Revoking FDA Approval: Right or Wrong? - Part IX

In my last post, I asked, "Why not, at least, make Avastin available to breast cancer patients who are doing well on it?"

Arguments for:

• Patients should have the ultimate say regarding what treatments -- and risks -- they take.
• The studies neglected a subgroup of women whose tumors respond well.
• Today's science lags behind today's patients' needs, so proceeding without the science is acceptable.
• We need to prevent government from overstepping bounds and abusing regulatory power.
• Without FDA approval, some insurance companies won't pay for it.

Arguments against:

• Decisions must be driven by scientific proof, not emotions or public pressure.
• Making such drugs available off-label will deter patients from enrolling in clinical trials.
• Controlling healthcare costs depends on funding only proven therapies.
• Revocation strengthens the accelerated approval process by respecting the results of subsequent studies

From my perspective, the problems stem from having a one-size-fits-all trial system bogged down in bureaucratic layers and conflicts of interest.

With today’s technology and resources, it is not wishful thinking to believe we could develop more efficient methods of moving promising treatments through the research and approval pipelines. I'd like to see Americans work to develop a healthcare system in which...

• Most patients are treated in trials.
• A variety of trial structures exists to match patients' circumstances (e.g., treatments for life-threatening diseases with no other treatment options, treatments of minor health problems, side effects, and so on).
• We optimize the use of computers to collect and analyze available data.
• The physician-patient bond is preserved.

Comments

[Kirk Hartley]

Well said - let's hope it happens !!

[Annette]

Wendy: I work as a cancer research coordinator and the real solution to this problem is for EVERY patient with cancer to participate in an available clinical trial if they meet eligibility criteria. Unfortunately only 3-5% of eligible cancer patients participate. This leaves a small segment of the patient population assuming treatment risks and the small sample size brings into question the generalizability of the clinical trial data. Additionally, yesterday I attended a lecture by David Casarett MD who spoke on "the science of hope: are we hardwired to overestimate our effectiveness." What I took away was this: without rigorous clinical trials and the strong desire to practice evidence-based medicine (versus anecdotal) we are not providing effective proven treatment to patients and our willingness to overlook data that doesn't fit our desired results harms patients.

[Wendy S. Harpham, MD]

Dear Annette,

Thank you for pointing out that relatively few adult Americans with cancer participate in trials. Many eligible patients don't know about trials. Some eligible patients decline because of misperceptions. In addition, I've known many patients who have wanted to be treated in a trial but could not find a single trial that would accept them. Either the trials were closed or these patients were ineligible due to their age (too young; too old), having received certain treatments in the past, having other medical conditions (such as heart disease), and so on.

Thanks also for sharing your conclusions after hearing Dr. Casarett's presentation. Professionals and patients alike can benefit from the wealth of information that has become available recently from studies designed to explore how patients make decisions and experience hope. I'll devote some upcoming blog posts to this provocative topic.

BTW, if interested, Casarett wrote an excellent book on end-of-life care, titled Last Acts. http://tinyurl.com/hs060910

Keep up the good work, Annette. We need you. With hope, Wendy