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With just 4.2% of its GDP being spent on healthcare in 2015-2016 – and a paltry 1.2% of GDP being public health spending – optimal healthcare for patients in India seems like a distant dream. Patients afflicted with rare diseases bear the physical and economic brunt of these conditions. In all, an estimated 72 million Indians have what is commonly defined as an orphan disease vs. 30 million seen for the United States. However, this seemingly large opportunity is being undercut by the underlying cost of orphan drugs, non-existent orphan drug policies, lack of awareness and unavailability of extensive diagnostics. As a result, patients with these diseases in India often fail to receive the care that they need.

The orphan drug market in India offers a great deal of potential but the lack of federal or state policies makes it inaccessible to pharmaceutical companies. However, recent events suggest that this might change soon in Karnataka – a state in South India – with the possibility of spreading to other parts of India.

Karnataka Vision Group on Biotechnology (VGBT), a state government organization, is working with various stakeholders including the Organization of Rare Disease India (ORDI), a non-profit organization, to draft a state orphan drug policy. Dr. Harsha Karur Rajasimha, one of the co-founders of ORDI, spoke with Decision Resources Group about the relevance of an orphan drug policy for an emerging market like India. He pointed to countries like the United States that were early adopters of orphan drug acts, adding that India and emerging markets must innovate on the ODA model to fit their biopharmaceutical and healthcare ecosystems. “Indian pharma is mostly reliant on low-cost biosimilar drugs with a majority of those resulting from R&D efforts in the US and EU where ODA provides incentives for an otherwise economically inviable investment in orphan drugs R&D,” he says. “With no incentives in India for investments in orphan drugs R&D, any orphan drug policy framework would need to consider making existing FDA approved orphan drugs accessible and affordable for patients with rare diseases in India.”

“The GOI (government of India) can allow duty-free import of orphan drugs into India and provide other incentives (e.g., sales tax exemption, etc.). The GOI also needs to take the lead in setting up patient registries for all rare diseases to help priorities the need for orphan drugs by disease area and by population. For the longer term, GOI also needs to adopt a policy that encourages innovation in orphan drugs research.”

The draft input provided by ORDI to Karnataka VGBT focuses on addressing these issues as well as providing free healthcare to patients with chronic and debilitating rare diseases. Currently, the government hospitals provide free healthcare to patients (usually from lower income categories) with common ailments. “However, for patients with rare diseases, often the drugs have to be imported and are extremely expensive,” Rajasimha says. “Hence the government is refusing to provide free treatments to such patients, leaving the patients and their families bankrupt and suffering.”

There have been some recent court judgments in support of the patients instructing the government to provide treatments to such patients. As a result, key aspects of both the orphan drugs policy and rare disease policy are included as objectives in the Karnataka draft.

Rajasimha hopes Karnataka’s health secretary builds upon the draft put forth by VGBT and becomes the first state in the country to adopt such a policy framework. While the average time to diagnose a rare disease in the United States or European Union is still 5-7 years, it is much longer in India. Reducing this time lag in India is particularly key with next-generation sequencing technologies that allow for diagnosing hundreds of rare diseases in a single test. Making such diagnostic tests more widely available and known to physicians will be part of his group’s policy draft. “This has tremendous applications for newborn screening for all known genetic tests if we can make them affordable,” he says.

The Karnataka Vision Group for Biotechnology had aimed to implement the Act by the end of 2015, but obstacles emerged. Nonetheless, the legislation appears to be moving its way through the process.

Ultimately, the policy could become a benchmark for other states across India. Rajasimha believes that GOK (government of Karnataka) can serve as an example for other states nationwide, anticipating that the policy will continuously evolve. “We have no excuse for not having a policy at all, which is the current status,” he said. ORDI has already initiated discussions with a number of states, pushing for a collaborative approach with relevant NGOs, disease specific patient advocacy groups, key opinion leaders, academicians, hospitals and government agencies. Such an approach, he believes, provides a single, strong voice rather than distributed isolated efforts that can be ignored.

If this policy is implemented, Karnataka would serve as an example to other parts of India. As these policies take root, rare disease patients and global pharmaceutical companies will benefit in treating serving this large population.

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