Pharmacists can serve as an important information portal for access to investigational drugs through clinical trials or via expanded-access mechanisms. Unfortunately, less than 1% of patients say that they learn about clinical trials from pharmacists, with most relying on the media or their physicians, said Kimberly Redic, PharmD, BCPS, at the Hematology/Oncology Pharmacy Association 9th Annual Conference. Most patients, however, indicated 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 Redic, manager, Research Pharmacy, University of Michigan Health System, Ann Arbor. Only 6% of clinical trials are completed on schedule, with three-fourths running over schedule by more than 1 month. Nearly one-fourth of cooperative group phase 3 trials fail to attain at least 90% of their recruitment goal.
Clinical trials can provide an opportunity for access to a treatment. Two government sources for open clinical trials are the National Institutes of Health (NIH) and the National Cancer Institute. Disease and foundation websites are another source of clinical trial information, and many institutions have websites that identify their open clinical trials. Of note, institutions that are not formal research sites can sometimes arrange to become a second institution; in these cases, patients are enrolled at another institution but receive doses at their home institution. “The patients may start at NIH but get their doses closer to home,” Redic said. Guidance on criteria for becoming a second institution is available at http://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 US Food and Drug Administration (FDA) at the time of the IND submission.
To qualify for expanded access, the patient must have no other treatment options and there cannot be any clinical trials open for which the patient qualifies. In addition, the risks of treatment with the IND must outweigh the risks of the disease or the condition, and expanded access must not create an adverse impact on the development of the drug.
The advanced search field on www.clinicaltrials.gov can be used to identify available expanded-access programs. “These will only be the treatment INDs, the ones which are available and open for large numbers of patients,” said 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 is limited, the regulatory work is deemed too costly, safety problems that may arise could jeopardize the FDA approval process, and the expanded access program may hinder enrollment in other clinical trials.
Redic reminds pharmacists to consider that providing a drug outside a clinical trial, in a patient population the drug hasn’t been tested in, may leave the patient worse off. “This is worth thinking about and reminding your team about as you’re wrestling with this idea of compassionate use,” she said. —WK l
Redic K. Accessing drugs in development: an update to expanded access (compassionate use) and participation in clinical trials. Presented at: Hematology/Oncology Pharmacy Association 9th Annual Conference; March 22, 2013; Los Angeles, CA.