India’s new draft national policy for rare disease and the road to accessing gene therapies

By Ciaran Cassidy, Snr Analyst

Email: [email protected]

With one of the fastest-growing economies and the second largest population in the world India is a market of increasing interest to the biopharmaceutical industry.^1,2 However, the potential for access to innovative rare disease therapies, such as gene therapies, is not particularly well supported by the central government and third parties may be responsible for its access in India.

While there is no official definition attributed to orphan drugs, the 2019 Ministry of Health’s (MoH) policy draft is the first to provide some clarity. The rules on the regulatory frameworks for clinical trials refer to orphan drugs as those intended to treat conditions affecting fewer than 200,000 people.³

The MOH also stipulates that orphan drugs are exempt from price controls but has yet to publish the list of drugs it would allow to be freely-priced, which suggests this could be decided on a case by case basis.⁴Despite the potential free pricing of orphan drugs, without a fully developed and easy to understand policy on orphan drug access manufacturers may lack incentive to consider MoH submission in India.

In February this year, a draft of the national policy on rare disease was debated in the Indian parliament including some significant intentions towards expanding rare disease funding.

Known as the `National Policy on Treatment of Rare Diseases’, this draft policy seeks to assist patients who are undergoing treatment for rare diseases categorised under three groups:⁵

Despite the draft policy’s move to increase access to rare disease therapies, the funding limit specified for group one in particular is very unlikely to suffice for gene therapies, which are currently priced in excess of hundreds of thousands or even millions of (US) dollars⁶.

Current Government Funding for Rare Diseases

The MoH and various state health ministries have set up technical committees, known as Central Technical Committees (CTCs) and State Technical Committees (STCs) respectively, to review rare disease patient applications for financial assistance.⁴

In India, it is also the case that courts can order the government to cover the cost of an ‘essential medicine’ if failing to access treatment would violate their right to health.

In a landmark court case in 2013, (Mohd. Ahmed (Minor) v. Union of India & Ors W.P.(C) No. 7279 of 2013), the Delhi High Court directed the Government to provide Mohd. Ahmed (minor patient in question) with enzyme replacement therapy (ERT) worth INR 600,000 (USD 8,334) per month as a treatment to Gaucher’s disease.⁴

However, the court’s reasoning for the judgement included arguments which may not extend to many orphan drugs, particularly gene therapies with high uncertainty of long-term outcomes.

“Whether a minor child born to parents belonging to the economically weaker section of the society suffering from a chronic and rare disease, Gaucher, is entitled to free medical treatment costing about six lakhs rupees per month especially when the treatment is known, the prognosis is good and there is every likelihood of petitioner leading a normal life”.⁷

Despite the government’s progress on a rare disease policy and the judicial system defending the right to access healthcare, the high-cost innovative therapies, such as gene therapies, may become more accessible via alternative routes.

Alternative Funding for Rare Diseases

One source of alternative funding for high-cost therapies would be from private insurers, which covers 9% of the total population.⁸ For example, Royal Sundaram’s Family Plus policy covers sums from 300,000-50,000,000RS (~4,000-660,000USD), which can be ‘reloaded’ once per policy year.⁹ Even though this is substantially more than current government funding available through CTCs this is restricted to a small part of the population and much lower than private funding in more developed markets.¹⁰ However, when considering the size of India’s population, the private market may be able to still provide a target for manufacturers with an appetite for a lower price in exchange for patient volume.

Another source of alternative funding for rare disease therapies is Corporate Social Responsibility (CSR) programs offered by companies such as Reliance Industries whose hospital specialises in complex diseases.¹¹ Through subsidized and free services, the Reliance Industries’ hospital benefitted 10,000 out-patients in 2018 alone.¹² There are numerous CSR programs and hospitals in India with rare disease expertise and autonomy.¹¹ A key consideration here is that India has a well-developed import route for autonomous hospitals to access innovative therapies not yet approved or launched in India, which may lead to CSR hospitals being some of the leading access points for gene therapies in India.

Import Licences for Therapies Unlicenced into India

An import license can be obtained for small quantities of an unlicensed drug via the Central Drugs Standard Control Organisation (CDSCO) at a national level. To gain approval for a Small Quantity license the therapy would need to be intended for patients suffering from life-threatening diseases, or diseases causing serious permanent disability, or such diseases requiring therapies for unmet medical needs. The therapy would also require marketing authorisation in the country of origin.^4,13

These can be imported by Government Hospitals or Autonomous Medical Institutions and shall not exceed 100 average doses per patient, but in exceptional circumstances, the CDSCO may sanction a larger quantity of a drug. ^4,13 With a large enough budget and potentially a strong relationship with a manufacturer, CSR hospitals may be able to source innovative rare disease therapies before the government’s plan can provide access to the higher cost therapies such as gene therapies.

Looking forward

With an increasing economic development and a focus on expanding healthcare, including care for rare disease patients, India is a key market of interest to manufacturers of innovative therapies. However, considering the current funding and policies from the government the expanding healthcare for rare disease patients does not appear likely to include gene therapies anytime soon. Despite this it will be interesting to see how alternative routes of access both for patients and manufacturers, are utilised in the near future and whether these can include substantial access to gene therapies.

References

1. Zhang 2019. India’s economy is a big issue during elections — Here’s how it’s growing at a faster pace than China {Accessed online} Available at: https://www.cnbc.com/2019/04/18/how-indias-economy-went-from-weak-to-fastest-growing-in-the-world.html
2. Rodwin VG, et al., (2018) BRIC Health Systems and Big Pharma: A Challenge for Health Policy and Management [Accessed Online] Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5890064/
3. Jishnu 2019. Encouraging Orphan Drugs in India [Accessed Online] Available at: https://www.downtoearth.org.in/blog/health/encouraging-orphan-drugs-in-india-67581
4. Shkula , Punnen, Antani. 2020 Orphan Drugs & Rare Diseases Nishith Desai & Associates/India [Accessed Online] Available at: https://pharmaboardroom.com/legal-articles/orphan-drugs-rare-diseases-india/
5. Government of India. National Policy for Treatment of Rare Diseases [Accessed Online] Available at: https://main.mohfw.gov.in/sites/default/files/Rare%20Diseases%20Policy%20FINAL.pdf
6. Love 2019. Why Didn’t non-profits and the NIH require ‘reasonable’ pricing for Zolgensma? That may happen in France [Accessed Online] Available at: https://www.statnews.com/2019/09/18/zolgensma-reasonable-pricing-france/
7. Bhatia 2014. Indian Constitutional Law and Philosophy [Accessed Online] Available at: https://indconlawphil.wordpress.com/2014/04/17/delhi-high-court-rules-on-article-21-and-access-to-medicine/
8. Keelery 2020. Number of people with health insurance across India from financial year 2014-2019, by business type [Accessed Online] Available at: https://www.statista.com/statistics/657244/number-of-people-with-health-insurance-india/
9. Royal Sundaram 2014. Family Plus Policy Book [Accessed Online] Available at: https://www.royalsundaram.in/documents/20147/0/FAMILY-PLUS-POLICY-BOOK.pdf/1d87ebcf-092a-5789-b893-94f17291a369
10. Insurance Information Institute. World Insurance Marketplace [Accessed Online 2020] Available at: https://www.iii.org/publications/insurance-handbook/economic-and-financial-data/world-insurance-marketplace
11. CSRBOX 2018. Top 10 Healthcare CSR Project in India in 2018 [Accessed Online] Available at: https://csrbox.org/India_CSR_news_Top-10-Healthcare-CSR-Projects-in-India-in-2018_367
12. CSRBOX. CSR Project by: Reliance Industries Ltd [Accessed Online 2020] Available at: https://csrbox.org/India_CSR_Project_Sir-HN-Reliance-Foundation-and-Research-Centre-Maharashtra_6992
13. Malhorta and Punia 2019. Growing Menace of Unlicensed and Spurious Drugs [Accessed Online] Available at: https://www.vantageasia.com/spurious-drugs-unlicensed/
14. 14.  Gazette Notification GSR no. 604 € dated 24.08.2001 [Accessed Online] Available at: https://www.drugscontrol.org/import-registration.php