Travel Once, Learn Twice and Save
Save when you register for both the Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development and the Advancing the Science of Study Endpoints: Seeking Practical Solutions
*You must register for both events at the same time.  Details in the Registration section listed on the left.

Patient Organizations/Patients are invited to attend the Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development at a reduced registration fee of $200. 

Questions? Contact:

Send Email
1.215.442.6158

Tutorial:  October 6*
Workshop Date:  October 7

*Tutorials require registration and are an additional fee

This is a one-day, intensive workshop on structuring rare disease therapeutic development programs that integrate patient, regulatory, and clinical perspectives to achieve the most meaningful outcomes for patients.  Knowledge will be shared on establishing the foundation of a cohesive regulatory framework for rare disease therapeutic development through scientific planning.  Workshop participants will examine the unique aspects of clinical endpoint and study design for rare disease therapies, and how engagement of patient organizations and regulators can facilitate the development of a program.  A case study on the development of a “mock” therapy will allow participants to explore practical issues in the development of the clinical program and in the integration of the patient and regulatory perspectives into a comprehensive strategy to meet patient needs.