Mitra Tavakkoli

Company: Formerly of 4D Molecular Therapeutics

Job title: Vice President, Clinical Development

Bio:

Executive physician scientist with extensive rare disease gene therapy drug development experience

Seminars:

Panel Discussion: Establishing Multistakeholder-Relevant Clinical Endpoints 11:30 am

How can we ensure clinical trial endpoints are meaningful to patients, agreeable to regulators, and measurable to payers Considerations for internal team structuring to ensure endpoints are relevant to all stakeholders Understanding EMA and FDA differences in deeming an outcome ‘clinically meaningful’Read more

day: Clinical - AM

Panel Discussion: Reaching, Enrolling, and Retaining Participants in Rare Disease Gene Therapy Trials 2:00 pm

Multi-stakeholder discussion of friction points in the development lifecycle and how to overcome them: what have we tried, what has been successful and under what circumstances Dissection of real-world examples over time and across different patient communities Analysis of recent trends and impacts of continuing advancesRead more

day: Clinical - PM

Rare Disease Trials: To Placebo or Not to Placebo? 11:00 am

Key considerations when considering incorporating a placebo control Outlining the implications to the sponsor, regulators, and patients When is a placebo considered unethical or unfeasible? How are global regulators’ attitudes on placebo control arms evolving?Read more

day: Clinical - AM