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FDA Outlines Considerations for Enrolling Pregnant Women in Clinical Trials

April 9, 2018

The FDA published a new draft guidance on the scientific and ethical considerations for including pregnant women in clinical trials, telling sponsors that data are needed to inform safe and effective treatment during pregnancy, and that it is appropriate to enroll pregnant women in certain situations. The agency said it considers it ethically justifiable to include pregnant women in clinical trials when adequate nonclinical studies have been completed, and when the trial holds the prospect of direct benefit to the women and/or the fetus that cannot be obtained by any other means. In the postmarket setting, an established safety database in nonpregnant women should be available, and inclusion should be allowed when efficacy cannot be extrapolated or safety cannot be assessed through other study methods.

For women that become pregnant during the course of a clinical trial, unblinding should occur so that counseling may be offered based on whether the fetus was exposed to the investigational drug, placebo or control, the FDA said. The risks and benefits of continuing can also be reviewed; those who choose to continue should undergo a second informed consent process. Sponsors should also gather reproductive and developmental toxicology data in nonclinical models, and consider the gestational timing of exposure. “Historically, pregnant women have been an understudied population, and there have been barriers to obtaining data from pregnant women in clinical trials, including concerns about protecting women and their fetuses from research-related risks,” the FDA said. Currently, collection of safety data on drugs used during pregnancy is largely gathered after approval. The agency said it hopes the draft guidance will advance scientific research in pregnant women, within the framework of human subject protection regulations, and serve as a focus for discussion among sponsors, manufacturers, institutional review boards and the academic community. The draft guidance is available here: www.fdanews.com/04-06-18-PregnantWomenGuidance.pdf.

Gottlieb Outlines Plan for Incorporating Patient Experience Data in Regulatory Decisions
FDA Commissioner Scott Gottlieb announced a formal initiative for incorporating patient perspectives into the agency’s risk-benefit analysis framework used in regulatory decision-making. The proposal, outlining steps under PDUFA VI from fiscal 2018 to 2022, includes new guidance by June 2020 articulating how patient experience data gathered by sponsors can be used to support approvals. “The FDA recognizes a need to learn about the clinical context more comprehensively and directly from the perspective of the patients who live with the disease and their caregivers,” Gottlieb said, including information on the severity of the disease, patient-centered endpoints and the degree of unmet medical need. The agency is planning several efforts to systematically incorporate patient experiences into the framework — such as hosting patient-focused drug development public meetings; encouraging external, stakeholder-led meetings; and providing more channels for patients, caregivers and advocates to provide meaningful input into drug development and regulatory decision-making. In addition, the agency plans to build a repository of risk-benefit frame-work examples to serve as references for its reviewers. “Industry stakeholders have indicated that having a clear understanding of FDA’s thinking can help inform a sponsor’s internal decision-making about their drug development programs, particularly early in the product development,” the agency said. The FDA’s proposed plan is available here: https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM602885.pdf.

Project Data Sphere Names GSK Alum Bill Louv as President
Bill Louv, former VP at GlaxoSmithKline, was named president of Project Data Sphere, an independent, not-for-profit cancer data sharing initiative of the CEO Roundtable on Cancer’s Life Sciences Consortium. Project Data Sphere, launched in 2014, is a free digital library and cancer research platform, warehousing patient-level data from cancer clinical trials linked to more than 120,000 participants. More than 1,700 registered users download the data for research use in tumor types such as bladder, breast, colorectal, gastric, kidney, lung, melanoma, pancreatic and prostate. Louv served as senior VP of core business services at GSK from 2010 to 2015, and has over 30 years of experience in the pharmaceutical industry, working in statistics, epidemiology and information technology.

FDA Proposed Fiscal 2019 Spending Including Steps to Foster Drug Development
The FDA published its justification for the Trump administration’s fiscal 2019 budget request, explaining what the agency would do with the funding, and listing its priorities to boost pharmaceutical research and development, among other regulatory initiatives. An increase of $400 million for medical product innovations would advance competition among drugmakers to lower drug development costs and foster innovation and investment in developing treatments for unmet medical needs, according to the FDA. The agency would also direct $58 million to promote domestic manufacturing with the goal of accelerating targeted therapies and enhancing the quality of products, as well as devote $21 million to modernize the FDA’s offices and laboratories nationwide. The administration unveiled its budget request in mid-February, calling for a 13 percent increase in budget authority for the FDA and $190 million in additional user fees, for a total budget of $5.8 billion. The 346-page FDA budget justification is available here: https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/BudgetReports/UCM603315.pdf.

FDA Holding Public Meeting on Inclusion, Exclusion Criteria
The FDA is hosting a public meeting April 16 in Washington, D.C., to evaluate inclusion and exclusion criteria in clinical trials, under a cooperative agreement with Duke University, with the goal of gathering information to assist in the development of formal agency guidance on the subject. The meeting was prescribed by the PDUFA reauthorization bill passed last year. “Certain eligibility criteria in clinical trials can exclude patient subgroups, resulting in the enrollment of study populations that may not be fully representative of that broader patient population,” the FDA said, stating continued efforts to encourage greater diversity in clinical trial populations, such as requirements that sponsors provide analyses of difference demographics and subgroups. Discussion topics will include: the risks and benefits of trial participation; potential regulatory, geographic and socioeconomic barriers; and the rationale for eligibility criteria, as well as the impact of exclusion criteria on the enrollment of infants, children, pregnant and lactating women, the elderly, individuals with advanced disease and individuals with comorbid conditions. In addition, the FDA would like to hear about alternative trial designs that may increase enrollment of more diverse patient populations, how changes to eligibility criteria may impact the complexity and length of clinical trials and opportunities for using data from expanded access trials.