1. The FDA memo ignores quality of life benefits and it's risk management of the drug ignores the opportunity to use electronic medical records and observational studies to determine which dose works for what patients. It rewrites the standard for demonstrating quality of life to require randomized controlled trials to demonstrate such benefits....
2. The FDA memo ignores patient preferences and would radically limit the freedom of doctors to prescribe drugs based on their real world experience as opposed to the results of clinical trials focusing on higher than label doses.
3. The FDA ignores the fact that there are no randomized clinical trials assessing the impact of transfusion on survival or mortality.
4. Rather it cites the decline in transfusion-related infections even though the principle reason for using ESAs in chemo-related anemia was to reduce fatigue and sustain hemoglobin levels more efficiently in tandem with newer and more powerful cytotoxic agents/regimens.
5. The FDA ignores the fact that requiring RCTs to establish safety would entail studies of such power that doing so will be nearly impossible. Imposing this standard on all drugs would eliminate many drugs from regular use.
The fix is in. To a large extent the companies have themselves to blame for not tracking the risk and benefits of these medicines more consistently. However denying access to patients who feel better on the drug and who know the relative and absolute risks associated with their use is wrong. And it sends a message to companies that efforts to demonstrate risks and benefits in the post market consistent with the Critical Path will be rejected. The FDA's use of unsophisticated arguments and models in pressing for ESA restrictions underscores that trying to create a patient-centered pathway is simply not worth it. And if it isn't, how serious can the agency be about Critical Path and including patient preferences in its evaluations?