Rajya Sabha MP Haris Beeran wrote to the Minister of Health and Family Welfare on December 20, 2024, urging the Central Government to invoke Section 100 (1) of the Patents Act with respect to local production of the rare disease Spinal Muscular Atrophy (SMA) treating drug Risdiplam. SMA is a genetic disease affecting the nerve cells that control voluntary muscle movement. SMA incidence in India is one in 10,000 live-born babies. Section 100 empowers the central government to use, or authorise to use, an invention (application or grant) for the purposes of the government, on a non-commercial basis. Importantly, this is not a provision that requires abuse of patent by the patentee. This includes but is not limited to using the invention in case of national emergency or other situations of extreme urgency. Risdiplam is currently under patent protection in India till May 11, 2035 (subject to payment of renewal fees) and is registered as Indian Patent No. IN 334397.
The Letter to MoHFW
The letter has requested for the production of Risdipalm by way of invoking Section 100, Patents Act to make the medicine more accessible. The letter points out that the maximum retail price for Risdiplam is Rs. 6,20,835 per bottle and therefore the total cost can reach Rs. 72 lakhs for children (12 bottles) and Rs 1.86 crores for adults (30 bottles). The letter also refers to an expert study in its annex that states Risdiplam, being a small molecule drug, its generic version can be made available to Indian patients for just Rs 3024 per year through local production. We could not find the annex but in all likelihood the letter is referring to the study by Yale Researcher Melissa Barber (more on this below). The invocation of Section 100, as the letter states, will operationalize Para XI of the Implementation Strategy, National Policy for Rare Diseases, 2021 (NPRD). The aforesaid para refers to the promotion of local development and manufacture of drugs for rare diseases by public and private sector pharmaceutical companies at affordable prices and taking legal/legislative measures for creating an environment for local development. The letter also requests research and development funds for the development of low-cost gene therapy Spinraza used in treating SMA, taking measures for screening of SMA along with establishing a national registry for SMA and other rare diseases, and establishing a national pool procurement of medicines and diagnostics for SMA and all other rare diseases.
The Letter from MP Haris Beeran to the Minister of Health and Family Welfare
Legal Development in Rare Disease Medicines
In a case concerning access to medicines for rare diseases, the Delhi HC in Master Arnesh Shaw v Union of India reiterated that the Right to Health is an integral part of Article 21 under the Constitution. (Also read Sunidhi’s post analysing the question of judicial overreach in Master Arnesh Shaw.) Previously, in Association of Medical Super Speciality Aspirants and Residents v. Union of India (2019), the Supreme Court categorically held that the Government has the constitutional obligation to provide health facilities. Amongst other issues, the Delhi High Court in the Arnesh Shaw case had identified three major problems with respect to rare disease treatment – first, non-identification of rare diseases in a timely manner; second, insufficient measures for indigenous research; third, non-invocation of measures under the law to ensure manufacturing of existing medicines in India. The judgment also pointed out the use of Section 100 to enforce production locally. Although the case did not deal with SMA specifically, but rare diseases in general, the SMA Foundation in Arnesh Shaw had submitted that there were currently 1800 patients with sensitive conditions. For SMA treatment, three drugs are primarily available and used – Zolgensma, Spinraza, and Risdiplam. Zolgensma is for infants under the age of 2 and is given as a one-time dose. Risdiplam is the cheapest of the three but still exorbitantly priced at Rs. 72,00,000 per patient/year under the Patient Support Program by Roche! The judgment also noted price negotiations between the government and Roche for Risdiplam on June 24th, 2024, accepted by the National Rare Diseases’ Committee, and that procurements and approvals would now be commenced. However, no details are present in the public domain.
Another similar petition (Seba P.A v Union of India – WP(C) No. 43275/2023) with respect to the treatment of SMA for a 24-year-old petitioner has been pending before the Kerala HC. The Court’s order on February 23, 2024, had directed the Union government to release Rs. 50 lakhs, the minimum amount she was entitled to under the National Policy of Rare Disease. In its order on June 3, 2024, the Court directed the government for a statement to be filed by the government with respect to the steps undertaken under the NPRD. The government had thereafter submitted a statement detailing its efforts. In its latest order dated November 29, 2024, the Court had directed the Centre of Excellence to continue the treatment without break, by procuring the requisite quantity of the drug, as an interim measure. The next date of hearing is today, i.e. January 20, 2025.
Local Development, Low Cost of Production, and the National Policy on Rare Disease
A paper by Yale Researcher Dr Melissa Barber has stated that Risdiplam is likely to be the only treatment, amongst other SMA treatments, for which access in LMICs could be realistically possible. Barber has claimed that Risdiplam in particular can cost as low as Rs. 3024 per patient/year versus the current pricing. Dr Barber had also shared her findings with the Kerala High Court on request by the Petitioner in Seba P.A. For the estimation of production costs, Dr. Barber’s costing algorithm includes costs of materials (API, excipients, vials) formulation and secondary packaging costs, a 20% mark-up and tax on profit. Explaining her conclusion, Dr. Barber has stated that API price is the ‘most important determinant’ in the cost of medicine production, and for Risdiplam ‘production in small volumes is feasible and cost-effective.’ She also takes into account other considerations such as supply chain and health system which include issues of refrigeration, storage, and ease of administration. To me, the factors and the calculations by Dr Barber, do make for a sound argument for a cheaper production cost. Notably, Dr Barber’s research on Risdiplam was part of her request to the 24th Expert Committee on the Selection and Use of Essential Medicines (WHO) to include Risdiplam for SMA. Do have a look at her estimations below.
Image 1. Extracts from Dr Barber’s submission to the Kerala HC in Seba P.A.
Image 2. Extracts from Dr Barber’s submission to the Kerala HC in Seba P.A.
Image 3. Extracts from Dr Barber’s submission to the Kerala HC in Seba P.A.
Image 4. Extracts from Dr Barber’s submission to the Kerala HC in Seba P.A.
Talking about local production for Risdiplam, several patent applications are pending by manufacturers like Natco, Harman Finochem Limited, and MSN Laboratories, awaiting examination or request for examination. At the same time, Roche had filed a petition in the Delhi HC seeking an injunction on the launch of the generic version of the drug by Natco. Over the course of the hearings in this case, the intervenors (which includes Seba P.A.) argued for a pro-public dimension that has been considered in the traditional test to grant/refuse an injunction. Interestingly, the intervenors also argued that for Risdiplam, many key discoveries emerged from publicly funded research. Therefore, the defence of massive R&D investment, which is usually relied upon by big pharmaceutical companies like Roche for patent protection and charging a fortune for the invention, should not be applicable here. The hearing in the case is now over and the judgment was reserved on December 24, 2024.
Varsha and Surbhi have written here and here on issues with the National Policy on Rare Disease (NPRD). Amongst others, the primary issue seems to be the failure to ensure indigenous research and manufacture or make bulk purchases and import drugs at lower negotiated prices. The financial support scheme of 50 lakhs (amended from 20 lakhs in 2022) is an insufficient and irrational ceiling. For some, as the Court noted in Arnesh Shaw as well, the cost may go way over 50 lakhs and for some, it may be less. A case-to-case approach is needed to be adopted for the same to be effectively implemented. One of the absurd additions to the NPRD was the use of a crowdfunding portal. Till the Arnesh Shaw judgment, an abysmally low amount of Rs. 349,280/- was collected on the crowdfunding platform. As Varsha and Surbhi note, crowdfunding is neither a short-term nor a long-term sustainable solution. The issue of treating patients is not a one-off situation, rare diseases will keep increasing, and so will the patient number, a long-term issue that is far from being solved by crowdfunding.
The Australian Government Initiative
A Reddit thread (published on January 9) took on the issue of SMA drug pricing in India on the platform. It compared the prices in India and Australia and surprisingly (or not), drugs such as Zolgensma (Onasemnogene Abeparvovec) covered under the PBS (Pharmaceutical Benefits Scheme) in Australia cost as low as $31.60, although the DPMQ (Dispensed Price for Maximum Quantity) is $2.5 million! The DPMQ represents the amount paid by the PBS when a pharmacist or supplier dispenses the maximum quantity of medication. Similarly, Risdiplam’s patient charge is $31.60 as well, with the DPMQ being $10841.89. (Screenshots attached below)
Is a similar wide coverage of critical medicine possible in India?
Cost of Risdiplam on the PBS Website
Cost of Zolgensma on the PBS Website
Kerala Rare Disease Policy and Health On State List
In February 2024, the Kerala government launched KARe (Kerala Against Rare Diseases) program aimed at providing free treatment up to a ceiling limit of Rs 50 lakhs. In April, it was reported that free treatment was given to children under 12 suffering from SMA. Health currently features on the state list which also means that several central government health schemes may or may not be implemented by the states. The 15th Finance Commission Chairman N.K. Singh had recommended that health should be moved to the concurrent list for better regulation and implementation. Could both the state and national policies be combined to formulate an effective delivery in tackling pressing issues on health such as rare diseases?
Use of Section 100, Patents Act
As discussed before, Section 100 empowers the government to use an invention for its purpose either by itself or by another entity authorised by it. Now that we know that Natco is facing an infringement suit for Risdiplam, it can be one of the entities authorised to produce the same. However, the government’s negotiation (as noted in Arnesh Shaw) with Roche for the pricing of Risdiplam will not create a compelling case for the use of Section 100. The lack of transparency on what exactly the negotiation was and how long it would take to be brought into effect remains a question and does not help the cause of the petitioners either. Alternatively, it will be interesting to see the judgment in Roche’s infringement suit against Natco. Either way, the Court’s decision will more or less set the ground for future actions. Tejaswini (here) noted the difficulties in invoking Section 100 and the repercussions such a step might have which included its impact on bilateral relations. As effective as the prospect may sound, Section 100 may not be the best solution for Risdiplam right now, especially given the Roche negotiation discussed above and factoring in the government’s intent to use the provision. Instead, if such a negotiation has been reached, the details of it must be sought to ascertain how viable the solution would be. However, these discussions make me wonder if the government will be able to use these levers within the Patents Act at all. The threat of bilateral consequences or similar allied issues will never be absent, how then the Right to Health will be guaranteed by the State, if not through its own enacted laws? Once touted as a uniquely modeled patent law system owing to the several flexibilities inbuilt, these flexibilities have rarely been exercised. Can it now be exercised for rare disease medicines?