Arthur L Caplan, J Russell Teagarden, Lisa Kearns, Alison S Bateman-House, Edith Mitchell, Thalia Arawi, Ross Upshur, Ilina Singh, Joanna Rozynska, Valerie Cwik, Sharon L Gardner

First published July 2018

In the USA, the public policy goal underlying the regulatory approval of drugs is ensuring that only safe and effective drugs reach the market. This requires extensive animal and human testing. But individuals who are dying or seriously ill often perceive themselves, rightly or wrongly, as unable to wait for these new drugs to be approved. They seek to maximise their individual chances of cure or remediation, while the regulatory process is oriented towards medical products for use by populations. In
this context, there is a fundamental moral tension between protecting the public and responding to the plight of very ill individuals. The latter may require nothing more than allowing informed, consenting individuals to pursue any chance regardless of lack of information about efficacy or safety. But, creating a system in which informed, consenting individuals can try anything would destroy the clinical trials that prove a new product’s worth, because those individuals would have no reason to risk being
randomised to a placebo or standard of care arm in a trial. The unintentional consequence would be harming the public that the regulatory system is supposed to protect. It is clear that neither a framework that values only individual ends nor one that
values solely public ends is desirable.

Patients who seek individual access to experimental treatments, sometimes called ‘compassionate use’ or ‘expanded access,’ have exhausted all available options. They are unable to benefit from existing, approved products; they do not qualify for clinical trials for a variety of reasons; they cannot enrol in an expanded access programme making the experimental treatment during the period between a clinical trial’s end and regulatory approval of the product or no such programme exists. For such patients, the only option is to seek access to an experimental treatment from a pharmaceutical company. Pharmaceutical companies, however, are not responsible for trying to save individual patients. Their mission is to develop products that will ultimately gain approval from the FDA and other regulatory bodies so they can sell them to all patients in need.