1. Version: 27 May 2007 New Models for Collaboration: Patients as Drivers and Partners in Neurological Research Integrating Patient Input Within the Drug Approval Process Russell Katz, M.D. Director Division of Neurology Products Center for Drug Evaluation and Research

2. 2 Two main areas in which patients can influence drug development and approval Patients can play an important role during the development, and conduct, of clinical trials (IND) Patients can play a role in the decision about whether a particular drug should or should not be approved (NDA)

3. 3 Patient input during drug development Patients can play an important role in the general development of drugs for a particular indication Patients can also play an important role in the development of a specific drug

4. 4 Patient input for a specific indication Numerous venues to interact with the FDA FDASIA (2012) –Public meetings to consider 20 different disease areas over the next 5 years –Federal Register (FR) Notice 9/24/12-invited public comment on potential disease areas- Meeting held 10/25 –Over 4000 comments received-1/3 for neurology

5. 5 Patient input for a specific indication Part 15 Hearing –This is a mechanism for the public to speak directly to a panel of FDA officials on a specific topic –Written comments are also solicited –Meeting on ALS held this week

6. 6 Patient input for a specific indication Meetings with specific patient “communities” –Small meetings with Agency staff –Larger, more public meetings arranged by “consortia” of patient (advocacy) groups –Similar meetings arranged by academic groups

7. 7 Patient input for a specific indication Formal qualification of outcome measures, (e.g., Patient Reported Outcomes [PROs]) These instruments are entirely dependent on solicitation of patient input (and not just PROs) The creation of outcome measures provide an extremely important mechanism for direct patient influence of drug development

8. 8 Patient input for the development of a specific drug FDASIA discusses the Patient Consultant Program –Patients with a specific condition may participate in meetings between FDA and sponsors –We have attempted this in the past-logistical problems have prevented very meaningful participation

9. 9 Patient input for the development of a specific drug Generation of outcome measures is relevant here also In particular, patient specific outcome measures (goals) can be extremely important, but are very rarely used This activity, in general, is, clearly, primarily the purview of the sponsor But we encourage these efforts

10. 10 Patient input in the decision on a specific application FDASIA refers to the Patient Representative Program –Representatives serve on Advisory Committees (in addition to a Consumer Representative); we have had such representatives in the recent past –Equally important (in my view) are comments from patients in the public sessions of AC meetings

11. 11 Other ways patients can contribute to drug development Especially for orphan conditions, advocacy groups can help facilitate the conduct of clinical trials In particular, advocacy groups can create and maintain patient registries Critically, advocacy groups can urge patients/families to enter clinical trials, especially for marketed drugs

12. 12 Summary Patients and advocates can play important roles in the design and facilitation of clinical trials Patients and advocates can play important roles in the decision on a specific application FDA’s actions must be driven by science and conform to regulations, and we recognize that these goals may be more efficiently achieved with more direct patient input along the way