Strengthening the Access to Rare Disease Treatment in India

In November 2021, Takeda Pharmaceutical Company Limited, a global values-based, R&D-driven biopharmaceutical leader, together with UNGCNI launched a national initiative for improving early access to treatment for Rare Disease Patients in India. The joint rare disease initiative took charge of addressing the challenges to ensure access to treatment for rare disease patients waiting on the crowdfunding portal in an integrated manner. Through the initiative, 300+ PSUs and Corporate leaders were reached, which has helped to generate a pool of insights and recommendations that will help in shaping the future of access to rare disease treatment in the country.

The 6-months intensive initiative has been able to bring together stakeholder groups (private sector, PSUs, corporate giants, etc.) that can support mobilizing and operationalizing funding for the rare disease under CSR grants and donations for strengthening the existing crowdfunding mechanism and pathways set in the India Rare Disease Policy. Furthermore, it has created the roadmap for addressing rare disease funding requirements in alignment with the Government policies and guidelines Ministry of Corporate Affairs, Department of Public Enterprises, and Ministry of Health and Family Welfare, Govt of India. 

The eminent experts from public and private sector organizations participated in the initiatives including MoHFW, AIIMS (New Delhi), Kerala State Legal Services Authority (KELSA), CoEs (SGPGI, Lucknow, KEM, Mumbai, SSKM, Kolkata), and 24 PSUs and Corporate Leaders (e.g., BHEL, Bharat Petroleum, ONGC etc).

Mr. Aseem Kumar, Officiating Executive Director, UNGCNI said, “Rare diseases, underpin a situation where health lies at an intersection of several Sustainable Development Goals, going well beyond only SDG 3 (Good Health and Well-being). The need to address rare diseases on priority was made a reality in 2019 when UN Member States adopted the historical Political Declaration on Universal Health Coverage. This included a commitment to strengthen efforts to address rare diseases as it was for the first-time that rare diseases were included within a UN declaration. This   helped in channelizing collaborative efforts with multi-stakeholders to strengthen the national rare disease policy execution”.

Ms. Serina Fischer, General Manager, Takeda India says, “Patients remain at the core of decision making at Takeda and we are fully committed to the fight against rare diseases by advancing treatments and addressing diagnostic delays and gaps in access to treatment. The need of the hour is to bring all stakeholders together for rare to strengthen capacity-building efforts and find sustainable funding mechanisms that ensure the treatment of people living with rare diseases. We are pleased that our association with UNGCNI has successfully generated insights and mobilized support which in the long run will be important in solving issues related to access of treatment for rare disease patients.”

“Rare disease presents itself as one of the most challenging health areas to be tackled. The first step to handling rare diseases for India was the release of NRDP in 2021 which has given direction to the efforts of rare disease players to start with. But, as in any health area, policy implementation faces its set of challenges, especially when policy provisions are there but awareness about those provisions is absent or blurred by perceived challenges. Therefore, for inspiring change in the rare disease ecosystem, it is important for policymakers, payors, providers, and patients to come together as one unit and work using the continuum of care approach for applying integrated solutions”, Dr. Ruchi Sogarwal, Head-Public Affairs & Patient Advocacy, Takeda India. 

Mr. Ajeet Kumar Sharma, AGM, Bharat Heavy Electricals Ltd. & Secretary, UNGCNI expressed, “CPSEs are key players in CSR domain, working relentlessly for equitable wellbeing of society through their CSR initiatives. They have the needed capacity of extending help to the needy patients suffering from rare diseases. However, there is a need for enhancing awareness and understating of rare diseases, issuing of appropriate guidelines & directions by concerned government agencies and enhanced connect between Centers of Excellence and CPSEs. This initiative has immensely helped in clarifying the perceived barriers in flow of CSR fund from CPSEs towards this cause.”

“The Government of India’s support to rare diseases in form of the National Policy for Rare disease-2021 is laudable and it has made rare diseases a national priority. The step has been vital in showcasing India's commitment not only to addressing the needs of the rare disease patients but also to achieving universal health coverage (UHC). The onus now lies on us as citizens of the country, be it individuals, large or small organizations, institutions or the general public to support the Government Notified Centre of Excellences in the smooth implementation of the policy. UN-GCNI is dedicated to Improving Early Access to Treatment for Rare Disease Patients by encouraging the stakeholders such as PSUs, CSR mandated organizations, corporates, and experts to come together in support of underserved patients, with the firm belief that UHC will never be completely realized if people living with rare illnesses are left behind”, Dr. Somnath Singh, Programme Manager, UNGCNI.