Wednesday, August 9, 2006
[1.] My first proposed change to the drug-approval process relates to the current lack of long-term safety data. . . .
To encourage the generation of such data, an extended period of exclusivity should be offered to drug manufacturers after the completion of FDA-mandated studies that demonstrate a drug's long-term safety.
[2.] My second proposed change to the drug-approval process relates to . . . unmet phase 4 commitments. I propose that we provide incentives for the completion of these commitments by offering only a limited, shorter period of exclusivity with accelerated approvals. I also suggest that we allow the normal period of extended exclusivity to be restored when the mandated phase 4 studies are completed and yield data that confirm the drug's efficacy and safety.
[3.] To encourage the development of drugs with high commercial risk to prevent chronic diseases, we need to develop a strategy that will convert the approval of such drugs on the basis of apparently beneficial changes on a relevant surrogate, imaging, or biologic marker to the demonstration of benefit on a clinically meaningful end point (e.g., prevention of cognitive deterioration in patients at risk for Alzheimer's disease or avoidance of the need for hip replacement in patients with early osteoarthritis).
When too much time is required to demonstrate a clinically meaningful benefit in a preapproval study, initial FDA approval could be given on the basis of a change in the end point of an imaging or biologic marker. However, that initial approval would provide only a limited period of exclusivity during which the sponsor would have to demonstrate that patients also benefited with respect to a clinically meaningful end point (i.e., meaningful improvement in function or a reduction in morbidity or mortality). The timely provision of such data would result in an extension of the period of exclusivity. . . .
[4.] Our current drug-approval system does not adequately reward the development of the most beneficial drugs. Manufacturers of drugs that are not demonstrably different from many others on the market are offered extended periods of exclusivity, although the risk involved in this drug development is relatively low. . . .
Once an area was designated as being high need and high risk, an extended (i.e., longer than current) period of exclusivity would be available to manufacturers of successfully developed drugs in this area, and the extended exclusivity would increase the value of these drugs.
Manufacturers of late drugs in a class, sometimes called "me too" drugs, would be given a shorter period of exclusivity unless or until they demonstrate that their drugs had some meaningful advantage (such as increased efficacy or improved safety) over other drugs in their class.