Over the last several months there has been increased media coverage of “compassionate use” cases – or the treatment of seriously ill patients with experimental drugs or biologics outside of the traditional clinical trial process, when other treatments are not working. These cases are always difficult and painful for everyone involved - the patients, their families, and the companies working to find cures and gain FDA approval of their products as quickly as possible.
While BIO does not comment on individual companies or their decision-making in regards to compassionate use, as the trade association representing the industry, we can comment on the issue of early access to experimental products that have not yet received FDA approval.
Biotech companies have an ethical obligation to develop safe and effective drugs and biologics and make them available to all patients as quickly as possible. There are instances when patients may request access to drugs and biologics prior to their approval. These requests typically come from seriously ill patients or their families who believe an experimental product could save or prolong their lives. In such cases, companies often have to address the challenge of equitable distribution of limited drug supply to a large number of patients in need. These decisions are particularly difficult and heart-wrenching when we know the personal stories of the individual patients. Today’s social media campaigns can make certain individual stories more visible than others, which makes the fair distribution of experimental drugs even more challenging.
People at biotech companies therefore often must make emotionally difficult decisions when trying to balance an individual's early access to a drug still in clinical trials against the company’s obligation to develop drugs for larger groups of patients and ensure these products gain regulatory approval as quickly as possible. In some cases, such early access programs could create a conflict between these two principles. In addition, even when compassionate use programs are in place, difficult decisions sometimes still must be made about which patients will get access to the limited amounts of the drug. These are issues of great concern to people in biotechnology companies, to regulators, and to patients and their families. All of the people concerned with public health, as well as the health of their own loved ones, want to ensure that safe and effective medicines are available as quickly as possible to people in need.
The scientists and researchers in biotech companies have dedicated their lives to helping others by conducting years of research to find cures for disease. Yet they know that even very promising early results do not mean that the experimental drug will help all patients in need or ultimately be approved as safe and effective; indeed the opposite may be true, and this is why further testing is always required. It also is essential for companies to consider whether the development and administration of a compassionate use program will be able to serve fairly all the patients potentially in need, and whether such a program will draw the attention of key company personnel and other resources away from the crucial task of getting the drug approved for a wider population.
We recognize that the complex balancing of factors related to compassionate use programs can at times create situations that appear on one hand as deeply unfair to a single patient or family facing the unthinkable and holding out hope for a miracle. Yet the reality facing companies presented with these situations is whether individualized decisions could delay FDA approval of a drug or therapy for use in a larger number of suffering patients. BIO believes that these complex and difficult decisions require thoughtful involvement of the people working in biotech companies, regulators, and representatives of patients and their families. While the case of each individual patient may be moving and compelling, very difficult decisions must be often made to ensure fair and optimal use of limited resources, in order to achieve full evaluation of a drug’s safety and effectiveness as quickly as possible.
Because of the importance of this issue to BIO members and the patients we serve, our Board Bioethics Committee reviewed the topic in depth and issued "Points to Consider" in April 2010 to help members analyze the many ethical challenges raised by early access programs. This document is available here: http://www.bio.org/sites/default/files/20100416.pdf . The Board Bioethics Committee will continue to work on developing a better path forward for all parties involved in such difficult and emotional cases.