Expanded Access: Myths, Truth and Behavior

Not all companies are big, small companies are scared, and myths drive behavior, stated Kay Holcombe, Senior Vice President, Science Policy at Biotechnology Industry Organization (BIO).

The National Organization for Rare Disease (NORD) ended its Rare Diseases and Orphan Products Breakthrough Summit in Alexandria, Virginia last week with a lively discussion on expanded access.

Holcombe’s statements came after Nancy Goodman, Founder and Executive Director, Kids v Cancer described the case of Josh Hardy, an 8-year-old boy, suffering from cancer who developed an adenovirus infection after being immunosuppressed following bone marrow transplantation. The family, along with mounting public pressure from Facebook, Twitter, etc., demanded a chance at Chimerix’ anti-viral drug, brincidofovir (CMX001) via expanded access, as Josh was not eligible to take part in the company’s trial.

Holcombe was also preceded by a pair of FDA officials, Richard Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research (CDER), and Amy McKee, Clinical Team Leader, Office of Hematology and Oncology Products, CDER, who attempted to dispel myths surrounding expanded access – chief of these myths being that granting expanded use for compassionate treatment may result in negative impact on a drug’s adverse event data.

Both FDA officials stated that after reviewing significant FDA proceedings, they had put to rest the idea that AEs in expanded access had affected the approvability of any product they had seen in their time at FDA.

Holcombe insisted that there needs to be greater transparency into how FDA makes decisions about using data from expanded access.

Small companies are desperately afraid of something going wrong, and FDA needs to do a better job helping companies understand what will happen if something goes wrong in expanded access, she said.

Though they look heartless, these companies do care about patients, Holcombe stressed. But the company’s priority remains focused on its primary trial(s).

Additionally, companies worry about fairness and do not want to be put in a position where they grant expanded access to one patient then have to say “no” to another.

One FDA official emphasized a need for balance as these patients are desperate; the potential for therapeutic benefit is often overstated, and the risk, understated. Patient autonomy must be balanced as well as the benefits to the individual versus the potential societal benefits.