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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 that

  • therapeutic and diagnostic products to be used within a country are manufactured within the country

  • 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 experience 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?


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 a 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: NGS datasets from which patient-selection markers can be identified, confirmed and transformed into companion diagnostic products.

Regulatory pathfinding is needed when prescriptive regulatory paths can not assess accurately the incremental benefit of precision medicine products.


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 a major 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.

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