A national publicity campaign recently succeeded in obtaining early access to an experimental treatment for a seriously ill child, touching off a broader discussion of compassionate use policies and their impact on drug development and approval. As 7-year-old cancer patient Josh Hardy gained relief from a life-threatening infection following a bone marrow transplant, thanks to Chimerix's promising new antiviral drug brincidofovir, hundreds of patients turned to the Internet and social media to intensify pressure for similar compassionate access. The trend demonstrates the need for sponsors, health professionals and government regulators to find new ways to handle these difficult requests, while also supporting clinical research and biomedical R&D.
Biopharmaceutical companies receive dozens of requests for early access to promising therapies, but often turn them down. Clinical supplies usually are very limited, often just enough to conduct a study. Production costs for biologics are high, particularly for small firms struggling to finance complex research programs.
Moreover, sponsors fear that adverse events with patient populations outside a clinical trial could delay development and approval. And expanded access can interfere with clinical trial accrual; if patients can obtain treatment outside a regulated study, they won't want to enroll in a trial where they risk getting a placebo or a less effective comparator drug.
The social media phenomena raises serious ethical issues about whether hard decisions about who gets access to scarce therapies should be made on the basis of catchy publicity campaigns and political pressure—as opposed to who is most seriously ill and who is most likely to respond to treatment. Some consider lotteries or independent third parties as fairer ways to decide how to distribute a scarce, highly valuable resource. The prime ethical obligation of biotech companies, says the Biotechnology Industry Organization (BIO), is to develop safe and effective drugs as quickly as possible so that broad patient populations can benefit. Diversion of resources to deal with individual access requests can delay development and stymie efforts to achieve equitable distribution of limited supplies.