By Nikkhil K Masurkar
In India, an estimated 96 million individuals are currently living with uncommon medical conditions, and sadly, their access to necessary treatments is severely limited or non-existent. The process of diagnosing these rare diseases often takes an average of seven years, leaving little time for effective intervention. Moreover, even when a diagnosis is finally made, the majority of treatments remain inaccessible to the average person or are simply unavailable within India.
The scarcity of these conditions poses a significant challenge as pharmaceutical companies are reluctant to invest in developing drug treatments due to the low prevalence. Consequently, these vital medical products are left in a state of neglect, labeled as ‘orphaned,’ with little to no further development or accessibility for those who depend on them.
Tragically, less than 10% of rare disease patients worldwide have been fortunate enough to receive treatments specific to their conditions. This statistic is particularly disheartening considering that these illnesses are typically chronic, progressive, and debilitating in nature. Alarmingly, approximately half of the rare disease patients are children, and regrettably, a third of them will not live to see their fifth birthday.
While it is true that only a small fraction, around 5%, of rare diseases have a known cure, many patients can still experience an improved quality of life with appropriate treatment. Unfortunately, the major concern lies in the exorbitant pricing of the medications required for rare diseases, compounded by the fact that none of these medications are manufactured in India. Lacking sufficient insurance coverage or adequate financial assistance, further exacerbates the financial hardships endured by patients and their families.
Government Efforts to Tackle Rare Diseases in the Country
In 2017, the Union Health Ministry of India introduced the National Policy for Rare Diseases (NPRD) as a comprehensive approach to address the challenges posed by rare diseases. This policy has been updated more recently in 2021, aiming to provide support to patients suffering from rare disorders listed in the policy, regardless of their economic background. As part of this initiative, a one-time financial assistance of Rs. 50 lakh is proposed for the treatment of these patients. The list of diseases mentioned in the policy is expected to be continuously updated to ensure its relevance.
The updated National Policy for Rare Diseases (2021) acknowledges the importance of promoting local drug development for rare diseases (RDs) by pharmaceutical companies. The Policy emphasizes the need for the Department of Pharmaceuticals and the Department for Promotion of Industry and Internal Trade to facilitate the indigenous manufacturing of affordable drugs for rare diseases. It suggests exploring legal and legislative measures to create a favorable environment for this purpose, and even encourages public sector units (PSUs) to participate in local drug manufacturing for RDs. While this recommendation is encouraging, the policy falls short in providing specific details regarding implementation deadlines, processes, approaches, and methodologies. Despite being nearly a couple of years since its release, patients affected by rare diseases continue to hope for relief from the financial burdens they face. It is crucial to expedite the implementation of this policy without any further delay, particularly by kickstarting domestic manufacturing of drugs for rare diseases in India.
Ways to Boost the Domestic Production of Rare Disease Drugs
According to forecasts, the sales of drugs for the treatment of rare diseases (RDs) are projected to reach a total of $262 billion by 2024. This indicates a significant demand, potential, and opportunity for the industry to capitalize on this market share. Recognizing the importance of addressing rare diseases, several countries worldwide have implemented legislation, comprehensive regulations, and laws specifically focused on RDs during the early stages of formulating their national healthcare policies.
In many of these economies, the laws actively promote local discovery and development of drugs for rare diseases, as well as encourage investments in research and development related to diagnosis, screening, and other aspects of RDs. A notable example is the United States, which introduced the ‘Orphan’ Drug Act as early as 1983. This act provides various incentives to sponsors of approved ‘orphan’ drugs, including regulatory fee waivers, a 50% tax credit on the cost of conducting clinical trials, a seven-year market exclusivity, and financial grants for clinical research and diagnosis of RDs.
Initiatives have also been taken by Asia Pacific Economic Cooperation (APEC) countries in this regard. For instance, Australia introduced the Orphan Drugs Policy in 1997, which offers benefits such as regulatory fee waivers and five-year market exclusivity. South Korea and Taiwan have also implemented their own guidelines and acts, namely the Orphan Drugs Guidelines and RDs Control & Orphan Drug Act, respectively, during the early 2000s.
India can indeed draw inspiration from these initiatives to bolster its domestic manufacturing capabilities for rare disease drugs. By studying successful models from other countries and incorporating their best practices, India can develop a robust ecosystem for the production of medications specifically designed for rare diseases.
From the perspective of the pharmaceutical industry in India, it is crucial to collaborate with the government to ensure access to treatments for patients in need. Implementing the National Policy for Rare Diseases would prove highly beneficial in this regard. The policy could be designed to incentivize the pharma industry to manufacture orphan drugs and introduce policies that encourage formulation within the country. This dual approach would not only address the demand for orphan drugs in India but also contribute to addressing the global cost issue associated with such drugs.
There is an urgent requirement, both from the industry and the government, to rapidly develop infrastructure and skilled manpower for indigenous research and development, as well as contract manufacturing of cell and gene therapy products. Strategic partnerships with institutions in the UK, USA, and Europe could play a crucial role in achieving this. By providing advanced GMP (Good Manufacturing Practice) and GLP (Good Laboratory Practice) infrastructure for research and development at the startup level, breakthrough products can be created, which the pharmaceutical industry in India can then further enhance.
Given that healthcare is a subject governed by states in India, it is crucial for all state governments to work cohesively with the central government, placing patients as a top priority. This would involve establishing a digital healthcare infrastructure to document the diagnostic and treatment journeys of patients. Collaboratively, they can collect data on patients with rare diseases and create a comprehensive national registry, capturing epidemiological information such as demographics, phenotypes, natural history, disease progression, treatment outcomes, and more. The pharma industry can also contribute by raising awareness about the importance of accurate diagnosis and proper interventions.
Additionally, the Indian Council of Medical Research (ICMR) has invited proposals from interested scientists to work on research and development for rare diseases. This initiative should continue to progress in parallel with the implementation of the national policy, fostering advancements in the field of rare disease treatment and management.
Final Say
The inclusion of rare diseases in India’s Universal Healthcare Coverage (UHC) agenda is long overdue, ensuring equitable access to healthcare for affected individuals and improving their care and support. The national policy and regulatory framework for rare diseases must prioritize the domestic manufacturing of drugs and boost investments in research and development (R&D). Streamlining regulations for orphan drug approval can enhance accessibility. Collaboration among stakeholders, including the government, healthcare providers, pharmaceutical industry, research institutions, and patient advocacy groups, is essential. Together, they can develop comprehensive policies and robust regulatory mechanisms to provide necessary care and treatment for individuals with rare diseases.
(The author is a CEO, Entod Pharmaceuticals. Views expressed are personal and do not reflect the official position or policy of the FinancialExpress.com.)
