Here is who is representing Anavex at the conference.
Harald Hampel, MD, PhD1, Mohammad Afshar, MD, PhD2, Fre´de´ric Parmentier, PhD2, Coralie Williams, MSc2, Adrien Etcheto, MSc2, Federico Goodsaid, PhD3, Christopher U Missling, PhD4; 1Department of Neurology, Sorbonne University, Paris, France, 2Ariana Pharma, Paris, France, 3Regulatory Pathfinders LLC, San Francisco, CA, 4Anavex Life Sciences Corp., New York, NY
Please note: 1. Hampel - Dept of Neurology, Sorbonne 2. 4 folks from Ariana including Pres. 3. Federico Goodsaid who is with Regulatory Pathfinders, San Francisco 4. Dr. Missling
I looked up Regulatory Pathfinders, as this is a new one on me. (The rest we know.)
Think you will find this company’s line of work very interesting, just as I did! So, new specialty on board for this.
Anavex has blockers, going in for the 6! This looks like a drive which has “GOAL” written on it to me. (Not practice, not a drill. Looking for scoreboard points here.) Bio
Strategic Regulatory Intelligence tools over the past decade include the Voluntary Exploratory Data Submission (VXDS) and Critical Path for Innovation (CPI) meetings at the FDA, as well as the Innovation Task Force (ITF) meetings at the EMA and the Consultation System (CS) at the PMDA. These and other Strategic Regulatory Intelligence initiatives have encouraged genuine partnerships between different stakeholders in the development of novel therapies. Specific examples of Strategic Regulatory Intelligence include: • sharing of emerging statistical tools for the analysis of clinical data in VXDS meetings • alignment strategy with the FDA and EMA on the molecular phenotype definition of patient populations to be used in precision medicine clinical product development • regulatory consensus reached through FDA pre-IDE meetings on multi-tiered genotyping labeling language excluding need for companion diagnostic • organization of consortia to address bioinformatics challenges in the analysis of Next Generation Sequencing data These and other strategic regulatory intelligence examples have had an impact on the goals and practice of regulatory affairs in medical product companies, as well as on the perspective of industry organizations on policy initiatives and government interactions. PLANNING
Planning for regulatory activities in precision medicine product developmenmt reflects a regulatory landscape for medical products throughout the world that is very diverse. Government regulation of medical products in different countries is associated with different goals linked to specific priorities in each country. These priorities may range from • an emphasis on making sure therapeutic products to be used within a country are manufactured within the country • making sure marketing authorizations are aligned with global health system support goals. This global regulatory landscape may also lead to inherently adversarial interactions between different patient, industry and government stakeholders. The regulatory framework for development of medical products in the United States is a result of a cumulative wisdom stemming from over a century of responses to threats to public health. The U.S. Food and Drug Administration (FDA) was born and grew from urgent and difficult federal government decisions in response to clear and imminent risks to food safety, drug safety and efficacy, and accuracy and clinical safety and efficacy of human diagnostic products. Throughout the evolution of this regulatory framework, the FDA has shown a consistent record in the support of public health, continuously linked to scientific and clinical knowledge available to support regulatory decision-making. Scientific and clinical knowledge needed to support regulatory decision-making originates from multiple academic, Government, patient organization and medical product industry sources. How do novel precision medicine products fit within this regulartory risk/benefit assessment framework? Today, there are therapeutic, diagnostic and companion diagnostic precision medicine products. Do regulatory paths associated with these product categories adequately cover all novel precision medicine products? STRATEGY
The value of medical product development knowledge is maximized when it can be shared between its source and regulatory agencies and integrated into coherent information. Strategic Regulatory Intelligence is a useful tool to drive regulatory discussions about novel precision medicine products. In addition to this tool, however, the global strategy for regulatory approval of precision medicine products may require the identification of regulatory paths which do not have a prescriptive designation associated with current regulations. Regulatory pathfinding identifies regulatory paths which are not prescriptively defined : • CASE 1: a precision medicine therapy targeting a small population of patients with a specific mutation, which may also be effective for patients with other mutations sharing a molecular phenotype defining a class of mutations; • CASE 2: a precision medicine therapy for which clinical surrogate endpoints accepted by regulatory agencies can only measure efficacy in a subset of patients, and for which more accurate surrogate biomarker is needed to assess efficacy in the whole patient population; • CASE 3: a precision medicine diagnostic with clinical utility initially demonstrated in conjunction with pre-existing clinical biomarkers and endpoints evolving into a stand-alone diagnostic with a threshold value required for a clinical decision-making; • CASE 4: a precision medicine risk-assessment marker for which clinical utility is defined as incremental accuracy in clinical decision-making. Regulatory pathfinding is needed when prescriptive regulatory paths can not assess accurately the incremental benefit of precision medicine products.
Federico Goodsaid (From the site. About)
Federico Goodsaid, PhD Principal
Bio
As Vice President for Strategic Regulatory Intelligence at Vertex Pharmaceuticals, he focused on early and effective collaborative interactions with regulatory agencies on exploratory, enrichment and surrogate biomarkers. At Vertex, he successfully set up a pre-submission seamless communication of critical biomarker data leading to a timely consensus with regulatory agencies on clinical trial design and on labeling language.
Before his work at Vertex, he was Associate Director for Operations in Genomics and Biomarker Qualification Coordinator at the Office of Clinical Pharmacology/Office of Translational Sciences/ Center for Drug Evaluation and Research/ U.S. FDA, where he chaired Voluntary Exploratory Data Submission (VXDS) meetings and developed the Pilot Biomarker Qualification Program (BQP). He coordinated development of the C-Path Institute Predictive Safety Testing Consortium and other industry/government collaborations to encourage submission of biomarker qualification requests to the FDA BQP.
His B.A. is in Biochemistry and Biophysics from the University of California at Berkeley and his Ph.D. from Yale University in Molecular Biophysics and Biochemistry.
Areas of Expertise
* development of optimized and novel regulatory strategies and policies for precision medicine products, leading to efficient regulatory pathways for product approval by regulatory agencies throughout the world * breakthrough therapies * rare diseases * surrogate endpoints * companion diagnostics
Outlook
Medical product development is a biomarker-driven task. Its success depends on the effective use of biomarkers and the effective communication with regulatory agencies of the scientific and clinical information supporting the use of these biomarkers. Strategic Regulatory Intelligence is a valuable tool for a successful communication with regulatory agencies of the science behind biomarkers. In addition to this effective communication, however, the path for regulatory approval of novel precision medicine products may not be currently prescribed as it is for other products. Regulatory Pathfinding may be needed for an accurate path to regulatory approval for precision medicine products.
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