FDA to seek answer to pipeline blockage

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Related tags: Food and drug administration, Better

The US Food and Drug Administration is to develop a blueprint for
speeding up the approval of new medical products to counteract the
slowdown in R&D productivity in the pharmaceutical industry,
writes Phil Taylor.

Among the areas identified as being fundamental to improving the 'Critical Path' to improvement are a number aimed at modernising the safety and efficacy assessments of new drugs, as well as manufacturing tools necessary for the mass production of therapies.

Announcing the White Paper - entitled Innovation or Stagnation?​ - FDA Commissioner Mark McClellan said: "Today, as never before, we face a tremendous potential for new medicines to prevent and cure diseases, but fewer new products are actually reaching the FDA."

Welcoming the advances triggered by recent investments in basic and translational research, the agency says that a third type of research, targetting the process of creating safe and effective products from new scientific discoveries, is urgently needed to complete the path from discovery to patient. The applied sciences needed for product development have not kept pace with the tremendous advances in basic sciences, it adds.

The FDA calls for a new focus on modernising the tools used by applied biomedical researchers and product developers to assess potential new products. Along with academia, patient groups, industry and other agencies, it needs to embark on "an aggressive, collaborative research effort to create a new generation of performance standards and predictive tools that provide better answers about investigational products' safety and effectiveness, faster and with more certainty."

Among the issues that are highlighted by the FDA include the expanded use of proteomics and toxicogenomics data in safety assessment, and also working out how to draw on the vast repository of in vitro​ data in the agency's files.

On the efficacy side, the document refers to developing new biomarkers that can serve as surrogate endpoints in clinical trials, as well as making more use of new diagnostic technologies such as molecular imaging tools and improving the design and conduct of clinical trials.

On the manufacturing side, the report notes that many product failures during development are ultimately attributable to problems relating to the transition from laboratory prototype to industrial product.

As a result it is crucial that technical standards (e.g. assays, procedures, or reference standards) and improved methods for the design, characterisation, and mass manufacture of products are available to improve predictability in this area.

"The pharmaceutical industry generally has been hesitant to introduce state-of-the-art science and technology into its manufacturing processes, in part due to concern about regulatory impact,"​notes the report. This led to high in-process inventories, low factory utilisation rates, significant product wastage and compliance problems, driving up costs and decreasing productivity, it adds.

Among the recommendations are an increasing emphasis on process analytical technologies - automated sensors that monitor and control processes - and other technologies that can improve efficiency.

The agency also said that it intends to look critically at areas where FDA regulation may have slowed adoption of technological improvements, in a process which led to internal changes and new collaborations with industry, academia etc.

"Not enough applied scientific work has been done in creating new tools to get fundamentally better answers about how the safety and effectiveness of new products can be demonstrated, in faster time frames, with more certainty, and at lower costs,"​ the report suggests.

The next steps in this initiative include a series of workshops and meetings, to start development of the National Critical Path Opportunities list and to identify the key priorities, said the agency.

R&D costs for new drugs have soared in recent years. In 2000, it cost $802 million (€654m) to make a new drug compared with $318 million in 1987, according to the US trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

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