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Drug Repurposing: Considerations for Selection Criteria and Prioritization Morning Sessions
Reagan-Udall Foundation for the FDA
Overview
This video discusses drug repurposing, focusing on the criteria and processes for selecting and prioritizing existing approved drugs for new therapeutic uses. It highlights the importance of leveraging existing safety and manufacturing data to accelerate treatment development, particularly for rare diseases and conditions with unmet medical needs. The discussion emphasizes that repurposing is not a shortcut around rigorous scientific evidence but a strategic approach to expand treatment options. It also covers regulatory pathways, technological advancements like AI, and the collaborative efforts between government agencies, researchers, and patient advocacy groups to overcome barriers and bring promising repurposed drugs to patients.
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Chapters
- Drug repurposing involves finding new uses for already approved drugs, building on existing safety and manufacturing knowledge.
- This initiative aligns with public health priorities, aiming to expand treatment options for patients with unmet medical needs.
- Repurposing requires rigorous scientific evidence and does not bypass standard safety and efficacy requirements.
- The FDA is exploring transparent, scientifically grounded approaches to identify and advance promising drug repurposing candidates.
Understanding the FDA's focus on drug repurposing is crucial for identifying opportunities to accelerate the development of new treatments for diseases that currently lack effective therapies.
The FDA issued a Request for Information (RFI) to gather input on priority disease areas, potential drug candidates, and innovative approaches to drug repurposing.
- The Modernization of Labeling Act provides a statutory pathway for updating labeling of generic drugs when there's a meaningful gap and a public health benefit.
- FDA is building on previous work, including a 2019 meeting and extensive research by organizations like Duke Margolis.
- Initiatives like Project Renewal and the Cure ID platform are actively supporting drug repurposing efforts.
- Success stories include label updates for countermeasures and calcium, demonstrating progress in repurposing.
Existing regulatory pathways and ongoing initiatives demonstrate a commitment to facilitating drug repurposing, offering concrete examples of how this process can be implemented.
The Modernization of Labeling Act allows FDA to update generic drug labels when they no longer reflect current scientific evidence or clinical context, provided there is a public health benefit.
- Artificial intelligence (AI) and machine learning are being used to identify potential drug repurposing candidates by analyzing vast biological knowledge graphs.
- Platforms like 'Matrix' (from Every Cure) and NIH's 'Translator' program help map biological relationships between drugs and diseases.
- Cure ID is a crowdsourcing tool that collects deidentified treatment experiences from healthcare providers and patients to identify potential repurposing signals.
- These tools are hypothesis-generating and aim to identify candidates for further scientific and clinical validation, especially for rare diseases.
Advanced technologies like AI and data-sharing platforms are revolutionizing how potential drug repurposing candidates are discovered, accelerating the initial stages of research.
The Cure ID platform collected deidentified patient data, which helped identify Nitroxylene as a potential treatment for a rare and deadly brain-eating amoeba infection (Balamuthia mandrillaris).
- NIH's National Center for Advancing Translational Sciences (NCATS) bridges the gap between basic research and clinical trials.
- NCATS maintains a physical library of approved small molecule drugs for screening potential repurposing opportunities.
- The 'Translator' program uses AI to mine biomedical data and predict generic medicines for specific diseases.
- Successes include identifying treatments for rare diseases like Niemann-Pick disease and Sanfilippo syndrome, often with limited commercial interest.
NIH's active involvement and investment in translational science, including drug repurposing, highlight the federal commitment to finding solutions for diseases with high unmet needs.
NCATS screened its library of approved drugs and identified a molecule that, after extensive pre-clinical and clinical development, is now under FDA review for a rare disease.
- The FDA's Request for Information (RFI) received over 600 public comments, indicating significant stakeholder interest.
- Key themes from public comments included priority disease areas (e.g., chronic diseases, rare diseases), specific drug candidates, and innovative identification approaches.
- Ketamine was the most frequently recommended drug candidate, suggested for various mental health and pain conditions.
- Long COVID and rare diseases also emerged as significant areas of focus, with numerous candidate recommendations.
- Barriers identified include access and affordability, commercial viability, maintaining evidentiary standards, and regulatory pathway uncertainties.
Analyzing public feedback through the RFI process is critical for the FDA to understand stakeholder priorities, identify common challenges, and shape its drug repurposing framework.
Public comments highlighted ketamine as a frequently suggested drug for repurposing, particularly for depression and pain management, indicating a strong interest in this area.
- Opportunities for innovation include adaptive, pragmatic, and decentralized clinical trial designs.
- Leveraging data, real-world evidence, and patient-generated data can accelerate development.
- Enhanced collaboration infrastructure, including new federal funding and incentive streams, is needed.
- Stakeholders requested regulatory and policy clarifications, such as guidance and streamlined pathways.
- FDA plans to publish its candidate selection and prioritization framework and continue engaging with stakeholders.
Identifying and acting on opportunities for innovation and collaboration is essential for overcoming existing barriers and successfully advancing drug repurposing efforts.
The FDA plans to develop and publish a candidate selection and prioritization framework based on the RFI feedback and today's discussions.
Key takeaways
- Drug repurposing leverages existing drug knowledge to find new uses, potentially accelerating access to treatments for unmet medical needs.
- Rigorous scientific evidence remains paramount; repurposing is not a shortcut around safety and efficacy standards.
- Technological advancements, particularly AI and data-sharing platforms, are crucial for identifying and evaluating potential repurposing candidates.
- Collaboration between government agencies (FDA, NIH), researchers, industry, and patient groups is vital for success.
- Addressing barriers such as commercial viability, access, and regulatory clarity is essential for advancing repurposed drugs.
- The FDA is actively seeking public input to inform its drug repurposing framework, emphasizing transparency and scientific grounding.
- Existing regulatory pathways, like the Modernization of Labeling Act, provide mechanisms to update drug labels and facilitate new uses.
Key terms
Drug RepurposingUnmet Medical NeedSmall Molecule DrugsCenter for Drug Evaluation and Research (CDER)Modernization of Labeling ActRequest for Information (RFI)Artificial Intelligence (AI)Cure IDTranslator ProgramTranslational ScienceEvidence StandardsCommercial Viability
Test your understanding
- What is the primary advantage of drug repurposing compared to developing a new drug from scratch?
- Why is it important for drug repurposing to still adhere to rigorous scientific evidence standards?
- How are technologies like AI and data platforms contributing to the identification of drug repurposing candidates?
- What are some of the key barriers that hinder the progress of drug repurposing efforts, according to the RFI feedback?
- What role do organizations like the Reagan Udall Foundation and NIH play in advancing drug repurposing initiatives?