Development of Medical Products, Drugs : Part 2

by Food and Drug Administration (FDA)

(Page 2 of 2)

The 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.

In addition, the FDA intends to refocus its own activities and take on new partnerships, as needed, to fulfill these priority opportunities. These actions promise not only to bring medical breakthroughs to patients more quickly, but also to do so in ways that ensure greater understanding of how to maximize patient benefits with a minimum of risk.

The Opportunities

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.

Binita Ashar, M.D., acting clinical deputy director for the FDA's Center for Devices and Radiological Health, says that, while medical devices don't seem to be experiencing the product development delay to the degree that drugs and other products have, the concerns about improving product development tools still apply.

"There doesn't necessarily have to be a problem to support the innovation process that critical path promotes," she says. Ashar sees the critical path initiative working together with the goal of providing the "least burdensome" path to market — a provision of FDAMA (the Food and Drug Administration Modernization Act of 1997) that applies only to devices.

For example, scientists involved in reviewing medical devices at the FDA report an urgent need for predictive software to model the human effects of design changes for rapidly evolving devices. Ashar adds, "There's a learning curve with devices, and we have to try to anticipate the problems users might encounter."

Thanks to the enormous growth in research, the FDA is dealing with more complex and innovative products struggling in development. As discoveries made in the laboratory begin the transformation toward effective medical products consumers can safely use, both the FDA and industry seek a new and better set of tools to accelerate that development and better predict the performance of these new products.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.