Taking a leaf out of Amul’s textbook to help rare-disease patients

The proposal addresses a critical gap in the National Policy for Rare Diseases, 2021, which emphasizes the need for an epidemiological database but lacks a robust mechanism for longitudinal data collection. The Indian Council of Medical Research (ICMR) currently maintains a rare disease registry, but its scope is limited compared to the scale of India's population. By establishing a PDC, the government can shift from fragmented data silos to a centralized, patient-centric ecosystem. This aligns with the objectives of the Ayushman Bharat Digital Health Mission (ABDM), which seeks to create a seamless online platform through the provision of a wide-range of data, information and infrastructure services, duly leveraging open, interoperable, standards-based digital systems. The proposed model suggests utilizing the ABDM framework to mandate secure digital health lockers, empowering patients with ownership of their medical histories. A key governance challenge will be ensuring data privacy and adhering to ethical standards, requiring robust oversight mechanisms and informed consent protocols, especially when sharing data with research entities.

The integration of Artificial Intelligence (AI) is central to maximizing the utility of the PDC. The article highlights the use of generative AI to synthesize medically relevant patterns from vast, unstructured datasets (like clinical notes and genetic reports). This technology can facilitate earlier diagnosis and more precise treatment pathways for rare diseases. Furthermore, the updated New Drugs and Clinical Trials Rules, 2019 in India encourage advanced computational modeling and non-animal testing. The PDC can support this by enabling the creation of virtual synthetic control groups. In traditional clinical trials, a control group is necessary for comparison; however, the scarcity of patients with rare diseases makes this difficult. The PDC’s repository of natural history studies—longitudinal data tracking disease progression without intervention—can serve as an 'external' control, making small-batch orphan drug evaluation clinically and financially viable for Indian biotech companies.

The cooperative model, inspired by Amul, offers a unique economic framework for healthcare data. In this model, the PDC acts on behalf of patients, holding their data and returning a portion of any revenue generated from research collaborations back to the patient community. This incentivizes participation and ensures equitable benefit-sharing, addressing a common ethical concern in medical research where data subjects are often uncompensated. The aggregation of granular data from India's genetically diverse, endogamous populations (groups that marry within a specific community) creates a highly valuable resource for global pharmaceutical companies seeking ethnically specific target-validation cohorts. This can attract foreign direct investment (FDI) in domestic research and development. However, realizing this potential requires initial funding from the government or philanthropic organizations to build the AI infrastructure and establish the governance framework, highlighting the need for strategic public-private partnerships in the health tech sector.