[This guest post is authored by our former blogger Varsha Jhavar and Surbhi Nautiyal. Varsha is a lawyer based in Delhi and is a graduate of Hidayatullah National Law University, Raipur. Surbhi is a lawyer based in Delhi and is a graduate of RGSOIPL, IIT Kharagpur. The views expressed here are those of the authors’ alone.]
In Part I of the post, the authors have analysed the National Policy on Rare Diseases, 2021 (Policy) and its implementation. In Part II, the authors explore the possible consequences of the introduction of legislation for encouraging the development of drugs for rare diseases. Additionally, they provide suggestions for increasing the availability of drugs/therapies for rare diseases in India, inter alia by indigenous development of drugs/therapies.
A Legislation for Encouraging the Development of Drugs/Therapies for Rare Diseases
India, like the EU & its member states, & the US, should consider bringing in a statute for encouraging the development and manufacture of drugs for rare diseases. However, it might not really serve any purpose in India, as the Orphan Drugs Act, 1983 in the US was useful because companies in the US were innovating and manufacturing domestically. It wasn’t a question of if they could try, it was a question of why should they try when there was no market power(!) and so incentives were helpful. In India, forget treatments for rare diseases, even the development of regular treatments for mass markets is an issue. In India, market power as well as local innovation capacity to help shape the design of what is being innovated, is lacking. Of course, some exceptions do exist.
In the absence of a specific legislation, probably, a way to ensure that the Government fulfills its responsibility under the Constitution towards its citizens is by amending the Rights of Persons with Disabilities Act, 2016 to include obligations for at least one of the rare diseases – Duchenne muscular dystrophy (DMD). DMD is covered under the above Act. Unlike the Policy, a legislation will be enforceable and the Government will be bound to do something about it.
Indigenous Development of Drugs
In the absence of significant innovation in the rare diseases space in India, the government needs to either create incentives for domestic manufacture, or it needs to bulk purchase and import drugs at lower prices from manufacturers abroad. Even though India is the largest manufacturer and exporter of generics, the demand for patented drugs is largely met through imports. One of the measures taken by the Department of Pharmaceuticals for tackling this issue has been the launching of the Production Linked Incentive Scheme for Pharmaceuticals in 2021 with a total quantum of incentive of INR 15,000 crore. The Scheme has been introduced in order to augment India’s manufacturing capabilities in the pharmaceutical sector, by providing financial incentives to certain selected indigenous manufacturers proposing to manufacture eligible products (list provided in the Operational Guidelines of the scheme), which includes orphan drugs and the incentives will be paid for a maximum period of 6 years.
Repurposing of drugs gained traction during the Pandemic and the Policy also mentions encouragement of the same. As India is already manufacturing generics in large quantities, repurposing of drugs can be explored as a viable alternative for addressing some of the unmet needs for rare diseases. In a study, it was found that 76% (i.e. 212/279) of the repurposed orphan drugs approved by the USFDA are entirely or partially available in India, in the same form or in a different Fixed Dose Combination. Further, 61 % (i.e. 170/279) of the repurposed orphan drugs have been approved by the Central Drugs Standard Control Organisation.
Cue can also be taken from the CalRx initiative launched by the state of California where “California is investing $100 million into developing a biosimilar insulin product and building a California-based drug manufacturing facility to lower insulin costs.” In terms of indigenous development, Hanugen Therapeutics is developing a therapy for DMD, and researchers at Myo-Mission (anchored by Ashoka University) are also working on developing treatments for DMD, GNE myopathy, and limb-girdle muscular dystrophy. It is heartening to see that academia and research organizations are coming together to collaborate for carrying out research & development of therapeutics for rare diseases, however, the same needs to be done by more companies and for other rare diseases as well.
IP Clauses in Grant Agreements
As per an MoU between Biotechnology Industry Research Assistance Council (BIRAC) & Hanugen Therapeutics, 50% of the fund for the development of therapy for DMD was to be contributed by the Government and the other 50% by Hanugen. Hanugen informed the DHC that it will be unable to fund their half, and taking note of the considerable number of patients of DMD and the need for indigenous development of therapies for DMD and lack of funding with Hanugen, the DHC had directed that BIRAC release the amount of INR 5.35 crore provided that the ‘intellectual property rights in the data, therapies, products/processes’ vest with the Government/BIRAC. The IP Framework for the MoU allows for the designation of a Project as ‘Nationally Important Project’ and such Project would be subject to certain restrictions/conditions on pricing, licensing and March-in-rights for public interest. It does not provide for vesting of IP rights with the Government. Upon the representative for Hanugen giving the all clear for vesting of the IP rights relating to the data as also in any therapeutics/treatment with the Govt., the DHC asked that a fresh agreement be drawn up for the same. In the endeavour to encourage the development of indigenous therapies and drugs for rare diseases, the Govt. taking control of the IP might not be the best way forward, as it takes away the inventors’ incentive to innovate and to invest in R&D when they might not be able to recoup the same.
As pointed out by Prashant Reddy and Saranya Ravindran, the definition/standards for determination of ‘Nationally Important Project’ is absent from BIRAC’s Grant-in-Aid agreements. Standards/parameters for designating a project as a ‘Nationally Important Project’ need to be determined and it should be in line with the provisions of the Patents Act, 1970, especially the general principles stated in Section 83.
Other Suggestions & Observations
Rule 80(7) read with Rule 101 of the New Drugs and Clinical Trials Rules, 2019 (Rules) allows for a local clinical trial waiver for manufacturing a new drug if it is ‘for the rare diseases for which drugs are not available or available at a high cost or if it is an orphan drug’ and is approved and marketed in countries specified by the Central Licensing Authority. Similarly, under Rule 75 (7) (iv) read with Rule 101, waiver of local clinical trial can be applied for importation of a new drug, if it is for rare diseases and has been approved and marketed in countries specified by the Central Licensing Authority. These provisions can be utilized for providing speedy access to certain drugs for patients suffering from rare diseases.
One of the objectives of the Policy is to “lower the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy encompassing awareness generation, premarital, post-marital, pre-conception and post-conception screening”. The present situation can be improved by integrating the existing public health mechanisms with treatment options available for rare diseases. The National Health Mission (NHM), under the MoHW, is a country-wide program that covers reproductive health, maternal health, family planning and newborn health. Pertinently, the MoHW has included rare/genetic diseases within the ambit of NHM. This may facilitate the prevention, identification and treatment of incidences of rare diseases. Further, the Government should consider partnering with diverse stakeholders, i.e., research institutions, government bodies, academia, patient advocacy groups, healthcare providers, and the pharmaceutical industry, as it is crucial for developing robust mechanisms covering all aspects of rare diseases. In fact, the DHC stated that the Committee is permitted to consult any other organisations or persons, whose participation would be required to make effective recommendations to work for the overall objective of the Policy.
It is surprising to note that only INR 7 crores were spent out of the INR 200 crores designated by MoHW for rare diseases in the previous years. This point has been noted by the DHC on multiple occasions and there seems absolutely no reason for it to not have been spent, especially considering the government has resorted to crowdfunding. The state governments can also put in place measures for providing funds and can develop and initiate comprehensive, holistic healthcare strategies for ensuring health infrastructure and uninterrupted medical aid. Recently, the Haryana government has announced a monthly financial pension of INR 2750 for patients with rare diseases. The Rajasthan government has launched a digital platform for facilitating voluntary donations for the treatment of patients with Rare Diseases. Though medical crowd-funding can help in bridging the gap in treatment access, however, for the reasons discussed previously, massive reliance on a platform for sourcing donations is questionable as a government policy.
A helpful development is that genetic disorders were made non-excludable from insurance coverage by the Insurance Regulatory and Development Authority of India (IRDAI) in 2019. This is favourable as most rare diseases are genetic.
The Policy, though well-intentioned, seems to be barely scratching the surface. It identifies the issues related to rare diseases in a broad manner – absence of sufficient data, lack of public awareness, and unavailability of treatment, and provides vague and broad suggestions/action points. There is an evident lack of harmony between health equity and healthcare affordability in the case of rare diseases.
India seems to be moving in the right direction, but much remains to be done. The DHC has been hearing matters relating to people suffering from rare diseases since early 2021 and has done an admirable job of pushing for the introduction of the Policy, encouraging the development of indigenous drugs, supervising the administration of timely treatment and therapies to patients of rare diseases. But still, there is a long way to go before we are able to provide treatment to all patients suffering from rare diseases.
The authors would like to thank Swaraj and Praharsh for their comments and input.