A manuscript detailing this study is under revision and awaiting acceptance by the British Journal of Clinical Pharmacology.  The study was funded using an unrestricted grant from myTomorrows, a global company that connects patients to expanded access programs and helps drug companies collect data from these programs. Polak is a real-world data lead at myTomorrows.

One reason why gathering expanded access data may be the only route for drug approval is if conducting a clinical trial would be unethical, as was the case for uridine triacetate. “You can’t deliberately overdose a person and then try to undo the overdose,” said Dr Bateman-House. “It was only in expanded access that they could collect [these] data.”

Another reason, one that is becoming more common in oncology, is if the disease is rare. As cancer types continue to be subtyped further and further, the number of rare cancers rises, and given this rise, the way drugs are assessed needs to change, according to Carin Uyl-de Groot, PhD, professor of health technology assessment at the Erasmus University in Rotterdam, the Netherlands. She is the person who helped conduct the research presented at the Expanded Access Summit and told this publication that using data from expanded access for regulatory submissions may be a “future model” for bringing drugs to market.

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Not So Fast

Despite the potential value of mining data from expanded access programs, the fact remains: Expanded access was never intended for research; it was only launched for the purpose of providing treatment. As a result, only a very limited amount of data can be collected and any efforts to collect more data or create more steps in the process could place undue burden on the patient and physician and ultimately delay access to a drug. Dr Bateman-House said this trend is “so new” that it’s going to take a while to come up with best practices.

In addition, unlike data from clinical trials, which are required to be reported (although not all sponsors are compliant to this rule), data from expanded access programs do not have to be posted. “It is completely up to the company at this point in time whether it wants to release that expanded access data,” said Dr Bateman-House. However, she said if transparency is required, that will “probably diminish company interest.”

The FDA is trying to maintain some level of transparency for expanded access data used to support drug approvals. A spokesperson for the Center for Drug Evaluation and Research (CDER) at the FDA told Cancer Therapy Advisor that a new drug application submission should include either “all” expanded access data, or evidence supporting a conclusion that the “partial data” provided did not introduce bias in the assessment of safety or efficacy.


  1. Reagan-Udall Foundation for the FDA. Leveraging real-world treatment experience from expanded access protocols. Public meeting report. November 19, 2018. Accessed February 20, 2020.
  2. Hyman D, Kummar S, Farago A, et al. Phase I and expanded access experience of LOXO-195 (BAY 2731954), a selective next-generation TRK inhibitor (TRKi). Presented at: American Association for Cancer Research (AACR) Annual Meeting 2019; March 29-April 3, 2019; Atlanta, GA. Abstract CT127.
  3. Susan E Prockop SE, Reshef R, Tsai DE, et al. Long-term outcomes of patients with Epstein-Barr virus-driven post-transplant lymphoproliferative disease following solid organ transplant or allogeneic hematopoietic cell transplant treated with tabelecleucel in a multicenter expanded access program study. Presented at: at the 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 81.
  4. van Rosmalen J, Uyl-de Groot C, Polak T. RWD from EA Programs: An overview of FDA and EMA approvals. Third Annual Expanded Access Summit; January 27–29, 2020; Washington, D.C.