Overview of FDA’s Expanded Access Program for Investigational Drugs

Jarow, Jonathan P.; Lurie, Peter; Ikenberry, Sarah Crowley; Lemery, Steven J.

doi:10.1177/2168479017694850

Cited by 67 publications

(75 citation statements)

References 5 publications

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“…Compassionate use is a mechanism to obtain investigational agents that are not yet FDA approved, and active involvement of a health care provider can facilitate success in obtaining an investigational drug . Our study highlights the perceived complexities and challenges associated with gaining access to investigational agents in pediatric oncology.…”

Section: Discussionmentioning

confidence: 99%

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Physician perspectives on compassionate use in pediatric oncology

Moerdler

Zhang

Gerasimov

et al. 2018

Pediatric Blood & Cancer

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Background Targeted cancer treatments are almost always first studied in adults, even when there is a biologically plausible potential for efficacy in children. Through compassionate use programs, children who are not eligible for a clinical trial and for whom there are no known effective therapies may obtain access to investigational agents, including drugs under development for adults. However, little is known about pediatric oncologists’ experiences with applying for and obtaining compassionate use agents. Methods This study surveyed 132 pediatric oncologists to assess awareness and utilization of compassionate use programs, to identify barriers to their use, and to evaluate available institutional support and resources. Results We found that the process of applying for access to drugs in development is poorly understood, which presents a barrier to obtaining investigational drugs. Fifty‐seven percent of the pediatric oncologists applied for compassionate use. Providers from larger institutions or with more than 15 years of clinical experience were more likely to complete an application and obtain investigational agents for their patients. Conclusion Identified perceived and actual barriers to compassionate use application submission suggest pediatric oncologists may benefit from educational resources and support to ensure children with cancer equal access to investigational agents and care.

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Section: Discussionmentioning

confidence: 99%

“…According to the FDA, clinical trials are “the most efficient means to make safe and effective treatments available to the largest number of patients,” but exclusion criteria make it impossible for some patients to enroll. For children, age is one of the leading factors that make them ineligible .…”

Section: Introductionmentioning

confidence: 99%

Physician perspectives on compassionate use in pediatric oncology

Moerdler

Zhang

Gerasimov

et al. 2018

Pediatric Blood & Cancer

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“…As noted above, if the FDA does not respond within 30 days, treatment may proceed. 6 (Certain expanded access protocols are not subject to the 30-day requirement.) Although expanded-access submissions represent approximately one-third of all IND submissions, the vast majority of these are for individual patients and do not typically require substantial agency resources to review.…”

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confidence: 99%

“…The FDA does not provide clinical advice for the individual patient (this is the responsibility of the requesting physician), but the FDA can recommend revisions to the treating physician's desired treatment plan to better protect the patient's safety. 6 The FDA acts quickly in response to individual patient expanded access requests and allows the vast majority of requests to proceed. For an emergency use application, access to the drug may begin on verbal authorization (usually over the telephone) by the reviewing FDA staff.…”

mentioning

confidence: 99%

How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

McKee

Markon

Chan-Tack

et al. 2017

The Journal of Clinical Pharma

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In this review of individual patient expanded-access requests to the Center for Drug Evaluation and Research for the period Fiscal Year 2010 to Fiscal Year 2014, we evaluated the number of applications received and the number allowed to proceed. We also evaluated whether drugs and certain biologics obtained under expanded access went on to be approved by the Food and Drug Administration. Finally, we considered concerns that adverse events occurring during expanded access might place sponsors at risk for legal liability. Overall, 98% of individual patient expanded-access requests were allowed to proceed. During the study period, among drugs without a previous approval for any indication or dosage form, 24% of unique drugs (ie, multiple applications for access to the same drug were considered to relate to 1 unique drug), and 20% of expanded-access applications received marketing approval by 1 year after initial submission; 43% and 33%, respectively, were approved by 5 years after initial submission. A search of 3 legal databases and a database of news articles did not appear to identify any product liability cases arising from the use of a product in expanded access. Our analyses seek to give physicians and patients a realistic perspective on the likelihood of a drug's approval as well as certain information regarding the product liability risks for commercial sponsors when providing expanded access to investigational drugs. The US Food and Drug Administration (FDA)'s expanded-access program maintains a careful balance between authorizing patient access to potentially beneficial drugs and protecting them from drugs that may have unknown risks. At the same time, the agency wishes to maintain the integrity of the clinical trials process, ultimately the best way to get safe and effective drugs to patients.

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“…Thus, it may help to overcome some of the ethical problems associated with expanded access. Systems for expanded access differ across countries, but have a set of conditions in common: Patients must be suffering from serious or life-threatening diseases must have exhausted standard treatment options, and must not be eligible for participation in clinical trials (Jarow et al 2017). The managing physician must believe that the potential benefits of the drug will outweigh the risks.…”

mentioning

confidence: 99%