Existing FDA Pathways Have Potential To Ensure Early Access To, And Appropriate Use Of, Specialty Drugs

Abstract

Specialty drugs are notable among prescription drugs in that they offer the possibility of substantial clinical improvement, come with important risks of adverse events and mortality, can be complex to manufacture or administer, and are usually extremely costly. The Food and Drug Administration (FDA) plays a critical role in ensuring that patients who could benefit from specialty drugs have access to them in a timely fashion. In this article we review the different strategies that the FDA can use to approve and influence the post-approval prescribing of specialty drugs. When specialty drugs show promise in early clinical trials, the FDA can expedite the drugs' availability to patients through expanded access programs and expedited approval pathways that speed regulatory authorization. After approval, to ensure that specialty drugs are directed to the patients who are most likely to benefit from them, the FDA can limit the scope of the drugs' indications, encourage the development of companion diagnostic tests to indicate which patients should receive the drugs, or require that manufacturers subject them to Risk Evaluation and Mitigation Strategies to ensure that their use is appropriately limited to a restricted population that is aware of the drugs' risks and benefits. Implementing these existing regulatory approaches can promote timely patient access to specialty drugs while preventing expensive and potentially inappropriate overuse.

Right-to-try laws and individual patient “compassionate use” of experimental oncology medications: A call for improved provider-patient communication

“…In this process, the FDA has typically played only a superficial role in decision making. Namely, in recent years the FDA has received approximately 1,000 applications per year and approved about 99.5% of these (Kesselheim et al, 2014; Servick, 2014). …”

“…Critics (Kesselheim et al, 2014; Roth-Cline & Nelson, 2014; Walker et al, 2014) traditionally have argued that while Expanded Access may afford benefits to individual patients, it allows the patients to use the medications without participating in clinical research. Thus, if widely adopted, Expanded Access could undermine public health by diverting resources from randomized clinical trials, (M.…”

“…Thus, if widely adopted, Expanded Access could undermine public health by diverting resources from randomized clinical trials, (M. Goodman, 2014; Kesselheim et al, 2014; Magnus, 2014; Walker et al, 2014) the gold-standard for evaluating experimental medications. Although the FDA’s application process requires confirmation that the use of Expanded Access will not interfere with ongoing clinical trials (FDA, 2013), five lines of reasoning raise concerns.…”

“…Although the FDA’s application process requires confirmation that the use of Expanded Access will not interfere with ongoing clinical trials (FDA, 2013), five lines of reasoning raise concerns. One, the FDA approves nearly all Expanded Access applications (Kesselheim et al, 2014; Servick, 2014), suggesting a low bar for evidence that ongoing trials are not being disrupted. Two, any particular research program has finite resources, and Expanded Access can shift staff time and resources away from conducting clinical trials toward monitoring individual non-research patients in the Expanded Access program (M.…”

“…For example, in the past 1.5 years, 16 U.S. states have passed “right-to-try” laws aimed at further deregulating access to experimental medications (Gaffney, 2015). In contrast, critics of Expanded Access (Kesselheim, Tan, Darrow, & Avorn, 2014; Roth-Cline & Nelson, 2014; Walker, Rogers, & Entwistle, 2014) have suggested that it poses complex ethical tradeoffs: it may benefit individual patients, while potentially undermining research that benefits society. This review reiterates those concerns, while arguing that the benefits for patient care are potentially dubious, given the risks of experimental oncology medications and the known benefits of palliative cancer care.…”