Should companies be allowed to deny patients access to experimental treatments for life-threatening diseases?

Last year Arthur Caplan and Kenneth Moch published a very thought provoking article in the Health Affairs Blog, “Rescue Me: The Challenge of Compassionate Use in the Social Media Era.”  In the article they touch on the ethics and policies of denying access to potentially life-saving treatments. 

Caplan and Moch (2014) state, “The question of whether there is a duty to pay for the use of unproven and experimental therapies to attempt to rescue individuals in dire medical straits, even those with poor odds of success, is one that health policy makers have not acknowledged as part of ongoing national health care debates in the United States and other nations…The duty to seek to rescue those in very dire straits is a powerful norm in American society and in major religious traditions around the world.  Whether it is coal miners trapped in a mine, mountaineers stranded on a peak or a child fallen in a long abandoned well, it is very clear that, despite difficultly and cost, society wants strenuous rescue efforts to be made.  Health policy discussions often fail to reflect this deep moral concern as shown in the absence of attention to the issue of ‘rescue’ and in the lack of funding in public and private insurance for attempting to do so.”    

It would seem then that unless there is in-your-face coverage of the injuries and death caused by denying patients access to potentially life-threatening disease, there will be no movement towards Expanded Access or Accelerated Access Programs for ALS or other rare diseases. 

Caplan and Moch (2014) discuss the case of 7-year old Josh Hardy who in March 2013 was denied access to the experimental drug Brincidofovir (Chimerix, Inc.).  Chimerix, the North Carolina based pharmaceutical company had previously made brincidofovir available to over 430 critically ill patients under an Expanded Access Program initiated by several individual doctor-sponsored emergency INDs in 2009, and later funded for 215 more patients through funding from the Health and Human Services Biomedical Advanced Research and Development Authority (BARDA).  It was only after a social media cry for help (#SaveJosh) followed by a smear campaign against Chimerix (#SaveJosh), which lead to televised media coverage and letters and phone calls to politicians that Hardy received the necessary treatment.  The irony of the situation is that after Chimerix released the drug to Hardy, the company was applauded online and stocks rose by fifty-percent. 

So I ask myself, why are companies developing ALS and rare disease treatments denied expanded access left and right?  Why are there only a few companies that have clear, transparent policies on Expanded Access, procedures, and contacts listed on their webpage?  Why are people who do participate in these clinical trials taken off the treatment to observe for follow-up even when their clinical measures have improved?  Why do we not yet have FDA Guidelines specific for conducting clinical trials research and expanded access programs with ALS and other rare diseases that include input from those actually afflicted? 

Speaking to advocates in cancer, HIV/AIDS, and tuberculosis, diseases where you can actually find Expanded Access Programs available, you will hear that they have faced these same issues.  It is history repeating itself.  You would think that people suffering rare, fatal diseases today would not have to suffer through the same hurdles.  What we should be doing as PALS, CALS, and advocates is work with the U.S. FDA, HHS, NIH, politicians, public and private insurance companies and pharmaceutical/biotechnology companies to overhaul the current system. 

The FDA permits pharmaceutical/biotechnology companies to conduct clinical trials in the United States. Public dollars from NIH, DOD, etc…fund their research and FDA has provided them with additional funding and expedited status for those investigating rare diseases.  In the end the companies negotiate treatment costs and extend patents to the maximum to maintain their high rates.  How are people with the disease benefiting?  How are those who participate in clinical trials benefiting when the treatment is taken away after the trial has ended?

The solution is the political will to rescue people with potentially life-saving treatments like they do for those caught in an avalanche, or buried underground.  We could require companies who conduct trials in the United States to have clear policies on Expanded Access, procedures, and contacts posted on their websites.  We could also require companies to provide a minimum quota of expanded access patients for each clinical trial they conduct.  And also require that if a treatment that is shown to be safe and improve clinical measures to a patient enrolled in a clinical trial, that they be allowed to continue that treatment once follow-up measures have been made.  It would also behoove many to create clear FDA Guidelines for clinical trials research and expanded access programs for ALS and other rare diseases in collaboration with researchers, treating doctors, companies, patients and caregivers.  When all else fails, make some noise.  Social media and televised stories can be powerful tools.