USA Today Op Ed Debate on Access to New Medicines for Terminally Ill Patients

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August 21, 2007 - 1:23 pm email to a friend printer friendly view / write comments

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Dr. West

   Today’s USA Today Opinion/Editorials page features a debate about whether patients with terminally ill patients should be permitted to have greater access to investigational agents.  The view of the newspaper staff is here and essentially says that patients who have exhausted the conventional, proven, and FDA-approved treatment opportunities should be allowed considerably greater access, not just in clinical trials and expanded access protocols, which can still be limiting, but also outside of trials, in “compassionate use” programs.  This commentary also describes the lawsuit by the Abigail Alliance for Better Access to Developmentantal Drugs, named for a 21 year-old woman who died of head and neck cancer and had been pursuing agents not approved for this cancer, that has argued that terminally ill patients have a constitutional right to experimental agents (since the US Constitution provides the “right to life, liberty, and the pursuit of happiness”).  A federal appeals court rejected this contention, however, in an 8-2 decision.  The Abigail Alliance is planning to bring this issue to the US Supreme Court. 

   The counter-argument in this Op Ed debate is written by Dr. Andrew von Eschenbach, Director of the US FDA.  He describes the clinical trial process, moving from phase I to phase II and then phase III, and then notes that many thousands of terminally ill patients have received novel agents through such trials.  He also suggests that increased access to investigational drugs in this setting, particularly if accompanied by marketing of such agents (not necessarily by the pharmaceutical company…internet and word of mouth buzz among patients have created significant enthusiasm for DCA or celebrex despite scant clinical data) could undermine the ability to conduct proper clinical trials that are the mechanism to new drug approval, as well potentially create false hope.

   As for me, I’m not so worried about false hope.  People with terminal diseases, which unfortunately includes farr too much lung cancer, are desperately seeking hope, and you can’t have that without some that turns out to be false.  As phenomenal as tarceva is for a small percentage of patients who have a dramatic response that lasts more than a year, there are also plenty of patients who get little or no benefit from it (and the majority with a mild but still real clinical benefit).   I’m not even sure that false hope is clearly a bad thing for people running out of options.  But the practical issues of how it might be done and whether it would undermine ongoing clinical research are real.  There are a nearly endless number of new agents out there, and insurance companies at this point are often not covering all treatments that have proven benefits (not just cancer, but in other settings), let alone every treatment that someone can identify.  I would have a hard time depleting funds for prenatal care to try something that is a lottery odds long shot as 8th line therapy.  Should patients be allowed to have access if they purchase it themselves?  Do we want a system where some people can buy access to far more treatments than other people (and how different is this from our current system where uninsured patients are often up a creek without a paddle while insured patients often are well covered?). 

   I do think it would work best for pharmaceuticals to provide very good compassionate use programs to allow patients without other options to receive their agents that are still early in clinical development, at no charge.  It could be possible for the companies and oncologists to obtain some early feedback on more or less promising agents.  However, there are some major obstacles to this.  First, doctors offices will often will not have the resources to provide lots of follow-up information for the company or to go through very time-consuming processes to facilitate the process.  And companies run the risk that their investigational drug will demonstrate an unforeseen toxicity and endanger the future development of the agent that is their own greatest hope.  Finally, there is a real risk that the clinical trial mechanism that now represents the means to novel drug approval will be short-circuited by back routes to novel agents. 

   It’s easy to say that insurance companies should just pay for everything, or that oncologists offices will just have to find a way to spend the time to coordinate this work.  That’s no more feasible than saying that patients and their families should pay for treatments they want and that they’ll just find a way if they need to.  I do think it would be better for patients to have more access to novel agents, but I think the best way to do that is through minimally labor-intensive compassionate use programs run by the manufacturing companies.  I’m not sure, though, that most would be inclined to assume the risk, or that it definitely be to their advantage in the effort to develop new agents.  But I can’t envision any new approaches that don’t have shortcomings.

   Your thoughts?

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