With the significant burden imposed by the life-threatening (mostly progressing) rare diseases, they present a fundamental threat to the health systems. This requires a streamlined approach to ensure a timely and quality health support to patients with rare diseases.
Not just the global figures, India also portrays a complex picture wherein it shares a significant proportion of rare diseases, with estimates of over 70 million people with rare genetic disorders. While on one hand, these numbers lack a validation in form of pan India primary evidences, there are also challenges as only few of these diseases have their treatments available and approved in India.
Highlighting this, the Health Issues India (HII) hosted National Roundtable Discussion on Rare Diseases Policy: Call to Action on 27th July 2019 in New Delhi. It brought to fore the imperative need for a National Rare Diseases Policy and potential implementation strategies for catering to the needs of the rare diseases’ patients.
Pointing towards the recent policy shift towards rare diseases, Dr. Eshwara Reddy, Drug Controller General of India, stressed on the first-time incorporation of definition of orphan drug in the Drugs and Cosmetics Act. Mentioning the need for a sustained finance system, he mentioned the government’s support in provisioning treatment by waving off clinical trials for molecules/ treatment approved in identified countries and a waiver of five lakhs for registration and clinical trials.
In lieu of the National Health Policy for Treatment of Rare Diseases formulated in 2017, a holistic policy on rare diseases is expected to be announced in November 2019. In this regard, the panellist discussed the aspects such as awareness generation, patient centric policies, centre-state coordination and a sustainable economic model to support patients with rare diseases.
Regarding the need of clear guidelines for the state-centre relationship, the participants deliberated on the role states should play in the health-related issues and not solely rely on the centre. So far, the state of Karnataka has been providing treatments for individual cases, and patient advocacy through courts has had a role to play in this. This leaves a lot of scope for other states to learn from. The states need to work in tandem with the centre government in implementing a smooth functioning of the proposed policy and an improved healthcare model.
Dr. Sonali Rawal, Technical Consultant- Health, NITI Aayog, said that in the resource constrained setting like that in India, it is important to formulate policies that are implementation centric. Hence, it is important to set immediate, mid and long-term goals for rare disease policy as well.
The panellists also discussed other funding alternatives which involve the scope of a dedicated CSR funding and PSUs to reduce the burden from the state and centre.
Dr. Dr G N Sanjeeva, Professor, IGICH Bengaluru, emphasized on the need of a clear funding methodology and pointed at the lack of institutional mechanisms to predict the financial requirements. He further added that although several patient organisations are focused on raising awareness and compassion for people living with a rare disease across India, we need a simplified process for data gathering and dissemination of the funds. Given the varied nature of rare diseases setting preliminary upper limits on disbursement may fail the inherent idea of a corpus fund.
Rare diseases are complex, heterogeneous, continuously evolving set of diseases that has grappled with a chronic lack of medical and scientific knowledge and research, inadequate awareness and adequate funds. Hence, the time is ripe to transition towards a patient centric policy embedded in right data and research, facilitating smooth implementation.
The discussion was moderated by Professor Amir Ullah Khan, Former Head of Research, Bill and Mellinda Gates Foundation and Professor, MCRHRDI, Government of Telangana. It was also attended by other prominent experts such as, Dr. S.K. Arora, Additional Director (Public Health) Department of Health and Family Welfare, State of Delhi, Dr. Madhulika Kabra, Professor, Division of Genetics, Department of Pediatrics, AIIMS, New Delhi, Dr. Anshu Sharma, Head Centre for Non-Communicable Diseases and Biochemistry, NCDC, Dr. Sailesh Mohan, Joint Director, Centre for Chronic Conditions and Injuries (CCCI) and patient group representatives such as, ORDI, LSDSS and GNE Myopathy.