Battling rare diseases

Improving access to drugs for treating rare diseases and critical-care conditions is of paramount importance. Although the draft National Policy for Rare Diseases (2020) seeks to address such concerns, it contains certain ambiguities and anomalies. To achieve its policy objectives, these lacunae must be plugged.

To begin with, there is no precise definition of a ‘rare disease’ in India, effectively making the policy a non-starter. Therefore, the Government needs to define it at the earliest. Here, the Centre can consider aligning the policy definition of a‘rare disease’ with the ‘orphan drug’ definition provided under the New Drugs and Clinical Trials Rules 2019. The Rules define ‘orphan drug’ as one meant to treat a condition that affects not more than five lakh people in India. Such an aligned definition will be in sync with the ICMR’s existing criteria for a ‘rare disease’ — as one that affects fewer than one in 2,500 individuals.

The constraints

As funding is a major constraint, patients with rare diseases should be provided funds irrespective of the group they come under. A minimum budget of ₹500 crore should be allocated for treating already diagnosed patients, who have been deprived of treatment and are in dire need of support from the Government. Around 350-400 diagnosed patients who are treatable with DCGI-approved therapies can come under the purview of this outlay. As some of these patients have been awaiting treatment for more than two years, they should be urgently considered for the same.

The Centre can consider devising a mechanism for sustained support where appropriate treatment is available without regard to the cost of treatment. Increased allocations must be considered in the case of diseases requiring lifelong treatment. Moreover, there should be provisions for including other disorders or new diseases under the existing groups of rare diseases as per the Technical Committee’s recommendations formed under the policy.

To boost funding, the Centre can make provisions incentivising public and private sector companies in declaring grants for rare diseases under their CSR policy. Also, there should be a complete waiver of taxes (GST and import duty) on drugs for treating rare diseases. Unfortunately, many of these drugs attract more than 20 per cent taxes

Another gap concerns awareness, screening, diagnosis and treatment modalities. It is imperative that State-level data is fed to the national registry, which will help in addressing the disease in a holistic manner. Accordingly, there should be treatment centres in all States, besides Centres of Excellence (CoEs) as proposed in the draft policy.

Further, a multi-sectoral convergent and linked model between various ministries for managing rare diseases is necessary. The respective roles for each ministry and the departments concerned should be predefined along with an implementation framework for combating rare diseases.

Public-private partnerships (PPPs) are vital and can focus on capacity building, providing equipment, reagents, personnel and improving diagnostics, awareness and education on rare diseases.

Finally, research is a critical parameter. A dedicated corpus for research on rare diseases would help improve medical awareness and the diagnostic capabilities of CoEs.

The writer is Founder Director, Patient Safety & Access Initiative of India Foundation, New Delhi

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