A rare disease is a disease that affects a small portion of the population. For such diseases, there is a lack of a market large enough to gain support and resources for discovering treatments for them. According to experts, most rare diseases are genetic and thus are present throughout the person’s entire life, even if symptoms do not immediately appear.
In India, rare diseases like Farber Disease, Gaucher Disease, Hirschsprung’s disease, Multiple Sclerosis, and Spinal Muscular Atrophy (SMA) are widely prevalent. 1 in 20 Indians is affected by one of these rare diseases.
Financial Express.com reached out to Praveen Sikri, CEO, Ikris Pharma Network on strategies to manufacture rare-disease drugs indigenously, challenges faced by the patients of rare diseases in the country, management of rare diseases among others. Excerpts:
What are the status and challenges of rare diseases in India? What are the treatment and diagnosis hurdles faced by the patients of rare diseases in the country?
There are more than 7000 defined genetic rare diseases out of which about 450 are treatable. In India, rare diseases pose a significant challenge to public health systems in terms of difficulty in collecting epidemiological data, which in turn impedes arriving at the burden of diseases and cost estimations, difficulty in research and development, making a correct and timely diagnosis, complex tertiary level management involving long term care and rehabilitation and unavailability and the prohibitive cost of treatment.
Why are drugs for the treatment of rare diseases extremely costly? According to you, how to make them accessible to all patients who struggle to afford such drugs?
Rare diseases constitute a significant economic burden independent of a country’s size and demographics, arising from increased healthcare spending. As resources are limited, there is a macroeconomic allocation dilemma due to the opportunity cost of funding rare disease treatment: on one hand, health problems of a much larger number of persons can be addressed by allocating a relatively smaller amount, on the other, much greater resources will be required for addressing health problems of a relatively smaller number of persons.
A policy is necessary to devise a multipronged and multisector approach to build India’s capacity to tackle rare diseases comprehensively, in areas of – epidemiological data for estimating burden, arriving at a definition and for cost estimation of treatment; research and development for the treatment and diagnostic modalities, including through international/regional collaborations; training of health care providers; awareness generation; creating a conducive environment for drug development and measures for ensuring affordability of treatment, etc.
Is it possible to manufacture rare-disease drugs indigenously and will it bring down the costs? What are strategies that the country needs to focus on to make this a possibility?
It’s not about manufacturing. Most of the innovations for such treatments are in the US or Europe. They are pretty much covered with patents, so if it has to be manufactured and sold in India by a company other than innovators then there might be litigation. Otherwise, it can be done after patent expiration. It also has to be seen if the volume of business commands that kind of investment that runs into millions of dollars.
What are your role and key milestone in the rare disease segment in India? What are your upcoming plans?
The thought behind laying the foundation of Ikris Pharma has been to ensure that no patient or his/her family in our country suffers due to the lack of access to a required medicine or a treatment option at any given point in time. With our global network of pharma manufacturers, distributors, and international retailers, we make available the most sought-after medicines – whether licensed or unlicensed, newly approved or otherwise, for people in our country, and also outside. And we do it all legitimately fulfilling all the necessary legal and regulatory compliance requirements.
Could you explain how you operate some of your programmes such as Managed Access Programme, Early Access Programme, Named Patient Import, and Compassionate User Programme?
Named Patient Program has been designed to ensure that medicines if required in exceptional medical circumstances can be supplied to a patient even when it is at investigational and clinical development stages, or is unregistered and unlicensed for marketing, distribution, and sale in a native country. The idea is to provide access to such medicines outside the scope of the regular legal and operational framework. As long as an expert certifies that all existing treatment options within the country have been exhausted for a given patient condition, and the only recourse to save the patient’s life could be in the form of that investigational or unregistered medicine, patients and families can access facilities under Named Patient Programme.
What is a Reference Listed Drug (RLD)? What has been Ikris Pharma’s contribution to this segment?
RLD stands for Reference Listed Drug. A reference-listed drug is an approved drug product to which new generic versions are compared to show that they are bioequivalent. A drug company seeking approval to market a generic equivalent must refer to the Reference Listed Drug. Ikris Pharma Network specifically is a single-stop platform for all your needs of innovator samples (single batch/multiple batches of specific quantities) for clinical trials as well as bio-equivalence studies. We specialize in order to source Reference Listed Drugs (RLDS)/ Innovator samples from across the globe, including India, USA, Europe, Canada, Australia, New Zealand, Korea, Japan, and Latin America.
According to you, what will India’s role in drug research and development be in the coming years? What are the immediate steps that need to be taken in this segment to improve patient care?
So far Indian pharmaceutical companies’ R&D focus has been on generic product developments. And this has been a big global opportunity. India accounts for more than 30% of global generic supplies. And new drug development requires the involvement of the Government, Research institutes, pharmaceutical companies, and lots of funding. There are a few companies that are trying to be in the innovator space but it may take time before we can make any significant impact. Currently, Health-tech is a big area that mainly works on healthcare delivery to patients. This is a very essential part to improve patient care in India.
India is the world’s largest maker of generic drugs. What are the other areas in which the country needs to take immediate steps to improve its position?
Some of the other areas where we should explore would be APIs and bulk drugs, formulations, biopharma, biosimilars and orphan drugs, among others.
What is Unregistered Imported Medicine? What are the expectations and real scenarios for Indian patients?
An unregistered imported medicine is one that has not undergone evaluation and/or approval by the National or Regional Regulatory Authority (NRRA) for the market in which it is marketed/distributed or used. In other words, it is not licensed for manufacturing, marketing, sale, and distribution within a country under normal circumstances. However in an emergency for the needs of an individual patient, there are ways in which an unregistered medicine can be sourced from outside legitimately. So in a real scenario, this is possible as long as an expert medical practitioner certifies that a given patient has exhausted all other available options within the country.