Vision for the Next Century

• Critical Path Initiative

  • Transform medical product development

  • Keep pace with expected medical advances

Decorative Image: Medical treatments and patients

But the recent slowdown--instead of the expected acceleration--of new medical treatments actually reaching patients concerns the FDA. Products fail before they reach the market because they could not be proved safe or effective, or they could not be manufactured commercially at a consistently high quality.

Despite recent innovations, many serious and life-threatening diseases still lack effective treatments. In the agency's view, the scientific tools needed to develop medical products have not kept pace with the rapid advances in product discovery. As a result, fewer of the sound ideas spawned in medical laboratories are becoming safe and effective treatments.

In the interest of public health, the FDA says that action is needed to modernize the product development process.

In March 2004, the agency issued a major report that identifies both the problems and the potential solutions for bringing more breakthroughs in medical science to patients as quickly and efficiently as possible. The report, "Innovation or Stagnation? Challenge and Opportunity on the Critical Path to New Medical Products," examines the crucial steps that determine whether and how quickly a discovery leads to a reliable treatment for patients.

The report, which looks at the development processes for drugs, biologics, and medical devices, calls for a joint effort of industry, academic researchers, product developers, patient groups, and the FDA to identify key problems and to develop solutions.

To meet the challenge, the FDA is calling for a new focus on modernizing the tools that researchers and product developers use to assess the safety and effectiveness of potential new products and to mass-produce high-quality therapies. New scientific and technical tools--including assays (tests), standards, computer modeling techniques, biomarkers, and clinical evaluation techniques--will improve predictability and efficiency of products along the development path, more likely resulting in safe products that benefit patients.

For example, the FDA rapidly developed standards and calibration tools that enabled product developers to design and produce test kits to screen donated blood for the presence of West Nile virus. This work involved extensive collaboration with public health laboratories, industry, and U.S. blood banks, as well as using applied research. During 2003, roughly 8.6 million blood donations were tested. Of these, more than 1,000 donations confirmed positive for West Nile virus were identified and removed from the blood supply.

The FDA also developed and implemented a more flexible and innovative approach to the clinical trials needed to evaluate medical screening devices. This new trial design allows small companies, which often cannot afford the large trials needed to evaluate screening devices, to use common protocols so that their data can be pooled for analysis. The design currently allows manufacturers to test the effectiveness of digital mammography for screening use.

Such success stories can only be accomplished through a concerted and joint effort by industry, academia, patient groups, and the FDA. Key to this effort will be the development of a "Critical Path Opportunities List" that will identify and prioritize the most pressing development problems and the areas that provide the greatest opportunities for rapid improvement and public health benefits.

To create this list, the FDA is consulting and soliciting suggestions from all interested parties to identify and address specific defined critical path opportunities to make product development more efficient and predictable. The agency will publicize the list and encourage collaborations to address the problems and create new product development tools.

The FDA is uniquely suited to take a major role in these efforts because of its experience overseeing medical product development, its vast clinical and animal databases, and its close interactions with all the major players in the critical path process. The agency sees the product development problems industry-wide.

Building on the agency's proven "best practices" for expediting the availability of promising medical technologies, there is an urgent need, for example, to develop tools to accurately assess the risk that a new drug will cause heart rhythm abnormalities. Ongoing international efforts include developing, testing, and validating nonclinical tools such as computer models that may be useful in predicting human risk. Examples of tools that the FDA says are urgently needed include better predictors of human immune responses to foreign substances, methods to further enhance the safety of transplanted human tissues, and new techniques for assessing drug-induced liver toxicity.

The FDA believes that patients have an important role to play in this effort.

"The FDA's critical path initiative now encourages patients and their advocates to join us as we roll up our sleeves to identify the difficult problems in drug development," says Theresa Toigo, the FDA's assistant commissioner for special health issues. This, she says, "may uncover a promising treatment or technology that otherwise might not be developed." The critical path to the development of effective therapies for HIV and AIDS, Toigo adds, "was cleared of overgrowth and underbrush by smart, articulate AIDS activists in the late 1980s." Much work, however, still needs to be done on clinical trial design and patient response measures to ensure that new therapies accurately reflect patient needs and values.

http://www.fda.gov/fdac/features/2004/504_tools.html