By Rose Bowen
The pharmaceutical company Johnson & Johnson became the first in the drug industry to create a Bioethics panel of ethicists, doctors and patient advocates to respond to requests for access to experimental medicines, called “compassionate use.” This is only applicable to investigational drugs, or drugs that have not yet been approved by the Food and Drug Administration (FDA). Arthur Caplan, Director of the NYU Division of Medical Ethics, will lead the panel. He has written extensively on compassionate drug use, which gives desperate and terminally ill patients access to experimental treatments. The practice is legal but tightly restricted; although the FDA grants almost all requests for compassionate use before letting the drug companies, who are developing the unapproved treatment, decide. The Bioethics panel would make recommendations to J&J about how to respond to the increasing number of requests for compassionate drug use.
This will be an important first step towards a uniform policy on compassionate drug use between companies, which would make it much less confusing for terminally ill patients requesting the drugs. If those patients are not eligible to participate or live too far away from where clinical trials are conducted, there are only two ways to obtain the drug: either their doctors can request that the patient be part of an expanded access program to drugs in Phase III of development, or the patient’s doctor can apply for single patient access. The doctor then works with the drug company to supply the drug if it is a good fit for the patient.
Companies such as J&J cannot simply grant all of the requests for compassionate drug use mostly due to the limited supply of such drugs. This could delay their approval and limit access for other patients who need the life-saving drug. In the case of the Ebola outbreak, too little of the experimental drug was available and the limited supply quickly ran out after being given mostly to American and European relief workers.
Sometimes when patients’ compassionate use requests are turned down, their families and advocates turn to social media to gain attention and plead their case. For example, “In 2014, the family of Josh Hardy, a 7-year-old boy suffering from a life-threatening infection, appealed to followers on Facebook and Twitter after Chimerix, a company that was developing an experimental antiviral treatment, turned down their request” (The New York Times 5/7/15). While this is sometimes a successful strategy, as in Josh’s case, this can create inequality in access to care and be unethical with regards to patients who are not social media campaign savvy. According to Dr. Caplan, “If we’re going to talk about fairness in providing experimental drugs, we can’t have a system that favors those who are savvy with social media or who have resources” (BU Today 3/18/15).
Another obstacle is cost – drug companies do not always pay for the treatments, and insurance companies generally do not cover them. The unapproved drugs may also not work or cause more harm than good and further shorten the patient’s life. The J&J panel will have the tough job of making recommendations to the company about who should receive compassionate use in a fair and equitable way.
Read more here.
