An Evolved Approach to Advisory Boards in Rare Disease Drug Development: 5-Step Model to Finding and Engaging Patient Advisors

Nearly all new product development teams at pharmaceutical companies will routinely conduct patient advisory boards. These board meetings will help collect and document the experience of patients and caregivers for medical product development and regulatory decision-making. Recently, in June 2020, The US Food and Drug Administration (FDA) published a final guidance on methodological patient-focused drug development (PFDD) to address, in a stepwise manner, how stakeholders (patients, researchers, medical product developers, and others) can successfully use these patient forums. In the process of developing this guidance, the FDA acknowledged that leading its own PFDD meetings, especially when limited to organized disease advocacy groups, cannot address the gaps in information on the patient perspective. So, it has expressed support for advancing the science and utilization of patient input other means. Because traditional methods of conducting patient advisory boards often do not achieve the full potential of patient centricity, the authors of this article share an approach to consider when selecting patient advisors, in order to gain the most actionable input to a product development team.

Statement of Data Sharing Goals for Nonprofit Funders of Clinical Trials (Preprint)

UNSTRUCTURED Sharing clinical trial data can provide value to research participants and communities by accelerating the development of new knowledge and therapies as investigators merge data sets to conduct new analyses, reproduce published findings to raise standards for original research, and learn from the work of others to generate new research questions. As a voice for the perspective of participants in clinical trials, nonprofit funders – including disease advocacy and patient-focused organizations – play a pivotal role in the promotion and implementation of data sharing policies. Funders are uniquely positioned to promote and support a culture of data sharing by serving as trusted liaisons between potential research participants and investigators who wish to access these participant networks for clinical trial recruitment. In short, nonprofit funders can drive policies and influence research culture. The purpose of this statement is to detail a set of aspirational goals and forward-thinking, collaborative solutions to data sharing for nonprofit funders to fold into existing funding policies. The goals in this statement convey the complexity of the opportunities and challenges facing nonprofit funders and the appropriate prioritization of data sharing within their organizations and may serve as a starting point for a data sharing “toolkit” for nonprofit funders of clinical trials, to provide the clarity of mission and mechanisms to enforce the data sharing practices their communities already expect are happening.

Rare Disease Interest Group (rareDIG) at McGill University: A Medical Education Pilot Project

One in 12 Canadians have a rare disease, yet medical education continues to espouse Dr. Woodward’s aphorism “when you hear hoofbeats think horses, not zebras.” This produces physician attitudes which are deleterious to the care of people with rare diseases. The McGill University Rare Disease Interest Group (rareDIG) has created programming which sensitizes medical students to the extent and reality of rare diseases.rareDIG helps them to develop attitudes and approaches which shorten the diagnostic odyssey and improve care of people with rare diseases. Success stems from drawing attention to the realities of rare disease through direct patient interaction, creating a strong social media presence, and building collaborations with rare disease advocacy groups and networks. Our inaugural Rare Disease Day event was attended by over 100 attendees including medical students, patients and their families, and a variety of health professionals.Other successes include a Patient Perspective Series addressing the holistic approach to rare disease, shadowing opportunities, “n = rare” journal clubs, and a “Humans of Rare Disease” advocacy project. Medical students represent an important cohort to target with rare disease awareness campaigns that has largely been overlooked by current advocacy efforts. By exposing medical students early in their education to the realities of rare diseases, student-run interest groups can improve medical students’ understanding and perception of rare diseases and ultimately improve patient care in the future. rareDIG strives to continue achieving its objectives in rare disease education and aide other medical schools in creating their own rare disease student groups.

Disease Constituencies

In the second half of the twentieth century, disease advocacy evolved from universal campaigns to patients’ constituencies. Changes in the experience of health and illness and the nationwide expansion of political advocacy laid the groundwork for patient-led campaigns. Then, AIDS and breast cancer activists constructed a new type of disease advocacy on the foundations of the gay rights and women’s health movements. Unlike the earlier disease crusades, these movements were led by patients banding together to fight diseases that affected them personally, and they blazed a trail for patients suffering from other diseases. As patients’ activism became increasingly legitimate, disease nonprofits proliferated, patients took over congressional hearings, and disease walks and ribbons became an inescapable feature of American public life.

Publicity Over Prevention, Cures Over Care

Focusing on diseases shapes the types of goals advocacy organizations pursue and the types of laws Congress passes. Over time, the pressure to adopt goals that fit neatly within disease categories, corporate influence, and the strategic avoidance of controversy encouraged disease advocates to prioritize awareness and research over prevention and access to treatment. This creates a health policy portfolio that subsidizes corporate interests, ignores collective risks, fails to challenge inequalities, and may actually make people less healthy by encouraging overtreatment. Yet while only a small proportion of organizations focus on prevention and treatment access, the phenomenal growth of disease advocacy means that large numbers of organizations continue to pursue the latter goals. Narrow goals outnumber broader goals but do not displace them.

The Rise of Health Activism

Biosociality has proven to be a generative concept for STS scholars, anthropologists and medical sociologists and has been subject to sustained engagement, development and critique. A number of researchers have taken the concept and tested it against a range of empirical sites of inquiry including patient, health and disease advocacy. In particular, when groups have formed in relation to genetic and disease conditions, classifications such as race and gender appear to be powerful mobilizing and shaping forces. But what about social class? Is class a regressive category of little salience today? Or does it help us to understand some of the dynamics of group formation and activism? Drawing on work in medical sociology on class, health and neoliberalism, this chapter explores the ways in which class is salient to discussions of biosociality and patient advocacy movements.