A special session on Monday afternoon titled “The Bioethics of Drug Development – You Make the Call!” featured an interactive discussion on the bioethics of a new drug on the market. This session involved a mock board meeting of a fictional company iCures. The panel moderator, Steve Usdin of BioCentury served as chairman of the board. The other panelists served as board members and included:
- Timothy Mackey from UC San Diego
- Rich Moscicki from the Food and Drug Administration
- Una Ryan from Bay Area BioEconomy Initiative
- Josh Sommer from Chordoma Foundation
- Russell Medford from Salutramed Group, who acted as CEO of iCures
Usdin started off the session by discussing the case of Joshua Hardy, an 8-year old boy from Fredericksburg, VA who had a rare cancer and was treated with bone marrow transplantation. Following his transplantation, Joshua developed a life-threatening adenovirus infection and the primary drug to fight this infection damaged his kidneys. At that stage, it was believed that an experimental drug brincidofovir (CMX001) was the only life-saving option for Josh. This drug was in clinical testing and not FDA-approved – hence unavailable in the market. Joshua’s parents hoped to get this drug under the compassionate use clause, from Chimerix, the biotech company that owns and manufactures brincidofovir.
However, the company refused, stating that it would ethically be wrong to do so for one patient and not for others who had approached them in a similar manner. What followed was a social media storm and pressure from people all over the country. Chimerix finally relented and made the drug available to Joshua, whose condition improved dramatically.
So, was it ethically right for Chimerix to initially withhold the drug for compassionate use before giving in to the pressure? Were patients justified in expecting to receive the drug for compassionate use? Where does the company draw the line?
At the BIO2014 session, the panel (board of the fictional iCures) talked about a similar but hypothetical case of a woman requesting an experimental drug based on compassionate grounds. As CEO of iCures, Russell Medford listed the main issues associated with approving an experimental drug in response to such requests:
- Limited supply of the drug that is primarily manufactured for the clinical trial at hand
- The price tag for off-the-trial drug treatment for the company
- More than one patient requesting drug for indications not being tested in the trial – if drug given to one, what about the other requests
- Can providing open access to drug for other indications affect chances of its regulatory approval for the intended clinical indication?
Medford conceded that a biotech company with a drug likely to benefit patients would like to help, but are faced with real challenges that need to be addressed as well.
Una Ray believes that regulatory issue may not be a concern, considering that the US Food and Drug Administration (FDA) already supports expanded access. However she insists that selection should be fair and medically indicated rather than “someone who has the best social media campaign”. Another factor to consider: if a patient benefits from the drug, a company may ethically “be required” to supply the drug for a long duration (or perhaps lifelong). A related practical consideration is the added costs it will entail. How does a company cover this expense? Do they go back to investors? Do they raise funds from other sources? Ray feels the best approach is to avoid another capital raise and prevent diluting these funds. She favors finding a non-diluted way to fund this added cost. Timothy Mackey suggested that patients or advocacy groups could using crowdfunding to support the costs involved for drug treatment under compassionate use.
In addition, compassionate use of a drug already in clinical testing bears the risk of affecting the ongoing clinical trial; it may not be able to recruit patients to the randomized control trial (with placebo group) that the FDA and the company have agreed to.
Russell Medford agrees and opines, “We have to somehow find a balance between compassionate use from a patient advocacy standpoint for individuals in crisis and the broader groups of patients that are patiently and sometime desperately waiting for us to finalize our clinical trial programs so that we can get the drug out.”
Leighton Read wondered if, by allowing compassionate use, we are going down a slippery slope – making sure that the drug is not toxic, but not really caring much about efficacy? This aspect needs to be focused on to prevent a drug being used indiscriminately.
Though compassionate use of a drug in clinical trials raises legitimate ethical concerns for the biotech company, for regulatory agencies and for society as a whole, the panelists at this BIO2014 session agreed unanimously on this use, after acknowledging these concerns. It remains to be seen how biotech and pharmaceutical companies, regulatory agencies, healthcare providers, patient advocacy groups, and patients can address these concerns.