Focus on policies to encourage made in India drugs against rare diseases - Hindustan Times

Focus on policies to encourage made in India drugs against rare diseases

ByHindustan Times
Mar 09, 2022 02:20 PM IST

The article is authored by Anjali Taneja, associate director of Science Policy Initiative at Ashoka University.

The Covid-19 pandemic has underscored the need to be more cautious with health and take a critical look at the pre-existing healthcare systems worldwide. Considerable reforms were also witnessed in the national healthcare services in India during times of emergency caused due to the pandemic.  For example, rapid approval processes and emergency authorisation of Covid-19 vaccines eventually turned out to be a highly effective recourse; this not only helped the distribution of vaccines to every nook and corner of this country within record-breaking time but also ensured the vaccination of our citizens and protection from the virus.  The treatment of patients with rare diseases (RDs) in India also deserves that kind of attention.  

In India, nearly 70-90 million people suffer from RDs; which is expected to be an under-reported figure; the actual number could be higher.(Twitter/This_Life_Mag) PREMIUM
In India, nearly 70-90 million people suffer from RDs; which is expected to be an under-reported figure; the actual number could be higher.(Twitter/This_Life_Mag)


In India, nearly 70-90 million people suffer from RDs; which is expected to be an under-reported figure; the actual number could be higher. Although each of these RDs could be impacting a relatively small percentage of the Indian population, together they affect the lives of millions. Moreover, the direct and indirect costs associated with these diseases are higher in comparison to others. This is largely because (i) most of these RDs are chronic and degenerative in nature, causing disabilities to rise in the patients as they grow old; (ii) lack of adequate or proper screening and diagnosis of RDs patients, especially in the remote areas of the country; (iii) lack of availability of treatments and therapies for almost 95% of RDs; for some others, the treatments have to be administered lifelong by the patients and (iv) most importantly, a huge dependency on imported drugs required for the treatment of RDs in India which escalates the cost of such treatments; making them exorbitantly expensive, and highly unaffordable. In the absence of appropriate insurance coverage or adequate financial support, this adds to the financial burden of the patients and their families.


The revised National Policy for Rare Diseases (2021) makes a mention of the need for promotion of local development of drugs for RDs by pharmaceutical companies.  The Policy states, “Department of Pharmaceuticals, Department for Promotion of Industry and Internal Trade will be requested to promote local development and manufacture of drugs for rare diseases by public and private sector pharmaceutical companies at affordable prices and take legal/legislative measures for creating conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices. PSUs could also be encouraged for local manufacturing of drugs for rare diseases.” While this is a welcome recommendation, the implementation deadlines, processes and approaches, methodology of implementing this strategy, etc have been kept outside the purview of the new policy. It is almost a year since its release and the patients with RDs are still hoping to get some respite from the financial burden. The need of the hour is to ensure that the policy gets implemented without any delay. There is an urgent need to get the domestic manufacturing of drugs for RDs kickstarted in India. 


Forecasts reveal that the sales of drugs for the treatment of RDs are expected to total $262 billion in 2024. So, from an industry’s point of view, there certainly lies the demand, potential and opportunity to capitalise on this market share. Several countries across the globe have introduced RDs legislation, comprehensive regulations and laws for RDs in the early stages of the formulation of national healthcare policies. The laws in most of these economies encourage the local discovery and development of drugs for RDs besides the promotion of investments in research and development of diagnosis, screening, etc. of RDs. For example, the United States introduced the ‘Orphan’ Drug Act as early as 1983 and offers regulatory fee waivers, 50% as a tax credit of the cost of conducting clinical trials, seven-year market exclusivity to sponsors of approved ‘orphan’ drugs and financial grants for clinical research and/or diagnosis of RDs.  Asia Pacific Economic Cooperation (APEC) countries have also taken a lot of initiatives in this direction. In Australia, the Orphan Drugs Policy was introduced in 1997. The country offers benefits like regulatory fee waivers, 5-year market exclusivity, etc. Similarly in Japan, financial subsidies upto 50% expenses for clinical and non-clinical research, 10 years’ exclusive marketing rights, 15% tax credits, user fee waivers, etc to encourage domestic R&D on RDs and local production of RDs’ drugs. South Korea and Taiwan have also introduced Orphan Drugs Guidelines and RDs Control & Orphan Drug Act, respectively in the early years of the millennium decade. Besides, they offer exclusive marketing rights, financial subsidies, medical reimbursements to patients to encourage the local production, R&D and accessibility to these drugs. 


Another inter-related aspect that requires concerted attention in the Indian context is a functional national registry for RDs which does not exist at the moment. Understanding the epidemiology and the burden of disease is extremely important to evaluate the nature of required treatments and drugs to cure RDs. The national policy for RDs also states that the government will have a hospital-based registry at the Indian Council of Medical Research (ICMR) to create a database of various RDs, yield information on the data and disease burden. According to the national policy, ICMR has taken steps in this direction. The need, however, is to strengthen mechanisms to have a comprehensive and functional national RDs registry in India. 


In this respect, it is interesting to note the initiatives taken by the APEC world. In 2018, APEC economies ratified the APEC Rare Disease Action Plan; one of the primary areas of the Plan being to manage the pooling and usage of patient data securely and effectively; besides the plan also advocates better use of technology to enhance the purposeful use of data in clinical decision making, among others. Bearing this in mind, the researchers in Australia have conceptualised the framework of a digital Rare Disease Registry and Analytics Platform (RD-RAP) for providing baseline disease data and innovative analytics – including data analysis, design, diagnostics, therapeutics, health economics, etc. - for informed health services planning and care. This involves leveraging necessary partnerships with industry, academia, clinicians, patient organisations, etc to pool data as well as to create an enabling environment for patient data sharing; ensuring full consent of patients/ families, strict ethical guidelines and data protection laws with respect to the usage of data. Such a framework could be studied extensively and explored in the Indian context. 

The patients with rare diseases undergo tremendous stress and experience various socio-economic challenges in their day-to-day lives. It is high time that RDs is considered an integral part of the Universal Healthcare Coverage (UHC) agenda in India. This could enable equitable access to healthcare for RDs patients and enable their greater care. ‘Cure for All’ must be the mantra of our national policy and the regulatory framework governing RDs. This policy must be implemented in letter and spirit with a priority focus on domestic manufacturing of drugs for RDs, a functional RD registry and enhanced investments in R&D on RDs. The patients with rare diseases are normal people living extraordinary lives. They have an equal right to avail the healthcare services of India as you and I.  

The article is authored by Anjali Taneja, associate director of Science Policy Initiative at Ashoka University.

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