[A big thanks to Swaraj for his inputs in this piece and to Aditi Bansal for her help in curating the database below.]
A Sweet Pill to Swallow?
On August 9, 2024, the Ministry of Finance (MoF), Government of India, issued a circular (pdf) for a Global Tender Enquiry to increase the threshold for procurement of 127 medicines instead of the previously decided 120, inviting tenders for up to 200 crore, further extendable as required. Drugs for rare diseases also make an appearance on the list. Rare diseases, as defined by the World Health Organisation (WHO), are severe, often lifelong conditions with a prevalence of 1 or fewer cases per 1,000 people. The challenges associated with rare diseases and the scarcity of accessible medication have been a recurring theme on this blog (here).
Last year, the Indian government introduced its first four domestically produced drugs for rare diseases. These drugs, also referred to as “orphan drugs,” are part of a broader strategy to reduce treatment costs by up to 100 times, thereby making them more affordable for Indian patients. Building on this, the Indian Council of Medical Research (ICMR) extended an invitation to local pharmaceutical and biotechnology firms in February 2024 (as reported by The Print), aiming to encourage the development of localised treatments for ‘priority rare genetic disorders.’ This latest development of August 2024—acquiring patented medications—represents a hopeful advancement in addressing the needs of patients with rare diseases.
As reported by Times Now, this list of drugs seems to include 17 specifically for ‘rare diseases’, while almost all other news sites have reported the government to be acquiring “120 patented” medicines. We will analyse the veracity of this claim, as well as similar assertions made by other news sources, later in this post. This post will also analyse this recent initiative within the context of the long-standing challenges related to rare medicines and evaluate whether this measure could finally bring meaningful change to the lives of affected patients.
To quickly mention the latest developments in the field of rare diseases, the Delhi High Court passed its judgement in Master Arnesh Shaw v. UoI wherein it inter alia directed the government to allocate a sum of Rs. 974 crore to the National Fund for Rare Diseases (NFRD). Meanwhile, Roche has filed a lawsuit against Natco Pharma, seeking a permanent injunction to prevent the launch of a generic version of Risdiplam, a drug used to treat Spinal Muscular Atrophy (SMA), highlighting the ongoing debate over patents and the availability of affordable treatments. Additionally, in the SEBA v. Union of India case (pdf) [a big thanks to an anonymous reader for sharing this development with us], the government has submitted a statement detailing its efforts to promote the indigenous manufacturing of drugs for rare diseases at more affordable prices, as part of the NPRD 2021 after the petitioner’s concerns about the depletion of funds and the uncertainty surrounding the continued supply of necessary medications.
Cash Before Cure?: A Timeline of Developments in the Area of Rare Disease Drugs
Let us first discuss the scheme of events that have taken place regarding the legislative framework for rare diseases in India. Having been unregulated and overlooked for decades, the government launched the National Policy for Rare Diseases (NPRD) in March 2021 for the treatment of rare disease patients after the DHC’s order (2021) and the constitution of a committee for the same effect. A thorough insight into the NPRD’s structure can be seen through the presentation excerpts published by the Indian Organisation for Rare Diseases (IORD) here, and its implications and critiques have been discussed on this blog before here, here, and here. In the NPRD, 57 rare diseases have been identified and categorised into three groups, and financial support of up to Rs. 50 lakhs are provided to patients suffering from any category of rare diseases for treatment at any of the Centres of Excellence (CoE) mentioned in NPRD. Moreover, a digital portal for crowdfunding and voluntary donations for the treatment of patients suffering from rare diseases has been launched. Now let us trace the progress in this domain after the 2021 developments, and whether there were actual developments in this 3-year gap.
In 2021, the Press Information Bureau (PIB) reported that ICMR’s rare diseases registry listed a total of 14778 rare diseases in India. Fast forward to 2024, as checked on 7th November 2024, the number of cases registered in the Rare Diseases Registry on ICMR was a whopping 13,881! This is significant progress that the ministry has made in accounting for all of this.
The Annual Report 2022-23 (pdf) published by the NITI Aayog dedicates one column to ‘orphan drugs and therapies for rare diseases’ (page 13 of the document). It states that more than 7,000 rare diseases are known today, affecting 300 million people worldwide and about 90 million in India alone. The number of disorders with a known molecular basis is rapidly rising, but the number of approved therapies lags far behind. Keeping this in view, it also mentions that a committee on ‘Drugs and Dosage Forms for Rare Diseases: Engagement with Manufacturers’ was constituted with the Member (Health), NITI Aayog as Chair, and representation from the Department of Pharmaceuticals and clinicians to consider the identification of a set of priority disorders/indications and their corresponding treatments that can be enabled for domestic manufacturing of orphan drugs. As of January 2023, the Committee had convened five meetings to engage with a shortlisted set of Active Pharmaceutical Ingredient (API) manufacturers. However, I could not find any conclusions/follow-ups to these claims/developments wrt the Committee in the latest Annual Report.
Instead, the Annual Report 2023-24 (pdf) (released by the MoHFW only a few days back) provides a briefer on all the gradual steps taken by the government in this area, from the policy to the digital portal for fundraising (page 100-101 of the document). As of the date of the report, the MoHFW has identified 12 government tertiary hospitals as CoEs for rare diseases. It claims a total of Rs. 34.99 crore and Rs. 74 crore were to CoEs for treatment of such patients for the FY 2022-23 and 2023-24, respectively.
Recently, homeopathy and AYUSH medication were also claimed to be effective complementary/alternative methods to enhance treatment for rare disorders. However, the Ministry of AYUSH, Government of India’s publication ‘A Decade of Transformative Growth in AYUSH Towards Holistic Health for All 2014-2024’ does not even mention such possibilities or any tangible steps taken to that effect.
Clearly, while there hasn’t been much discussion on the rare disease angle for medication procurement and availability since the 2021 policy was announced, the initiative under the policy’s ambit and the creation of committees have been the major highlights in this area over the past 3 years. The most recent government publications fail to address this aspect at all. It is good to see that the government is refocusing its efforts on this matter of concern, as the problem of drug availability for rare diseases is far from solved.
A Deeper Look into the Circular: Which Drugs Are Being Acquired by the Government?
The following is a preliminary database we’ve put together (curated with significant assistance from Aditi Bansal) with details that we could find regarding the 127 drugs that the government plans to acquire. This is based on information that we could find online and some of it is probably incomplete. And while efforts were made to ensure its accuracy, we request readers to let us know if they find any mistakes in this, by leaving a comment below. This database includes information on the drugs’ usage, major manufacturers in India, and their availability as generic medicines. While this database does not (and neither the circular for that matter) conclusively determine whether these drugs are patented, the information provided is suggestive rather than definitive. It aims to help assess the accuracy of the claim that 120 of these drugs are patented.
From the above database, a few major assertions can be drawn. Firstly, 33 cancer drugs can be identified in the list (highlighted in green), while 3 drugs assist in cancer treatment and surgery (highlighted in blue). This is good to see. Moreover, the list does seem to include several drugs for rare diseases, such as Fabry Disease and Hunter syndrome.
But a really interesting aspect to note in the above database is that many of the drugs also have generics available in the Indian market. In light of this fact, it calls into question most of the news reporting on this matter by major news platforms—though not to pinpoint any specific one, as nearly all have reported the circular in the same manner—that claimed: ‘120 patented drugs are being acquired by the government.’ The official circular also does not mention that all the drugs are ‘patented.’ Moreover, it is rather difficult to conclusively determine that a whopping 17 drugs are for rare diseases. This assertion is not in the circular, and on a cursory review of the above table, it seems to be definitely fewer than 17.
What Could Be a Sure Cure?: A Look at What Still Hampers Affordable Medication for Rare Diseases
The main concern about rare diseases is that treatment is not always present (only 5% of rare diseases worldwide are treatable). If treatment is available, options remain limited and overtly expensive for many to afford. Even commercially, these drugs are not profitable (as discussed in terms of ‘incentive’ by Thomas here) for drug companies since rare diseases affect a relatively small number of people. The sunk cost of R&D for such drugs itself becomes too high, so research for their creation takes a back seat for pharmaceutical industries. There also comes a sense of monopolisation of such medicines once created, which restricts general public access to the drugs through higher costs to recoup the high sunk costs.
As per the MoF’s gazette notification from last March under the Customs Act, 1962, there is full exemption from basic customs duty on all drugs and food for special medical purposes imported for personal use for the treatment of all rare diseases listed under the NPRD. So even though customs duties don’t apply to rare medicines, and a DHC order by Justice Pratibha Singh in the matter of Master Arnesh Shaw v. Union of India (2023) had further reiterated the same, the overall costs still run too high to be affordable. The latest Order in this case does not comment on the aspect of custom exemptions. While a full exemption of tax of this kind acts as a longer-term solution, similar to the earlier one-time decision by the government to facilitate the delivery of the drug Zolgensma by waiving off the import duty and GST amounting to Rs. 6 crores, effective legislative/regulatory change might be more worthwhile than such actions which merely cut off the crust from the patient’s expenditure without any substantial reliefs in the innovation domain.
Moreover, while a digital portal (which seems to be pretty active and updated) for fundraising has been opened, solely fundraising methods are largely insufficient to address the actual concerns related to these drugs. While fundraising may assist singular patients to become financially capable of receiving treatment, it doesn’t create a fund to spur innovation for such expensive drugs. Interestingly, compulsory licensing mechanisms also fail in the cases of medicines serving rare diseases. No aspect of compulsory license (CL) provisions specifically addresses the needs of those with conditions that do not align with ‘larger public demand.’ ‘Orphan drugs,’ by their definition itself in the New Drugs and Clinical Trials Rules, 2019, are ‘drugs intended to treat a condition which affects not more than five lakh persons in India.’ As argued by Akhil Satheesh in an earlier post discussing a case for the grant of a CL to the rare disease drug Zolgensma, the criteria for issuing compulsory licenses should be revised to better address the specific challenges faced by individuals with rare diseases.
In these situations, the government’s efforts to acquire patented medicines to enhance access and affordability are a commendable step. However, there do lie questions about the lack of specificity of the quantity of the drugs that will be procured under the tenders. The Circular is silent on these details. Largely, the effectiveness of this measure will end up depending upon the bids and what decision is finally taken on the disbursement of funds towards this cause by the MoF. Nevertheless, this approach is likely to have a more significant impact than merely superficial financial assistance to isolated patients.