5th Workshop in a Series on Pharmacogenomics: Generating and Weighing Evidence in Drug Development and Regulatory Decision Making

The field of genetics and its applications continue to evolve rapidly with the publication of multiple genome-wide association studies, the availability of new DNA sequencing technologies, and examples of biomarkers that are being used to help define patient response in myriad diseases (e.g., oncology, HIV, autoimmune, cardiovascular). In addition, the increased pressure to demonstrate value of medicines is refocusing efforts and attention on how to effectively and practically define patients who are grouped by biomarkers during and following clinical development.

Between 2002 and 2007 the FDA, in collaboration with industry, has co-sponsored four major workshops on pharmacogenetics and pharmacogenomics (PGx) that have facilitated understanding of issues that surround implementation of PGx studies during clinical development and led to the development and drafting of several documents pertaining to the use of PGx in clinical development.1 This workshop will develop and advance approaches and ideas to improve the value of PGx and other biomarker studies during clinical development and for regulatory decision making and provide networking opportunities with colleagues from academia, regulatory authorities, industry, payors and providors who work on personalized medicines.

Featured Topics

·         When PGx data will be required during clinical development

·         Defining parameters that allow retrospective/prospective analyses to be conducted for regulatory approval of compounds in biomarker defined cohorts

·         Case studies in efficacy, safety, and dosing which have integrated PGx

·         Translation of genomic information to labels that are useful to prescribers and patients

·         Challenges with sample collection for PGx in global development programs and ways to overcome them

·         Critical analyses and recommendations for drugdiagnostic co-development paradigms which are feasible in the competing hurdles to develop new medicines

·         Discussion panel of stakeholders including regulators, industry, third party payers, medical researchers and practitioners

At the conclusion of this workshop, participants should be able to:

·         Discuss how the FDA and other international regulatory authorities view the use of retrospective analyses of PGx data in regulatory decision making;

·         Describe issues surrounding incorporation and use of PGx information in drug labels;

·         Explain barriers to sample acquisition in drug development programs in different geographic regions, and their potential implications; and

·         Outline enabling factors for successful drug-diagnostic co-development (as well as development after drug approval) and examples in which this paradigm shift is most relevant.

·         Physicians

·         Legal community

·         Statisticians

·         Nurses

·         Pharmacologists

·         Clinicians

·         Biologists

·         Healthcare providers

·         Molecular biologists

·         Reimbursement specialists

·         Clinical scientists

·         Human geneticists

·         Regulatory affairs professionals