Anavex Life Sciences Corp (AVXL)

[nidan7500]

Biostockclub...WOW.

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.

Some have recognized the FDA rules 21CFR parts as a compilation of everything that ever went wrong. This says that.

You are right, they are planning to lead a change process which is long overdue and which is vital to the application of Precision Medicine. These words read like a combination of the US Bill of Rights and the Declaration of independence for Medicine and science.