Description of Work:
My current research agenda involves comprehensive multi-dimensional assessments of various aspects of the Food and Drug Administration’s (FDA) pharmaceuticals regulatory process. Component projects of this research agenda are focused on different sites within the FDA’s drug regulation landscape, specifically the summary review process and the drug advisory committee program. In sum, the projects are designed to assess: 1) the impacts of financial conflicts of interest on drugs policy deliberation and decision making, 2) the role of patient and consumer representatives in deliberation and decision making, 3) the effects of differential expert inclusion regimes on pharmaceuticals policy, and 4) the nature and role of environmental hazards analysis in the new drug approval process. These projects use a variety of data types including participant demographics for advisory committees, rhetorically-informed content analysis, policy outcomes data, and FDA safety reports to assess which features or combination of features are most likely to predict certain results (new drug approval, new drug rejection, more or more dangerous side effects, etc).
The first of these projects aims to add to long-standing calls in in medicine, bioethics, and health policy studies for patients and consumer stakeholders to be meaningfully included in health policy deliberation and decision making. A principal concern of these scholarly and advocacy efforts is to ensure that patients and consumers are adequately represented in policy forums traditionally dominated by biomedical researchers, healthcare practitioners, and industry concerns. This inclusion serves not only to make policy deliberation and decision making more ethical but also to improve outcomes. To the FDA’s credit, they have been among the first federal agencies to systematically integrate patient and consumer voices into its decision-making practices. Although these efforts have been ongoing for more than 20 years, there have been no systematic assessments of their effectiveness in terms of ensuring patient and consumer representation. It is precisely this gap my work aims to fill.
Who do patient representatives actually represent?
Since at least the 1980s, a great deal of scholarship in medicine, bioethics, and health policy has argued that patients and consumer stakeholders must be included in health policy deliberation and decision making. This scholarship echoes long-standing calls for stakeholder inclusion from a wide variety of patient and disease advocacy organizations. A principal concern of these scholarly and advocacy efforts is to ensure that patients and consumers are adequately represented in policy forums traditionally dominated by biomedical researchers and healthcare practitioners. In 1991, the US Food and Drug Administration (FDA) responded to such calls by integrating Consumer Representative (CR) and Patient Representative (PR) programs into its long-standing Drug Advisory Committees (DACs). DACs are expert advisory bodies designed to 1) provide independent evaluation of newly proposed pharmaceuticals products and devices, 2) offer independent assessments of newly proposed uses for existing pharmaceuticals products and devices, and 3) deliberate about and evaluate proposed methods for investigating pharmaceuticals products and devices. With the addition of the PR and CR programs, DACs constitute an important venue for expert-stakeholder engagement in pharmaceuticals policy deliberation. According to the FDA, a primary role of PRs is to “provide [the] FDA with the unique perspective of patients and family members affected by a serious or life-threatening disease.” Similarly, CRs are described as “[a] key method of ensuring that FDA obtains the points of view of consumers.”
The histories of these programs notwithstanding, there remain questions as to how effectively the FDA integrates patient and consumer stakeholders into policy decision making. Indeed, the FDA is currently holding a series of special hearings on this matter, which included an open public comment period during November 2014 on regulations.gov. While these meetings and opportunities for public comment are an important part of evaluating the future of the FDA’s patient inclusion practices, the meetings are occurring in the context of a dearth of evidence on the nature of the FDA’s current patient and consumer inclusion programs. To date, there has been no systematic evaluation of the PR and CR programs in terms of participant selection, participation, or policy outcomes. In this article, we take the first of these issues as our primary object of inquiry. Available evidence indicates that many PRs and CRs are also healthcare providers and/or biomedical researchers. Subsequently, this raises significant concerns as to whether or not PRs and CRs truly participate as representatives of their assigned constituencies. Additionally, the data also indicate that many PRs and CRs hold undisclosed financial conflicts of interest (COI) with the pharmaceuticals industry. Although DACs have been subjected to significant scholarly attention regarding the presence and impacts of COI among expert participants, there has been no systematic evaluation of the extent to which PRs and CRs hold similar financial COI. Subsequently, in order to begin the process of providing an evidentiary foundation for ongoing deliberations about key stakeholder inclusion in FDA decision making, we offer a comprehensive analysis of PR and CR selection.
As a result of transparency regulations, the FDA maintains a publically accessible online database of DAC meeting materials including meeting announcements, draft agendas, committee rosters, transcripts, and waivers of COI. Using these online archives, we collected the transcripts for 167 DAC meetings held by 16 DACs between 2009 and 2012. The 167 collected meetings include all meetings transcripts available online at the outset of this study. Using the available transcripts, we identified all PRs and CRs who served at each DAC meeting. PRs and CRs were then subjected to online background and employment history research. We conducted an iterative series of online search queries using Google, Facebook, LinkedIn, and the Internet Archives Wayback Machine. These online background and social media searches were conducted in order to identify any actual or potential COI that were neither waved nor disclosed during the assessed meeting. PR and CR employment histories, resumes or CVs, publically available social media accounts, and advocacy organization affiliations were also located.
Ultimately, our results indicate that PRs and CRs served 315 times at 167 DAC specific matters meetings. 164 (100%) of the consumer opportunities for service involved CRs who were determined to be employed in the healthcare industry. 88 (58%) of the patient opportunities for service involved PRs employed in the healthcare industry. Furthermore, the study identified that 62 (20%) of the opportunities for PR or CR service involved financial COI)with industry. 35 (11%) of these instances were determined to involve a COI rated as high intensity (>$50,000) per FDA guidelines. None of these conflicts was waived or disclosed at the relevant DAC meetings, as required by federal regulations on Special Government Employees. The majority of these unwaived and undisclosed financial COI were imputed through patient advocacy organizations. Because PRs and CRs tend to serve at multiple DAC meetings, 162 (97%) of the meetings included at least one PR or CR with employment in the healthcare industry and/or a COI. Additionally, the data indicate conclusively that every DAC member (including PRs and CRs) in at least 110 (66%) and as many as 162 (97%) of the DAC meetings evaluated held employment in the healthcare industry or a financial conflict.
These findings strongly suggest that FDA PR and CR selection criteria require significant revision. The PR and CR programs are designed to offer patients an opportunity to provide a perspective that may differ from that of biomedical researchers or healthcare practitioners. In so far as up to 97% of DAC meetings included PRs or CRs employed in the healthcare industry employment or holding financial COI, one cannot reliably conclude that PRs and CRs truly participate as representatives of their assigned constituencies. Furthermore, these data indicate the need for more rigorous approaches to COI management for PRs and CRs, especially as relates to relationships with patient advocacy organizations.