Expanding Patient Access to Compassionate-Use Drugs via Clinical Trials

Los Angeles, CA—Pharmacists can serve as an important information portal for access to investigational drugs through clinical trials or via expanded-access mechanisms, according to Kim Redic, PharmD, BCPS, Manager, Research Pharmacy, University of Michigan Health System, Ann Arbor.

“Fewer than 1% of patients say that they learn about clinical trials from pharmacists, with most relying on the media or on their physicians,” Dr Redic said at the 2013 Hematology/Oncology Pharmacy Association Annual Conference. Most patients, however, indicate that they are willing to hear about clinical trials from their pharmacists.

Clinical trials may take longer than expected to complete, because of recruitment delays. “This is an area where we can play a part,” said Dr Redic. Only 6% of clinical trials are completed on schedule, with 75% running over schedule by more than 1 month. Nearly 25% of National Cancer Institute (NCI) Cooperative Group phase 3 trials fail to attain at least 90% of their recruitment goal.

Enrolling Patients in Clinical Trials
Clinical trials can provide an opportunity for access to a treatment. Government sources for open clinical trials include the National Institutes of Health (NIH) and the NCI. Disease and foundation websites are another source of clinical trial information. Many institutions have websites that identify clinical trials that are open at that particular institution.

The institutions that are not formally a research site can sometimes arrange to become a second institution; in these cases, the patient is enrolled at another institution but receives doses at their home institution.

“The patients may start at NIH but get their doses closer to home,” Dr Redic said. Guidance on criteria for becoming a second institution is available from the US Food and Drug Administration (FDA) at www.fda.gov/RegulatoryInformation/Guidances/ucm126432.htm.

Regulations for expanded access to investigational new drugs (INDs) were revised in 2009. The new regulations have a provision for charging for INDs, which must be approved by the FDA at the time of the IND’s submission.

To qualify for expanded access, the patient must not have any other treatment options, and there cannot be any clinical trials open for which the patient qualifies. In addition, the risks of treatment with the investigational drug must outweigh the risk of the disease or of the condition, and expanded access must not create an adverse impact on the development of the drug.

There is an advanced search field on www.clinicaltrials.gov to search for available expanded-access programs. “These will only be the treatment INDs, the ones which are available and open for large numbers of patients,” said Dr Redic. “If you don’t find anything, you can contact individual sponsors or individual companies that have these drugs in development and see if they are making them available for single patient access.”

Expanded access to a drug may be denied if the supply of the drug is limited, the regulatory work may be deemed too costly, safety problems that may arise could jeopardize the FDA’s approval process, or the expanded-access program may hinder enrollment in other clinical trials.

Dr Redic pointed out that pharmacists must consider that providing a drug outside of a clinical trial in a patient population that it has not been tested in may leave the patient worse off. “This is worth thinking about and reminding your team about, as you are wrestling with this idea of compassionate use,” she advised.

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