Unlocking Hope: Orphan Drugs, Rare Diseases, and India's Emerging role

In conversation with FE Healthcare, Dr Mathew T Thomas, former US FDA leader and India Director, discusses the global orphan drug ecosystem and India’s opportunity in rare disease drug development.

How has the 1983 Orphan Drug Act influenced orphan drug development in the United States, and what impact has it had globally, including in India?

The orphan drug development in the United States evolved mostly after the 1983 Orphan Drug Act, and since then has contributed to the development of many, many products that have benefited the rare disease population, particularly a population that otherwise would not be able to afford the drugs, as well as an industry that was not willing to invest in such drugs. What the Orphan Drug Act did was offer incentives for a lot of pharmaceutical companies or sponsor investigators to find ways to develop products that they otherwise would not have been able to do over the years. This message has now impacted the globe, and I am particularly pleased that India's awareness of the rare disease has increased and does have the potential for developing rare disease drugs.

What is India's current position in the global pharmaceutical industry, and how do you see its potential for innovation and research, especially in the field of rare diseases?

India continues to be the pharmaceutical of the world, supplying most of the drugs for the world, but it has been lagging behind with innovative research. We have some of the brightest minds in the country and I think that the evolution and progression of the awareness of rare disease populations and the potential for advanced research and development of products is definitely going to be very, very beneficial. Over the years, I see a lot of new products evolving from India, and I look forward to it. 

What are the key challenges involved in developing and conducting clinical research for new medical products, especially in a diverse and vast country like India?

There are many challenges in this domain. It begins with identifying molecules that are going to be beneficial. I think with the advent of artificial intelligence and some of the newer technologies, it becomes a lot easier to identify products. The other challenge is to design a protocol or initiate a research clinical trial that has the appropriate objectives and questions that need to be answered. Identifying the patient population in such a vast country with people spread across multiple states. Once those people are identified, the challenge is to get them to agree to participate in research. This follows the task of educating the people who conduct the research and doing it appropriately with ethics in mind. Finally comes the objective of collecting the data that is of very high quality so that it can be used for decision-making. Over the years, the regulatory agencies and the physicians across the globe would benefit a lot from getting the knowledge of what has been researched and proven effective and safe. 

What role does the U.S. FDA’s Office of Orphan Products Development (OPD) play in encouraging the development of orphan drugs, and what incentives are provided to support this process?

During my time at the United States Food and Drug Administration, which we call the U.S. FDA, I've had the privilege of serving in the Office of Orphan Products Development. We call it the OPD. It is a separate office set apart for encouraging the development of orphan drugs, or drugs for rare diseases, through this office. Incentives are provided for people who want to develop products. So, it begins with somebody identifying a product that they could designate as an orphan drug. They could also get grants. Applications for grants are supported by the government if research is involved. There are other benefits for products that are designated; for example, without getting into the specifics, they get increased exclusivity for marketing, which is not available for regular products. 

They also have taxes waived for the research they conduct. And, once they have a product that is adequately studied and comes ready for an FDA application process, they could get waivers of what it's called the Prescription Drug User Fee Act, which is that the other companies have to pay user fees. And orphan drugs that are designated may be able to get waivers. 

What motivated you to start your own consulting firm after retiring from a 34-year career at the U.S. FDA, and how do your diverse experiences influence the services you offer?

I retired from the FDA in June of 2023. And I found that I had two options. One is to just keep doing all the things I love to do outside of work. But, since I have had a 34-year career in the agency, with varied experiences as a medical reviewer, you know, as somebody who worked in the Office of Scientific Investigations doing numerous investigation sessions of data quality, and working in the Office of Products Division I, working in many administrative and leadership positions, including as the former country director of the US FDA India Office and as the associate director for the Office of Generic Drugs. 

As the Associate Director for the Office of Global Affairs. As the health attaché here in India for the US embassy. All these experiences, I thought, would be useful in providing advice and guidance for people who need it. So I've established a consulting firm called MDT Consulting, LLC.