• Examining the Impact of Real-World Evidence on Medical Product Development: A Workshop Series | Workshop 1: Incentives Date: September 19, 2017 - September 20, 2017 (8:30 AM Eastern)

    An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine (NASEM) will plan and conduct a three-part workshop series to be held over the course of a two-year period. As part of FDA's continued focus on building a national governance system for evidence generation, the proposed focus of these workshops will be on the generation and utilization of real-world evidence to evaluate efficacy, effectiveness, tolerability, and safety for both review of new indications and postapproval studies.
  • Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development – A Workshop Date: March 8, 2017 (8:00 AM Eastern)

    This one-day public workshop will examine and discuss successes, challenges, and best practices for effectively utilizing genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. Topics covered could include strategies for including genetically focused populations in clinical trial design, logistical challenges in conducting genetics-based clinical trials, and mechanisms for engaging with and educating potential trial participants.
  • The Drug Development Paradigm in Oncology: A Workshop Date: December 12, 2016 - December 13, 2016 (8:00 AM Eastern)

    This workshop will examine the challenges of improving the efficiency of the drug development and regulatory review process, while ensuring the safety of new therapies.
  • Real-World Evidence Generation and Evaluation of Therapeutics—A Workshop Date: October 19, 2016 (8:00 AM Eastern)

    The traditional process for evaluating new therapeutics does not produce the evidence that patients, clinicians, and payers need for real-world decisions.
  • Deriving Drug Discovery Value from Large-Scale Genetic Bioresources: A Workshop Date: March 22, 2016 (8:30 AM Eastern)

    On March 22, 2016, the Roundtable on Translating Genomic-Based Research for Health and the Forum on Drug Discovery, Development, and Translation hosted a one day public workshop to examine and discuss how large-scale genetic data could be used to improve the likelihood of bringing effective and targeted therapies to patients. The goal of the workshop was to address how progress could be made in discovering and validating promising targets and medicines for those targets by using the data collected from large-scale genetic studies. Discussions were held with a broad array of stakeholders which included representatives from pharmaceutical and biotech companies, IT and data science companies, research institutes, investors, providers, patients, payers, and regulators.
  • Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: An IOM Workshop Date: October 20, 2015 - October 21, 2015 (8:00 AM Eastern)

    This workshop will provide a venue to review progress in building the foundations of regulatory science and to explore a forward-looking agenda for bolstering the field. Participants will examine the current state and scope of the discipline, highlight opportunities to address barriers to success, and explore ways to foster collaboration.
  • Enabling Rapid Response and Sustained Capability with Medical Countermeasures to Mitigate Risk of Emerging Infectious Diseases: An Institute of Medicine Workshop Date: March 26, 2015 - March 27, 2015 (8:30 AM Eastern)

    This workshop, hosted by the Forum on Medical and Public Health Preparedness for Catastrophic Events, the Forum on Drug Discovery, Development, and Translation, and the Forum on Microbial Threats will bring together public and private stakeholders to discuss how to achieve rapid and nimble medical countermeasure development for new and emerging threats. Discussions will include real-world case studies to elucidate how past events were handled from a policy, budget, and operational standpoint, and contribute to a better aggregate picture of how future “worst case scenarios” might unfold. This workshop will examine principles that either enable or challenge strong public/private partnerships and consider the needs of the private sector to successfully ramp up research, development, and production in a rapid and nimble manner once a threat warrants the need.
  • Financial Incentives to Support Unmet Medical Needs for Nervous System Disorders: A Workshop Date: January 20, 2015 - January 21, 2015 (8:30 AM Eastern)

    This workshop will explore opportunities to foster private sector innovation by supporting new investments directed toward the development of novel therapeutics to meet unmet needs for nervous system disorders.
  • Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products Date: May 12, 2014 (9:00 AM Eastern)

    This two-part public workshop series will address the opportunity to advance the development of more systematic and structured approaches to characterize and communicate (a) the sources of uncertainty in the assessment of benefits and risks; and (b) their implications for pharmaceutical regulatory decisions. Specifically, the workshop series will explore potential analytical and communication approaches and identify key considerations on their development, evaluation, and incorporation into the assessment of benefits and risks in pharmaceuticals.
  • Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products Date: February 12, 2014 - February 13, 2014 (9:00 AM Eastern)

    Discuss the challenges in applying more systematic approaches to characterizing and communicating uncertainty in the assessment of a drug’s benefits and risks.