India Rare Disease Policy Proposes Crowdfunding But Patients Feel Let Down
Executive Summary
Draft policy moots crowdfunding for high-cost rare diseases alongside financial support for those "amenable to one-time treatment" but falls way short of some patient groups' expectations.
India’s draft National Policy for Rare Diseases 2020 has outlined plans for financial assistance to treat specific disorders while also proposing “voluntary crowdfunding” for certain high-cost rare diseases, an idea that has been derided by some.
The draft, which some patient groups see as a significant let-down, moots financial support of up to INR1.5m ($21,118) for the treatment of those rare diseases "amenable to one-time treatment" and specified under Group I, which comprises disorders that can be treated through hematopoietic stem cell transplantation and organ transplantation.
Financial assistance will be provided under the government’s Rashtriya Arogaya Nidhi scheme, which essentially helps below poverty line (BPL) patients ailing with major life threatening diseases to receive medical treatment at any of the super specialty government hospitals/institutes.
The draft policy clarified that beneficiaries would not be limited to BPL families but extended to 40% of the population “eligible as per norms” of the Pradhan Mantri Jan Arogya Yojana (PMJAY), for treatment in government tertiary hospitals only. Launched in September 2018, PMJAY, also dubbed "Modicare", plans to provide health insurance cover of up to INR500,000 per family per year.
The scheme aims to cover about 500 million beneficiaries for secondary and tertiary care at all public and empanelled private hospitals in India, making it the world's largest government-funded health care program by number of beneficiaries.
Crowdfunding Plans
The rare disease policy draft - open for public comments until 10 February - also outlines plans to tap into alternative financing sources for such disorders. It proposes crowdfunding as an option, explaining that resource constraints and competing health priorities make it difficult for the government to fully finance treatment of high-cost rare diseases.
“The gap can be filled by creating a digital platform for bringing together notified hospitals where such patients are receiving treatment or come for treatment, on the one hand, and prospective individual or corporate donors willing to support treatment of such patients,” the policy draft stated.
Under the proposed initiative, notified hospitals are to share information relating to the patients, their rare disease, prognosis, estimated cost of treatment and details of bank accounts for donation/contribution through an online system. “Donors will be able to view the details of patients and donate funds to a particular hospital. This will enable donors from various sections of the society to donate funds, which will be utilized for treatment of patients suffering from rare diseases, especially those under category III,” the draft specified.
This category refers to rare diseases for which definitive treatment is available but challenges entail ensuring optimal patient selection, very high-cost and lifelong therapy.
Lacks Definitive Measures
Some patient groups, however, were disappointed at the seeming half-hearted policy initiatives. Prasanna Shirol, co-founder of the Organization for Rare Diseases India (ORDI), a national organization representing patients with rare diseases in the country, told the Pink Sheet that the draft was “no way near the expectations” of rare disease patients “nor serves any purpose.”
“While the direction is right, it lacks definitive measures, clear guidelines and [provides] no financial commitment,” Shirol maintained.
In 2017, India’s Ministry of Health and Family Welfare (MHFW) had formulated a National Policy for the treatment of Rare Diseases, proposing at the time a corpus fund with an initial amount of INR1bn towards financing treatment of rare genetic diseases, though the new draft has no specific mention of this.
Shirol explained that while the financial assistance proposed is “useful” in a few conditions, it is largely insufficient for rare disease - even one time treatments or solutions like liver transplantation require regular and often life-long medicines and supportive care which are not addressed, he said.
The ORDI co-founder also saw little merit in the crowdfunding proposal, terming it a “joke”. “It is indirectly saying that, we cannot treat and [patients] are being left to one’s fate,” he lamented, emphasizing the need for government funding.
Harsha Rajasimha, founder and chairman of IndoUSrare, a non-profit organization focused on helping patients with rare diseases of Indian origin in the US, India and globally, said that the policy “leaves out a lot of the unmet needs” of rare disease patients communities. "We urge patients' communities in India to now approach the ministry of health and family welfare officials and policymakers to express their concerns and seek to have a more active voice in amending the policy to meet their needs," Rajasimha, also the founder of Jeeva Informatics Solutions, a life sciences informatics firm, told the Pink Sheet.
He added that IndoUSrare could provide policy expertise and perspectives from the US if that can help.
'Part Of A Broader Strategy'
Other experts were, however, more optimistic of the proposed financing initiatives. Chetan Desai, chief medical officer, India, and senior medical director, medical ethics & research, IQVIA, said that the draft policy acknowledges the challenges - resource constraints and competing health priorities - in providing affordable healthcare to patients suffering from rare diseases and outlines various measures to improve access to patients, voluntary crowdfunding being only one of them.
“It should be seen as part of a broader strategy that includes the setting up of centers of excellence at premier government hospitals and financial support from both central and state governments,” Desai told the Pink Sheet.
Public health and hospitals is a state subject in India and the draft policy urges state governments to consider “supporting” patients of rare diseases that can be managed with special diets or hormonal supplements or other relatively low -cost interventions listed in group II of the draft. This group lists several such rare disorders including phenylketonuria (PKU), Non-PKU hyperphenylalaninemia conditions and osteogenesis imperfecta.
The central government expects to support the state governments in implementation of a targeted preventive strategy; this encompasses education of the general public and health professionals at large but more specifically at the preconception stage, antenatal, neonatal and even after disease identification at various levels.
The IQVIA executive also explained that considering the nature of rare diseases, limited awareness and high cost of therapy, there is need for greater public private collaboration among various stakeholders – including pharmaceutical companies, researchers and private and government healthcare providers. “Private organizations can also contribute significantly to greater disease awareness through their Corporate Social Responsibility programs,” he said, also referring to IQVIA’s own effort where it has partnered with the ORDI for four consecutive years to sponsor Race for 7.
The event is a 7km walk/run to raise awareness for the 7,000 known rare diseases and the estimated 70 million rare disease patients in India.
Rare Disease Definition
Significantly, the draft policy has also decided against laying out a specific definition of rare disease in India, at least for now. It notes the varying definitions of rare diseases internationally and emphasizes the need for further research to better understand the extent of the existing diversity of definitions for rare diseases and to examine the scope of arriving at a definition which is best suited to conditions in India.
“It shall be done on a priority basis as soon as sufficient data is available. Steps have already been taken for creation of a registry of rare diseases in India by the Indian Council of Medical Research (ICMR),” the draft said.
Key opinion leaders had earlier adopted a definition for rare diseases in the country, specifying that this covers a disease or disorder that affects fewer than one in 2,500 individuals. However, that does not appear to stand going by the new draft policy. (Also see "India Pushing Ahead With Rare Disease Policy Plan" - Pink Sheet, 16 May, 2017.)
The government now expects to have a National Registry for Rare Diseases at the ICMR with the objective of creating a database of various rare diseases. Over a period of time, the registry is expected to yield enough information to be able to arrive at a definition of rare diseases best suited for India, the draft added.
IndoUSrare’s Rajasimha said that the policy seems to define a rare disease narrowly, covering only a few hundred well-known, diagnosable and treatable diseases within India. “This leaves out a vast majority of patients who either remain undiagnosed for a long time, or those other known rare diseases not covered under the policy framework. It is not clear if, when and how new diseases will be added to the list,” he said.
IQVIA’s Desai was nuanced, suggesting that a national registry will help generate data on disease prevalence that is crucial for health policy makers to plan and prioritize interventions. “The plan to set up select centers of excellence at premier Government hospitals for comprehensive management of rare diseases is welcome,” he said.
The IQVIA executive also noted that the New Drugs & Clinical Trial Rules, 2019 have clearly defined an “orphan drug” for the first time in the Indian context and included a provision for expedited review and local trial waiver for orphan drugs. The rules specify that an orphan drug is one intended to treat a condition which affects not more than 500,000 persons in India.
“A robust rare drug policy, coupled with a conducive regulatory environment, will go a long way in improving access to novel therapies for rare (orphan) diseases,” Desai said.
He also believes that there exists tremendous scope for greater public private partnership to help create more awareness, improve screening and diagnosis, accelerate research and improve patient access.