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Abstract Structure

ACTRaD 2025

National Conference on

Advancing Clinical Trials in Rare Diseases

Date: 25th and 26th of July, 2025​

Venue: Indian National Science Academy (INSA), New Delhi

Organizers:

Tata institute for Genetics and Society, Bengaluru | World Without GNE Myopathy (WWGM), New Delhi |

AIIMS, Guwahati | YKG Academy, New Delhi | Organization for Rare Diseases India, Bengaluru

Knowledge Partner: Indian Council of Medical Research (ICMR), New Delhi

India faces unique challenges in the diagnosis and treatment of rare diseases, including limited access to therapies, high treatment costs, and lack of clinical data. ACTRaD aims to bring together researchers, clinicians, regulators, industry leaders, and patient groups to address these gaps through collaboration and innovation.

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This two-day national conference will focus on creating actionable guidance for advancing clinical trials in rare diseases, strengthening indigenous capabilities, and streamlining regulatory processes to accelerate the availability of affordable therapies.

Topics of Discussion: 

  1. Challenges, opportunities and designing early phase clinical trials for rare diseases

  2. Navigating ethical frontiers: Addressing challenges and ensuring equity in rare disease clinical trials

  3. Sharing early experiences of clinical trials

  4. Opportunities for developing drugs for rare diseases

  5. Innovation in clinical trials designs in rare diseases Exploring the role of AI-ML in data analysis, RWE research, decentralized trials in rare diseases

  6. Transforming rare disease therapeutics: Harnessing genomics

  7. Role of disease progression models and early indicators, Biomarkers for success of clinical trial design

Conference Flyer:

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