In August 2012, Pro Te: Solutio reported on the issue of diversity in clinical trials.¹ That report focused on five key points:

(1) Because ethnic differences may be one factor in determining the risk-benefit ratio of a drug therapy in a specific patient, these differences should be considered during drug development and, more specifically during clinical trials;

(2) Failing to ensure diversity and the proper reporting of demographics in clinical trials may result in an inability to secure study funding from such sources as the National Institutes of Health (NIH) and may also result in the FDA’s refusal to accept the sponsor’s application for approval;

(3) The FDA has taken action, primarily through Guidances, to provide standardized methods of defining, collecting, and reporting race and ethnicity information in clinical trials in order to ensure consistency in demographic subset analyses, to compare results across studies, and to assess potential subgroup differences in safety and effectiveness;

(4) Despite a near consensus that diversity in clinical trials is important, barriers to achieving adequate representation persist, despite the best efforts of sponsors; and

(5) Although barriers persist, there are opportunities to improve diversity in clinical trials.

The August 2012 Pro Te: Solutio report detailed a number of recommendations to increase diversity in clinical trials, including, but not limited to:

• Design clinical trials that include healthcare needs specific to ethnically-diverse populations.
• Work with ethnically diverse physicians to recruit patients;
• Ensure clinical trials involve ethnically diverse investigators; and
• Develop and support community outreach programs.²

This update focuses on important developments that have begun to break down these barriers.

Barriers Persist, But So Does Progress

Since the August 2012, report there have been a number of steps taken by both private entities and the FDA to improve diversity in clinical trials. For example, in June 2013, Pharmaceutical Research and Manufacturers of America (PhRMA) announced that it would partner with the National Minority Quality Forum (NMQF)³ and Microsoft to increase diversity in clinical trials.⁴ Noting the importance of achieving this goal, the Vice President of Scientific Affairs for PhRMA noted that “[p]romoting awareness and creating connectivity that can translate into enhanced participation in clinical trials by a diverse patient population is a priority for PhRMA and our member companies[.]”⁵ He further noted that “[t]his collaboration brings clinical research and healthcare closer to each other to prevent disparities in the evaluation and access to innovative medicines.”⁶

The partnership immediately created a process which could seize opportunities. It included joint outreach efforts, such as those listed in the August 2012, report and agreed to create by Q4 2013 the National Clinical Trial Network (NCTN) online portal, which is “an interactive portal linking communities of patients, practicing physicians and researchers to increase participation and diversity in clinical trials[.]⁷ The goal of the NCTN portal is “to provide a permanent IT infrastructure enabling research investigators to quickly identify minority populations who share a medical need and, when appropriate facilitate their recruitment into clinical trials in a timely and cost-efficient manner.”⁸ This national database allows “clinical trial sponsors [to] locate patients by geographical and demographic characteristics who meet a unique study protocol while simultaneously identifying points of care and community resources that can assist with site locations, investigator and patient recruitment.”⁹ To say the least, a partnership comprised of such influential medical and technology organizations as PhRMA and Microsoft is a significant step taken by private entities to increase diversity in clinical trials.

There are other beacons of hope as well. The Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 required the FDA to study the availability of data on the participation of demographic subgroups (sex, age, race, and ethnicity) in the clinical trial data used to support applications for new drugs, biologics, and medical devices.¹⁰ In particular, the FDASIA required the FDA to report on:

(1) The extent to which subgroups participate in clinical trials.

(2) Whether reports of subgroup safety and effectiveness are reported to the FDA in a manner consistent with the FDA requirements and guidance.

(3) Whether and how safety and effectiveness data by subgroup is made public.¹¹

In August 2013, the FDA issued its report, and concluded that “the statutes, regulations, and polices currently in place generally give product sponsors a solid framework for providing data in their applications on the inclusion and analysis of demographic subgroups.”¹² The FDA also concluded that sponsors were, in fact, describing demographic profiles of clinical trial participants and that FDA was sharing this information with the public in various ways.¹³

Despite the FDA’s conclusion that the current framework is “solid,” it did note areas of improvement.¹⁴ Perhaps the most important area needing improvement was stated as follows: “Whites represented a high percentage of clinical trial study participants for biologic, drug, and medical device applications. In many cases, other racial subgroups were underrepresented.”¹⁵ Fortunately, FDASIA also tasked the FDA with publishing and implementing an “action plan” within one year after the publication of its Report.¹⁶ The FDA established a docket in the Federal Register to allow for submission of comments to the FDA, which will be used to help develop the action plan.

Conclusion

The combination of private partnerships and public/government efforts to increase diversity in clinical trials represents a near universal recognition that this is a worthy goal and that there are still barriers to achieving the goal. But, it also demonstrates a significant commitment to achieving the diversity needed to ensure the highest level of care for all patients. The forthcoming action plan should shed light on how the FDA believes these goals can be accomplished.

---

[1] Spicer, Adam J., No Missing Pieces: The Importance of Diversity in Clinical Trials, Pro Te: Solutio, Vol: 5, Number: 3 (August 2012) at 3.

[2] Id. at 4 – 5.

[3] NMQF is a Washington, DC–based not-for-profit, non-partisan, independent research and education organization dedicated to improving the quality of health care that is available for and provided to all populations.

[4] National Minority Quality Forum (2013). PhRMA Joins with National Minority Quality Forum and Microsoft to Address Diversity in Clinical Trials [Press Release]. Retrieved from http://www.nmqf.org/phrma-joins-with-nationalminority-quality-forum-and-microsoft-to-address-diversity-in-clinical-trials.

[5] Id.

[6] Id.

[7] Id.

[8] Id.

[9] PRNewswire (2013). National Minority Quality Forum Announces Addition of PhRMA to Collaboration to Increase Diversity in Clinical Trials [Press Release]. Retrieved from http://www.prnewswire.com/news-releases/national-minorityquality-forum-announces-addition-of-phrma-to-collaboration-to-increasediversity-in-clinical-trials-214644401.html.

[10] Pub. L. 112-144, 126 Stat. 993 (2012).

[11] 9 Id. at § 907.

[12] 10 FDA Report: Collection, Analysis, and Availability of Demographic Subgroup Data for FDA-Approved Medical Products, August 2013, at 2.

[13] 11 Id.

[14] 12 FDA Report at 58.

[15] 13 Id. at 59.

[16] 14 Pub. L. 112-144, 126 Stat. 993 (2012) at § 907.

Finis