Orphan drugs, critical for the treatment of rare diseases, are gaining increasing attention in India after the implementation of the National Policy for Rare Diseases (NPRD) in 2021. However, India faces significant challenges in ensuring the development, affordability, and accessibility of drugs. orphan. , especially compared to other countries such as the United States and the European Union. While global frameworks such as the Orphan Drug Act (ODA) of 1983 in the US have been successful in incentivizing pharmaceutical companies to invest in these drugs, India is still in the early stages of creating a robust system that can address the needs of rare disease patients. .
Orphan drugs are pharmaceutical agents developed specifically to treat rare (orphan) diseases. These diseases, although affecting only a small part of the population, often lead to life-threatening or chronically debilitating conditions. The definition of an orphan drug varies depending on the regulatory framework. In the US, a disease is considered rare if it affects less than 2,00,000 people, while in the European Union, a disease must affect less than 1 in 10,000 people to be considered rare. Although there is no official prevalence-based definition in India, the NPRD of 2021 outlines a framework for diagnosing and treating rare diseases, with a low prevalence threshold. The lack of a clear definition leads to the identification of orphan drugs and addressing the needs of patients affected by these conditions.
Classification of orphan drugs
Orphan drugs are classified based on the type of disease they target and their regulatory status. Diseases such as genetic disorders, rare cancers, metabolic disorders, and autoimmune conditions are often included in the orphan disease category. Genetic disorders include conditions like cystic fibrosis and Duchenne muscular dystrophy, while rare cancers like neuroblastoma and glioma also qualify for orphan drug development. Metabolic disorders, such as Gaucher disease and Fabry disease, and autoimmune diseases, such as systemic sclerosis, also benefit from orphan drugs. Orphan drugs are further classified as approved by regulatory agencies like the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) or orphan drug candidates that are still undergoing clinical trials. These classifications are critical in determining the availability and potential efficacy of treatments for rare diseases.
In India’s NPRD, rare diseases are classified into three categories to facilitate treatment approaches. Group 1 includes disorders that can be treated through a single intervention, such as Lysosomal Storage Disorders (LSDs) requiring Hematopoietic Stem Cell Transplantation (HSCT). Group 2 includes diseases that require long-term or lifelong treatment but relatively low treatment costs, such as Phenylketonuria (PKU) and Maple Syrup Urine Disease (MSUD). Group 3 includes conditions like Gaucher’s Disease and Pompe’s Disease, where treatment is available but complicated by high costs and the need for lifelong treatment.
For a drug to receive orphan drug designation, it must meet certain criteria that vary from country to country. Usually, the disease should have a low prevalence. In addition, the condition must have no approved treatment, or the orphan drug must offer a significant benefit over current treatment options. Orphan drug developers must also provide scientific evidence that the drug has the potential to treat or alleviate the condition. This evidence can be presented at any stage of drug development, from preclinical research to late-phase clinical trials. Once designated, orphan drugs receive a number of incentives to encourage their development, including market exclusivity, tax credits for research and development (R&D) costs, and fee waivers for regulatory applications.
Challenges for India
Although the development of orphan drugs has been incentivized globally, significant challenges remain, especially in countries like India. High research and development costs are a major obstacle, as orphan drugs often target small patient populations, making it difficult for pharmaceutical companies to justify the financial risk. Clinical trials for orphan drugs also face obstacles due to the number of patients available, lengthening the development timeline. Pricing and accessibility are additional challenges, as the high cost of orphan drugs often discourages patients in low- and middle-income countries like India. For example, enzyme replacement therapy (ERT) for diseases like Gaucher’s disease can cost several crores a year, out of reach for most Indian patients.
India faces unique challenges in the development and accessibility of orphan drugs despite efforts like the NPRD. The country lacks an official definition and comprehensive data on the prevalence of rare diseases, which hinders drug development efforts. Without a centralized national registry for rare diseases, it is difficult to estimate the true burden of the condition, limiting pharmaceutical investment in orphan drug research.
At the time of this publication, 14,615 cases were registered on the portal in the rare disease registry. The cost of expensive orphan drugs is further complicated, as many internationally available treatments are prohibitively expensive in India. While the NPRD provides a framework for the diagnosis and treatment of rare diseases, it fails to provide the financial or regulatory incentives that would support the development and marketing of orphan drugs. Unlike the US and the European Union, India has not implemented substantial tax breaks, periods of market exclusivity, or other incentives that could stimulate R&D.
Financial incentives are required hourly
To overcome these challenges, India needs to take some important steps. India is establishing a national rare disease registry to provide accurate prevalence data, guiding the development of targeted treatments. In addition, the government should introduce increased financial incentives, such as tax breaks, research grants and subsidies, to encourage pharmaceutical companies to invest in the development of orphan drugs. Implementing policies that regulate the price of orphan drugs and provide government subsidies can make these treatments more affordable for Indian patients.
Orphan drugs are important for the treatment of rare diseases, which affect a small but significant part of the population. With the right policy support, financial incentives and infrastructure development, India can improve the orphan drug landscape and provide much-needed treatment options to patients with rare diseases.
Published – November 04, 2024 12:28 IST
