2004N-0181 - Critical Path Initiative; Establishment of Docket
FDA Comment Number : EC14
Submitter : Dr. Robert Beall Date & Time: 08/02/2004 06:08:08
Organization : Cystic Fibrosis
Consumer Group
Category :
Issue Areas/Comments
CF Foundation Submission - Part 3 of 3, see Parts 1 & 2 and attachment to Submission part 1, temporary comment number 4179

New Uses for Old Drugs
We encourage Congress and the FDA to examine ways to encourage investigations into off-label uses of approved medications for rare diseases. The CF Foundation has been successful in demonstrating the benefits of the drug, Azithromycin, in a Phase III trial which enhanced the health of people with CF through improved lung function and weight gain, and reduced hospitalizations compared with placebo. However, these large studies are costly, and companies do not have sufficient incentives to identify new uses for already approved medications for rare diseases. We encourage Congress to provide incentives for off-label uses of drugs for orphan populations, comparable to the incentives in the Best Pharmaceuticals for Children Act or the Orphan Drug Act.

In addition, we are facing greater difficulty achieving rapid accrual to important CF trials, despite the general willingness of CF patients to participate and the efforts of CF clinical centers to educate patients about trials. We encourage the FDA to undertake steps to educate the public about the essential role of individuals who participate in clinical trials.

Standardization of Devices
People with CF must use several medical devices to maintain or improve lung health. With the availability of more drugs that must be aerosolized for delivery to the lungs, the development of user-friendly delivery devices for multiple types of drugs and biologics is critical. We urge the FDA to identify ways to encourage manufacturers to standardize device performance specifications or to create more uniformity in devices for aerosol delivery. This will enable product development to be undertaken from a patient-centered approach, which is critical for overall patient acceptance and adherence for improved health.

Partnership for Progress
To achieve our goal of curing disease, the CF Foundation regards the FDA as a critical partner in the development process and encourages the agency to work closely with nonprofit research advocacy organizations in developing life-saving products. Just as the 'Roadmap for Medical Research' by the NIH will guide basic research, the FDA's commitment to improving the Critical Path plan has the potential to influence clinical development for generations.

In summary, the CF Foundation is pleased that the FDA is undertaking a thorough review of its role in the product development process and striving to identify areas for improvement. We believe this examination is essential to bring products to patients more quickly and safely. Our suggested areas for improvement include the following:
- FDA leadership on regulatory oversight and streamlining of IRB review;
- Continued improvement in the orphan product grant effectiveness for rare diseases;
- Identification of processes for the FDA to determine and accept clinical endpoints;
- International harmonization regarding clinical development overseas;
- Incentives for clinical trials for off-label uses of approved medications for rare diseases;
- Education of the public about the importance of clinical trial participation; and
- Standardization of performance specifications for certain medical devices.

Thank you for the opportunity to comment on the FDA's Critical Path to reevaluate agency programs in order to bring life-saving products to patients more rapidly, more efficiently and without compromising patient safety. The CF Foundation looks forward to working closely with the FDA to achieve these goals for people with CF.


Robert J. Beall, Ph.D.
President & CEO
Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814