Docket Management
Docket: 01N-0450 - Prescription Drug User Fee Act (PDUFA); Public Meeting
Comment Number: EC -4

Accepted - Volume 1

Comment Record
Commentor Mr. Karl Schwartz Date/Time 2001-11-21 19:57:04
Organization NHL-info (lymphoma support and information)
Category Other

Comments for FDA General
1. General Comments Q. How can FDA ensure that PDUFA goals are met if there continues to be a funding shortfall? If the funding shortfall persists, should FDA, in order to best protect and promote the public health, set review priorities and, if so, how? Should there be flexibility in setting user fees to cover the increased cost of the program? A. History shows that PDUFA has dramatically improved FDA efficiency, so it must be maintained in some form. Post approval may be the most appropriate time to increase user fund fees as the sponsor will be better able to pay at that time. Fees prior to approval should be lowered to encourage investigation of new agents, if possible. As for review priorities, I believe that agents that have high specificity and that are not myelosuppressive should be given priority for agents that treat cancer. (Systemic treatments for incurable cancers may get the canoe out of the rapids for a time, but they also damage the boat. Thus, agents in this category appear to be an obstacle to improving survival for many cancers.) Agents with specificity are more likely to have safe toxicity profiles and may well be used in combination (once approved) to better control cancer and thus improve survival and quality of life. Certainly, promising agents that have the potential to cure or more safely manage *incurable* cancers should also be given the highest priority. The invisible costs to individuals with cancer of delays in approving effective agents are tragic. Finally, and this may be testing the scope of this discussion, we might also consider the creation of provisional drug approvals for agents that treat incurable cancers, especially for agents with high specificity and good safety profiles, so that large numbers of patients do not lose a chance to benefit. In some circumstances, it is the novel treatment provided early that provides the most hope for improved outcome. During a provisional approval the sponsors can profit, but the agent must be provided at a reduce cost. The sponsor must also pay higher user fees to support additional FDA outcome and safety studies during this time. We can anticipate that a provisional approval system will encourage additional creativity and investments in cancer research. It will certainly reduce financial risk. Importantly, it will provide faster access to treatments that offer a chance to improve survival for patients with incurable cancers. We worry that the current system is too inflexible and system-bound to work efficiently with the explosion of novel therapies that are sure to come. -Karl Schwartz (patient advocate)

EC -4