctor’s office, feeling hopeful about getting a diagnosis—only to hear, “Your condition is so rare, we don’t have a treatment for it.” Sounds terrifying, right? Well, for millions of people worldwide, this isn’t just a bad dream—it’s their reality.   Rare diseases, by definition, affect a small percentage of the population, but their impact is anything but small. In fact, over 300 million people globally suffer from a rare disease. The challenge? Most pharmaceutical companies don’t see profit in developing treatments for such a small market. That’s where orphan drugs for rare diseases come in.   But what exactly are these drugs, and how do they help people battling rare diseases [https://www.mrmed.in/health-library/health-care/rare-disease-day]? Let’s break it down in the simplest way possible.   WHAT ARE RARE DISEASES, AND WHY ARE THEY SO HARD TO TREAT?   A rare disease is any condition that affects around 70-96 million people in India or fewer than 200,000 people in the U.S. Some well-known rare diseases include Huntington’s disease, muscular dystrophy, and cystic fibrosis, but there are thousands more—many of which you’ve probably never heard of.   Why is treatment so difficult?   1. Lack of Research – Since these diseases affect a small population, they don’t get the same attention or funding as common illnesses like diabetes [https://www.mrmed.in/health-library/diabetes/common-side-effects-of-diabetes] or heart disease [https://www.mrmed.in/health-library/heart-care/six-most-common-heart-diseases].   2. Few Patients for Clinical Trials – To develop a drug, scientists need test subjects. However, with rare diseases, finding enough patients for a clinical trial is tough.   3. High Costs – Pharmaceutical companies spend billions on drug development, and when there’s a small market, they struggle to recover those costs.   This is where orphan drug development becomes critical.   HOW DO ORPHAN DRUGS HELP RARE DISEASE PATIENTS?   Orphan drugs are speciality medications for rare diseases. They’re called “orphan” drugs because, for a long time, pharmaceutical companies abandoned them due to low profitability. But that changed when governments worldwide introduced incentives to encourage research and production.   Why do orphan drugs matter?   * They give hope to people with conditions that were previously untreatable. * They help manage symptoms and improve the quality of life. * In some cases, they completely change the course of a disease, slowing or even stopping its progression.   In India, the government introduced the National Policy for Rare Diseases (NPRD) 2021, which provides financial support of up to ₹50 lakhs for patients with certain rare diseases, making orphan drugs more accessible.   In India, the approval and availability of orphan drugs have increased significantly since the introduction of the National Policy for Rare Diseases (NPRD) 2021. For instance, Zolgensma (Onasemnogene Abeparvovec), a groundbreaking gene therapy for Spinal Muscular Atrophy (SMA), gave a new lease on life to a 6-month-old baby named Ayaansh Gupta from Hyderabad [https://www.indiatoday.in/cities/hyderabad/story/hyderabad-kid-spinal-muscular-atrophy-16-crore-injection-costliest-1814216-2021-06-13], improving his muscle strength and mobility within months of treatment.   LIST OF FDA-APPROVED ORPHAN DRUGS MAKING AN IMPACT IN INDIA   Several FDA-approved orphan drugs have been introduced in India, transforming treatment outcomes for patients with rare diseases.   1. Kalydeco (Ivacaftor) for Cystic Fibrosis – One of the first precision medicines for cystic fibrosis, significantly improving lung function and quality of life. 2. Brineura (Cerliponase Alfa) for Batten Disease – Approved in 2017, it slows down the progression of neuronal ceroid lipofuscinosis type 2 (CLN2), giving children a chance at an improved quality of life. 3. Zolgensma (Onasemnogene Abeparvovec) for SMA – The world’s most expensive gene therapy that offers a one-time cure for SMA. Though costly, it has been used in India through crowdfunding and government-supported initiatives.   WHAT ARE THE BIGGEST CHALLENGES IN RARE DISEASE TREATMENT?   Even with orphan drug development, treating rare diseases is still an uphill battle. Some of the biggest roadblocks include:   1. High Drug Prices   Since rare disease drugs cater to a small population, they often come with staggering price tags—sometimes hundreds of thousands of dollars per year. Insurance coverage isn’t always guaranteed, making access difficult for many patients.     2. Delayed Diagnoses   The average time for a rare disease diagnosis is 4-5 years. That’s because many doctors have never encountered these conditions before, leading to misdiagnoses and long periods of uncertainty for patients.     3. Limited Availability   Even if an orphan drug exists, it might not be readily available in every country. Many patients are forced to travel abroad or wait years for approvals.     4. Ethical Dilemmas in Drug Development   When pharmaceutical companies focus on profits, they may abandon research on a drug that doesn’t seem financially viable—even if it has the potential to save lives.   ARE GENE THERAPY AND BIOLOGICS THE FUTURE OF RARE DISEASE TREATMENT?   Two of the most exciting advancements in rare disease treatment are gene therapy and biologics for rare diseases.   What is Gene Therapy?   Think of gene therapy for rare diseases as reprogramming your body to fix itself. Scientists modify or replace defective genes to correct the underlying cause of a disease. For some rare diseases, this could mean a one-time treatment instead of lifelong medication!   For instance, Pompe disease is a lysosomal storage disorder that leads to progressive muscle weakness and respiratory issues due to an enzyme deficiency. Researchers are now exploring gene therapy as a treatment for Pompe disease, aiming to replace the faulty gene and restore normal enzyme production. If successful, this could significantly improve patients’ quality of life and reduce dependency on lifelong enzyme replacement therapy.     How Do Biologics Help?   Unlike traditional drugs, which are made from chemicals, biologics are made from living cells. They’re designed to target specific disease processes and are often more effective with fewer side effects.   While both treatments are still developing, they offer hope for the future of rare disease treatment.   WHAT IS THE ROLE OF PHARMACEUTICAL COMPANIES IN RARE DISEASE DRUGS?   Pharmaceutical companies play a crucial role in bringing orphan drugs to the market, but their motivations can be complex.   The Good:   * Many companies invest heavily in rare disease research, helping patients who previously had no treatment options. * Some companies partner with patient advocacy groups to push for faster drug approvals.     The Not-So-Good:   * Some companies use orphan drug status to extend monopolies, keeping prices high even after the research costs have been recovered. * Delays in manufacturing or distribution can leave patients without access to life-saving medications.   Still, without pharmaceutical companies, orphan drug development would not be possible—so it’s all about finding a balance between innovation and affordability.   WHY IS ACCESS TO RARE DISEASE DRUGS STILL A STRUGGLE?   Despite advancements in rare disease drug availability, patients face several challenges in getting the treatment they need:   1. Regulatory Hurdles – Drug approvals take time, and many rare disease treatments are stuck in bureaucratic limbo.   2. Geographic Barriers – Some drugs are available in one country but not in another, forcing patients to travel or wait years for local approvals.   3. Affordability Issues – Even when treatments exist, they can be unaffordable for the average patient without government or insurance support.   This is why patient advocacy groups and global initiatives push for better access to speciality medications for rare diseases.   FREQUENTLY ASKED QUESTIONS (FAQS)   1. Why are rare diseases so expensive to treat?   Since rare diseases affect a small population, drug companies recover research costs by setting higher prices. Additionally, the cost of clinical trials, regulatory approvals, and limited patient numbers all contribute to the high expense.     2. How can governments help improve access to orphan drugs?   Governments can provide tax incentives, funding for research, faster approval processes, and price regulations to make rare disease drugs more accessible and affordable.     3. What are some recent breakthroughs in rare disease treatment?   Advancements in gene therapy, biologics, and personalised medicine have revolutionised the way rare diseases are treated. Some of these therapies offer one-time cures instead of lifelong symptom management.     4. Can rare diseases be prevented?   Some rare diseases, especially those with genetic origins, cannot be prevented. However, early genetic testing and screening can help with early diagnosis and better treatment planning.     5. What should someone do if they suspect they have a rare disease?   If you experience unusual, unexplained symptoms, consult a specialist or genetic counsellor. You can also connect with rare disease advocacy groups to find resources and support.   FINAL THOUGHTS   While the fight against rare diseases is far from over, progress is happening. With orphan drug development, gene therapy, and biologics, we’re closer than ever to finding treatments that give hope to millions. But there’s still work to be done. Governments, pharmaceutical companies, and healthcare systems need to make these treatments accessible and affordable so no patient is left behind.   If there’s one thing to take away from this, it’s that rare diseases may be uncommon, but their impact is huge—and finding a cure should never be a question of profitability. Rare diseases may be “rare,” but the fight for better treatments and accessibility is a global challenge. The more we understand and advocate, the closer we get to making life better for millions of people.   PEOPLE ALSO READ   Understanding Biosimilar Drugs: Are They as Effective? [https://www.mrmed.in/health-library/health-care/biosimilar-drugs]   REFERENCES   1. https://www.ncbi.nlm.nih.gov/books/NBK572052/ [https://www.ncbi.nlm.nih.gov/books/NBK572052/] 2. https://pmc.ncbi.nlm.nih.gov/articles/PMC10084869/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC10084869/] 3. https://www.ncbi.nlm.nih.gov/books/NBK56187/ [https://www.ncbi.nlm.nih.gov/books/NBK56187/] 4. https://www.liebertpub.com/doi/10.1089/ipm.10.03.09 [https://www.liebertpub.com/doi/10.1089/ipm.10.03.09]
Dr. Vijayalakshmi
Are Biosimilars Really Effective
18 Mar 2025
8 Mins
Understanding Biosimilar Drugs: Are They as Effective?In modern healthcare, the high cost of biologic therapies has sparked interest in biosimilars, which offer similar benefits at a lower price. But are biosimilars as effective as biologics? This blog will explore the key differences between the two, assess the effectiveness of biosimilars, and discuss whether they can be trusted as reliable alternatives to traditional treatments.    The objective of this blog is to provide clarity on the potential of biosimilars in healthcare.   WHAT ARE BIOSIMILAR DRUGS, AND HOW DO THEY WORK?   Biosimilar drugs are highly similar to an existing biologic drug (the reference product) in terms of structure, biological activity, and efficacy, but they are not identical. Unlike generic drugs, which are exact copies of small-molecule medications, biosimilars are developed to have no clinically meaningful differences in safety, effectiveness, and quality.   * Biologics: Complex drugs derived from living organisms, used to treat conditions like cancer [https://cancerlibrary.mrmed.in/], rheumatoid arthritis [https://www.mrmed.in/health-library/rheumatology/rheumatoid-arthritis-causes-treatment], and autoimmune diseases [https://www.mrmed.in/health-library/immune-disorders/fatal-autoimmune-diseases]. Examples include monoclonal antibodies, vaccines, and gene therapies.   * Development Process: Biosimilars undergo rigorous clinical trials to prove they are highly similar to the original biologic product.   * Regulatory Approval: Biosimilars are approved by regulatory bodies like the FDA (U.S.) or EMA (Europe) after demonstrating no clinically meaningful differences in safety, efficacy, and immunogenicity.   * Healthcare Impact: Biosimilars help reduce healthcare costs by introducing competition to original biologic drugs.   * Key Differences from Generics: Unlike generic drugs for small molecules, biosimilars are not exact replicas but closely match the reference biologic in terms of therapeutic effect.   * Popular Biosimilar Drugs: Some of the widely recognised biosimilar drugs include Zarxio (filgrastim), Inflectra (infliximab), Erelzi (etanercept), Amjevita (adalimumab), and Hulio (adalimumab). These biosimilars are used to treat a variety of conditions, including autoimmune diseases, cancers, and chronic inflammatory conditions.   A 2023 systematic review [https://www.fda.gov/drugs/spotlight-cder-science/safety-outcomes-when-switching-between-biosimilars-and-reference-products] and meta-analysis from the US FDA concluded that there were no significant safety differences between patients who switched between biosimilars and reference products and those who did not switch. The study found no differences in death rates, serious adverse events, treatment discontinuations, or immunogenicity between the two groups, supporting the safety of switching between biosimilars and reference products.   HOW DO BIOSIMILARS COMPARE TO GENERIC DRUGS?   Biosimilars and generic drugs are often confused, but they are fundamentally different in terms of composition, approval process, and how they are manufactured. Here’s a detailed comparison of biosimilar and generic drugs:   Aspect Generic Drugs Biosimilars CompositionChemically identical to the original small-molecule drug.Not identical, but highly similar to the reference biologic drug due to the complexity of biologic molecules.Approval ProcessA simpler, faster approval process, requiring evidence of bioequivalence (same active ingredients and effects).More rigorous approval process, requiring extensive clinical testing and comparative studies to demonstrate similarity in safety, efficacy, and quality.Molecular StructureExact molecular structure as the original.Similar, but with minor structural differences due to the complexity and variability in biologic manufacturing.BioequivalenceBioequivalent (performs the same in the body as the original).Not bioequivalent; biosimilar medications only need to show similarity, not identical performance. Minor differences are acceptable if they don’t affect safety or efficacy.Manufacturing ProcessStandard, well-established chemical synthesis process.Complex, involving living organisms, which can result in minor variations. Highly sensitive to changes in production processes.CostGenerally 20-30% cheaper than the brand-name drug.Typically 10-30% cheaper than the reference biologic, but savings can vary based on market dynamics.Regulatory OversightApproved by regulatory agencies like the FDA or EMA through an abbreviated pathway.Requires a more complex approval pathway, such as through the FDA’s 351(k) pathway in the U.S. or similar regulations internationally.   ARE BIOSIMILARS SAFE?   Biosimilars are approved based on a rigorous assessment process to ensure their safety. The approval process for biosimilars is hard and designed to ensure that these drugs are safe for patients to use.    After regulatory approval, biosimilars continue to be monitored for safety through ongoing pharmacovigilance programs, which track adverse reactions and long-term effects.   However, it’s important to note that not all biosimilars are interchangeable with their reference biologics. Some biosimilars may require specific recommendations from healthcare professionals, especially in terms of switching or transitioning between drugs.    Interchangeability is a distinct status granted by regulatory bodies, and not all biosimilars have received this designation. In countries like the U.S., the FDA has a specific process to designate whether a biosimilar can be substituted for a reference biologic without the involvement of a physician.   ARE BIOSIMILARS AS EFFECTIVE AS BIOLOGICS?   Biosimilars are designed to be highly similar to their reference biologic drugs, but there are some important factors to consider when evaluating Biosimilar effectiveness.   * Comparable Efficacy: Extensive clinical studies demonstrate that biosimilars are just as effective as their reference biologics in treating the same conditions.   * Rigorous Testing: Biosimilars undergo comprehensive clinical trials to prove they are highly similar in terms of safety, efficacy, and quality, though they are not identical to the original biologic.   * Minor Differences: While biosimilars are nearly identical in molecular structure, minor variations in manufacturing can result in slight differences in how they interact with the body, but these differences do not significantly impact overall efficacy.   * Post-Marketing Surveillance: After approval, ongoing monitoring and real-world data further confirm that biosimilars perform similarly to biologics in diverse patient populations.   * Approval Process: Regulatory bodies such as the FDA and EMA ensure that biosimilars meet stringent standards before they are approved for use, ensuring they can be trusted to offer similar benefits to patients.   WHAT ARE THE BENEFITS OF USING BIOSIMILARS?   Biosimilars offer several key benefits, making them an appealing option in modern healthcare, especially in terms of accessibility and affordability.   * Lower Cost: Biosimilars are typically priced 10-30% lower than their reference biologic counterparts, making them more affordable for patients and healthcare systems.   * Increased Access to Treatment: The reduced cost of biosimilars helps expand access to life-saving biologic therapies for a broader patient population, especially in lower-income areas.   * Same Safety and Efficacy: Extensive clinical testing ensures that biosimilars are just as safe and effective as their reference biologics, offering similar therapeutic benefits for patients.   * Encourages Competition: The availability of biosimilars introduces competition in the market, which can drive prices down and improve healthcare system sustainability.   * Better Treatment Options: With more options available, healthcare providers can tailor treatment plans to individual patients, providing a broader range of effective therapies.   * Potential for Cost Savings in Healthcare: The use of biosimilars can lead to significant savings in healthcare spending, which can be reinvested into other areas of patient care and treatment innovations.   HOW DO BIOSIMILARS IMPACT THE COST OF HEALTHCARE?   Biosimilars in healthcare have the potential to significantly impact the cost of healthcare by providing more affordable alternatives to expensive biologic therapies.   * Lower Medication Costs: Biosimilars are priced 10-30% lower than reference biologics, reducing the direct cost of medications for patients and healthcare providers.   * Reduced Healthcare Spending: The widespread use of biosimilars can lower overall healthcare spending by providing a cost-effective alternative to high-priced biologics, especially for chronic and long-term treatments.   * Savings for Healthcare Systems: Governments and insurance companies can save significant amounts on biologic therapies, which can help alleviate financial strain on public health systems and private insurers.   * Increased Market Competition: By introducing more treatment options, biosimilars foster market competition, which can lead to further price reductions across both biologics and biosimilars.   * Reinvestment in Care: The cost savings from biosimilars can be reinvested into other aspects of patient care, such as preventative treatments, additional research, or more accessible healthcare services.   * Improved Patient Access: With lower costs, more patients are able to access necessary treatments, improving health outcomes and reducing the financial burden on individuals.   FREQUENTLY ASKED QUESTIONS   1. What is the difference between a biosimilar and a biologic drug?   The key difference between Biosimilars vs biologics is that a biologic drug is the original, complex medication derived from living organisms, while a biosimilar is a highly similar version developed after the original patent expires. Biosimilars must demonstrate no clinically meaningful differences in safety, efficacy, and quality compared to the reference biologic.   2. Can biosimilars be used to treat cancer and autoimmune diseases?   Yes, biosimilars can be used to treat cancer and autoimmune diseases [https://www.mrmed.in/health-library/immune-disorders/most-common-types-of-autoimmune-diseases]. They are developed to closely match the reference biologic drugs, which are commonly used for these conditions, ensuring similar safety and efficacy.   3. Are biosimilars safe for long-term use?   Yes, biosimilars are safe for long-term use. They undergo rigorous testing to ensure they meet the same safety and efficacy standards as the reference biologic.   4. Can biosimilars cure cancer?   Biosimilars cannot cure cancer, but they can effectively treat certain types of cancer. They are designed to closely match the reference biologic drugs used in cancer treatment [https://www.mrmed.in/health-library/cancer-care/what-is-cancer-and-symptoms-treatment], helping manage the disease and improve outcomes. While not a cure, biosimilars offer a cost-effective alternative with similar safety and efficacy.   5. What biosimilars are available for rheumatoid arthritis?   Several biosimilars are available for rheumatoid arthritis [https://www.mrmed.in/health-library/immune-disorders/rheumatoid-arthritis], including those that target TNF inhibitors like adalimumab [https://www.mrmed.in/molecule/adalimumab] and infliximab [https://www.mrmed.in/molecule/infliximab]. These biosimilars provide similar efficacy and safety to the reference biologics while being more cost-effective.   WRAPPING UP   Biosimilar drugs are transforming healthcare by offering affordable, effective alternatives to biologics without compromising safety or efficacy. With rigorous testing supporting their reliability, biosimilars help reduce the financial burden of treatments, making essential medications more accessible. As their use expands, patients and healthcare providers can trust in their effectiveness and potential to drive cost savings.    ALSO, READ OUR OTHER BLOGS:   1. The Latest Breakthroughs in Autoimmune Arthritis Treatment [https://www.mrmed.in/health-library/rheumatology/autoimmune-arthritis-treatment-options-] 2. Revolutionary 3D Robotic Phantom: India’s Breakthrough for Lung Cancer [https://www.mrmed.in/health-library/respiratory-disorder/lung-cancer-treatment]   REFERENCES    1. https://www.fda.gov/drugs/therapeutic-biologics-applications-bla/biosimilars [https://www.fda.gov/drugs/therapeutic-biologics-applications-bla/biosimilars] 2. https://www.ema.europa.eu/en/human-regulatory-overview/biosimilar-medicines-overview [https://www.ema.europa.eu/en/human-regulatory-overview/biosimilar-medicines-overview] 3. Chan JCN, Chan ATC. Biologics and biosimilars: what, why and how? ESMO Open. https://pmc.ncbi.nlm.nih.gov/articles/PMC5519784/ [https://pmc.ncbi.nlm.nih.gov/articles/PMC5519784/]
Dr. Vijayalakshmi