FDA calls for collaboration in new medicines

The US Food and Drug Administration has issued a major report identifying both the problems and potential solutions to the task of ensuring that the unprecedented breakthroughs in medical science are demonstrated to be safe and effective for patients as quickly and inexpensively as possible.

Entitled 'Innovation or Stagnation? - Challenge and Opportunity on the Critical Path to New Medical Products,' the report examines the 'Critical Path' of medical product development - the crucial steps that determine whether and how quickly a medical discovery becomes a reliable medical treatment for patients.

It examines the development path for all types of medical products - drugs, biologics and medical devices - to identify what problems exist and what steps need to be taken to bring the critical path into the 21st Century. The report particularly focuses on the unique opportunities for FDA to collaborate with academic researchers, product developers, patient groups, and other stakeholders to make the critical path much faster, predictable, and less costly.

'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,' said its commissioner Dr Mark McClellan. 'With so much promising technology in development in the clinical labs, ranging from engineered tissues to new kinds of biologicals and genomics-based treatments, we need to turn the process of bringing these technologies to patients from a costly and time-consuming art form to a well-understood science. FDA intends to launch a new effort with our public and private partners in improving the public health to turn the critical path of product development into a fast, certain, and more affordable process.'

The report notes that despite notable advances in innovative fields of biomedical research as genomics, proteomics and nanotechnology, there has been a downward trend in recent years in the number of innovative medical product applications to FDA and its counterpart agencies throughout the world. While the number of new product applications and approvals was modestly higher in 2003, the fact remains that most of these new scientific fields are not yet having a fundamental impact on patient care.

The report applauds the important advances triggered by recent investments in basic and translational research, and concludes that a third type of research, targeting the process of creating safe and effective products from new scientific discoveries, is urgently needed to complete the path from discovery to patient. In FDA's view, the applied sciences needed for medical product development have not kept pace with the tremendous advances in the basic sciences.

To meet this challenge, FDA calls for a new focus on modernising the tools that applied biomedical researchers and product developers use to assess the safety and effectiveness of potential new products, and the manufacturing tools necessary for high-quality mass production of cutting-edge therapies. The report describes the need for the FDA, together with academia, patient groups, industry, and other government agencies, to embark on an aggressive, collaborative research effort to create a new generation of performance standards and predictive tools that will provide better answers about the safety and effectiveness of investigational products, faster and with more certainty.

Key to this effort will be the collaborative development of a Critical Path Opportunities List, which will identify those areas of product development that could most benefit from innovative approaches and emerging technological advances, to fundamentally change and modernise the critical path for medical product testing and manufacture in the 21st Century.

FDA also intends to make internal changes and implement new collaborations to realise these priority opportunities. This action promises not only to bring medical breakthroughs to patients more quickly, but to do so in ways that ensure greater understanding about how to maximise patient benefits and minimise their risks. Moreover, the efficiencies gained through these innovations could bring significant economies that could provide both more affordable medical products and a much greater payoff from greater predictability and speed for investment in medical research and development.

The report notes that FDA is suited to take a major role in this effort because of its experience overseeing medical product development, assessment and manufacturing/marketing; its vast clinical and animal databases; and its close interactions with all the major players in the critical path process. This initiative is not a fundamental departure for FDA, but rather builds on the agency's proven 'best practices' for expediting the availability of promising medical technologies. Examples of such efforts include:

Developing new animal models for speeding the assessment of safety and effectiveness of next generation medicines against anthrax, smallpox and other bioterrorist threats.

Developing a four company-sponsored 40,000 patient study of the comparative accuracy of digital and conventional mammography - a study no one company could develop or mount, but which is of vital importance to millions of women.

Using the unique incentives of the Orphan Drug Act and FDA's knowledge of the drug development process to collaborate with industry, government and patient groups to produce more than 250 approvals of Orphan Drugs over the last 21 years. These products, in the aggregate, treat more than 12m patients in the US.

'The FDA is fully committed to moving this process forward. Bringing the stakeholders together is vital for identifying ways to overcome the obstacles to new product development in the interest of the public health,' said FDA deputy commissioner Dr Lester Crawford.