In response to a US Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine (NASEM) will conduct a study in the United States and the European Union on the processes used to evaluate the safety and efficacy of drugs for rare diseases or conditions. NASEM has invited Xabier Garcia de Albeniz Martinez, MD, PhD, to present at an upcoming meeting for the study.
During the Novel Methodologies session, Dr. Garcia de Albeniz Martinez, Director of Epidemiology at RTI Health Solutions, will share his expertise in causal inference analysis.
Analysis Methods – Causal Inference
7 February 2024
9:35-10:30 am ET
This is the third meeting of the committee; the meeting will be conducted in a virtual environment. There will be an opportunity for interested stakeholders to share public comments with the committee on 7 February 2024 at 12:00 pm.
Public comments will provide the committee with additional insight into key issues related to the study’s statement of task. These include, but are not limited to:
- The use of regulatory flexibilities and supplementary data (e.g., natural history studies and patient registries) when evaluating the safety and efficacy of drugs for rare diseases and conditions
- FDA and EMA engagement of people with rare diseases and conditions to elicit their lived experience to help the FDA and EMA develop guidance, policies, and programs
Information here was sourced from the NASEM website. To learn more about this meeting, click the link below.
Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases in the United States and the European Union Meeting #3 (Virtual): 6-7 February 2024
