India should chart own path in treatment of rare diseases: ICMR chief
Speaking at a two-day national conference on rare diseases organized by the Union Health Ministry here, the director general of the Indian Council of Medical Research (ICMR) said India should chart its own path in diagnosis, treatment and prevention rather than relying solely on Western frameworks.
He noted that while developed countries have more resources, India can leverage its strengths through effective use of digital technologies, including population-based approaches, preventive strategies and emerging tools such as social media and artificial intelligence, to expand social media and improve early detection.
Noting the progress over the past three decades, Bahl said: “In the 1990s, identifying a patient with a suspected rare disease often led to a feeling of helplessness because diagnosis was extremely difficult and treatment options were almost non-existent.”
“While even financial support of Rs 50 lakh per patient may seem inadequate today, given the high cost of treatments, this represents a remarkable progress through which the country is now able to meaningfully support children affected by rare diseases,” he said.
He emphasized that this evolution reflects a broader shift in healthcare priorities; Here, attention is given not just to common diseases but also to those affected by rare, often genetic, conditions, describing the government’s rare disease program as “a source of hope for thousands of children”. Dr. Emphasizing the need to optimize existing resources, Bahl said that careful planning of diagnostic and treatment pathways, with a defined financial support framework, is vital.
He emphasized the value of family-based approaches, including parental genetic analysis and prenatal diagnosis, as cost-effective strategies for prevention and early intervention.
Highlighting ongoing efforts, he said ICMR is actively working to expand the range of tools available for the management of rare diseases, including promoting indigenization of therapies and promoting indigenous manufacturing of affordable alternatives to high-cost drugs in collaboration with industry partners, supported by clinical evaluation through Centers of Excellence.
He also spoke about the use of repurposed drug which are “medications that may not fully cure but can significantly improve outcomes and quality of life”, adding that six such drugs have been identified and clinical efforts are being initiated for their use in rare diseases.
Regarding advances in advanced technologies, Bahl stated that gene therapy efforts are being continued in collaboration with institutions such as the Department of Biotechnology (DBT) and CSIR. Referring to developments such as CAR-T cell therapy, he stated that “India is making steady progress in this field and is closer than ever to realizing such advanced treatments.”
He expressed confidence that continued collaboration, innovation and commitment from all stakeholders will significantly improve the rare disease care landscape in India and deliver tangible benefits to patients and their families.
Union Health Secretary Punya Salila Srivastava said that the main objective of the conference is to understand the challenges faced by stakeholders, promote innovations and generate new ideas to strengthen the management of rare diseases in the country.
He noted that the need to address rare diseases was first highlighted in the 2017 National Health Policy and subsequently institutionalized through the National Rare Diseases Policy (2021), which provides a comprehensive framework.
Highlighting the implementation, he added that the policy has been operationalized through Centers of Excellence (CoEs) and their number has increased from eight to 15, including two in the Northeast.
“The financial assistance under the policy has been increased gradually to Rs 50 lakh, providing better access to treatment for patients suffering from identified rare diseases,” he said.
He also emphasized the importance of awareness and early diagnosis, stating that “rare diseases require joint and sustained efforts from all stakeholders” and expressed confidence that the discussions at the conference will help strengthen the national response.
The two-day conference, which begins on Tuesday, aims to bring together key stakeholders to reflect on challenges and strengthen the national response to rare diseases.
