FDA issues list of critical path projects.

Earlier this month, the FDA released the Critical Path Opportunities List, a list of 76 initial projects designed to accelerate drug development. Under the Critical Path Initiative, introduced in 2004 (see IBO 4/15/04), the FDA aims to identify and address problems in transforming scientific discoveries into new drugs. The Initiative aims to facilitate, coordinate and promote the creation of new tools, frameworks and collaborations to shorten drug development timelines and ensure safer products.

The opportunities created by such an undertaking are enormous and can be expected to influence drug development and testing in the years to come as the FDA's guidance and testing is necessary for drug approval. For instrument companies, the Initiative presents further opportunities for innovation, collaboration and standardization.

The Critical Path Opportunities List, the result of consultations with stakeholders such as drug companies and FDA scientists, addresses six broad areas: development of biomarkers; clinical trial designs, bioinformatics; manufacturing; public health needs; and pediatrics. The two areas of greatest concern cited by stakeholders were biomarker development and streamlining of clinical trials.

Of interest to instrument companies will be the drug development techniques advocated by the Critical Path Initiative. Thirty-three of the 76 opportunities on the List address the development of better evaluation tools, specifically, tools that will predict a drug's potential performance earlier and more reliably. The Opportunities List highlights biomarkers and animal models as two such tools. In particular, it states the need for biomarker qualification and standards, including standards for microarrays, RNA and DNA extraction methodologies, and RNA conversion and labeling. The List also addresses the need for qualified disease and disorder-specific biomarkers and the development of biomarkers for safety, such as genomic biomarkers to predict toxicology.

Issues addressed by the List regarding imaging techniques include the development of standards and the use of imaging biomarkers. The List also targets the improvement of predictions of human responses from animal models, such as better predictive disease models for biological product toxicity.

Also on the Opportunities List are 13 projects targeting manufacturing, scale-up and quality management. Specifically, the List cites the need for tools to better characterize cells, detect contamination of biological products and assess the effects of manufacturing changes on products. The List also calls for new methods for measuring physical characteristics of biological properties and cites NMR, x-ray crystallography and mass spectrometry as techniques for linking these characteristics to clinical outcomes. It also specifies the need for new testing instruments for novel dosage forms and drug-device combination products as well as the need for standards for spectroscopic instruments.

Over the coming months, the FDA plans to evaluate its ability to support the Opportunities List and announce which projects it plans to undertake this year. And, as the FDA emphasizes, private and public entities are already working on such projects on their own.

On the same day as the release of the Opportunities List, the FDA provided an example of Critical Path Initiative in action when it announced the formation of the Predictive Safety Testing Consortium. The Consortium will coordinate the sharing of lab methods for predictive safety tests, allowing member companies to test each other's methods, ultimately resulting in the recommendation of methods to the FDA for use in the development of guidelines. The Consortium members are Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson Pharmaceutical Research and Development, Merck, Novartis, Pfizer, Roche, Schering Plough Research Institute and SRI International.