By Roslyn Schneider, RozMD Patient Affairs Consulting
Clinical research has long been considered by most life science professionals to be a way to serve patients — developing novel solutions to address unmet needs. Nevertheless, these professionals have also believed a perceived firewall prevented them from directly engaging with patients, given the highly regulated environment and the attempt to homogenize control groups for the sake of research integrity. Therefore, clinical research professionals most often turned to healthcare professionals to critically assess the complex prioritization and planning of clinical research and to act as surrogates for the patient perspective.
This paradigm has changed dramatically over the past several years. Real-world experiences of people who are impacted by diseases and corresponding treatments — including patients, family members, caregivers, and their advocates — are often referred to collectively as “patient voice.” The value of the patient voice lies in patients’ own experiences, sentiments, words and those of their caregivers and has presented itself in bold relief. Conclusively, what matters to patients — in life, in treatment, in clinical trials — is more specific, and sometimes entirely different, than the priorities of their treating healthcare professionals and the clinical outcomes historically studied in clinical trials.
Most of the life science industry now more regularly conducts what is often referred to as “patient insights work” or “patient engagement” in order to bring direct patient input to all aspects of new product development. The needle is rapidly moving from developing a medicine that is safe and effective in treating a disease to one that additionally improves the quality of life and sense of empowerment for people living with the respective disease. Make no mistake, these goals are not the same.
Examples of patient insights work include meaningful interactions with patients, and caregivers when appropriate, to understand their emotions, needs, and decisions along their patient journey — from seeking diagnoses (recognizing there is a “problem”) through obtaining medical care. It can also include discussion around topics such as their expectations for potential treatment in the future, how clinical trials can be more patient-considerate, barriers to healthcare or accessing medicine once it is on the market, considerations about medicine formulation and packaging, and feasibility of adherence to a medical plan once a medicine is available on the market, to name just a few.
Not only are life science organizations making inclusion of patient voice a best practice for product development, health authorities are asking patients to also contribute to product approval discussions. Even health technology assessments (HTAs) require patients’ input on the value of a medicine.
With fast evolution and new opportunities to collaborate with patient communities, there are also new challenges. Regulations around the world help protect patients from unscrupulous research methods. Undue or inappropriate influence is an area all researchers and organizations must avoid. For this reason, while not directly regulated, determining compensation by the life science industry for this unique, powerful research methodology and business-shaping insights work has been challenging. Further complexity is introduced since similar insights work is conducted by nonprofits and academia. Ideally, consistency and alignment around the principles and methodology to determine fair market value (FMV) could be established across stakeholders with common considerations applied by all researchers.
There is a long history and infrastructure for compensation of healthcare professionals, scientific experts, and consumers participating in market research. While to some it may seem obvious to simply apply consumer market research compensation methods and standards to patient insights work, many have argued that there is strong rationale for a more customized, patient-specific approach to be developed. Typical consumer market research consists of indirectly gathering opinions and preferences from members of the general public, most often without direct contact with the organization sponsoring the research — or the researchers conducting it. In contrast, patient insights work involves patients sharing much more personal and private information directly with researchers, and sometimes even allowing researchers into their homes (with the appropriate agreements around privacy and how information may be utilized).
As you might expect, due to lack of consensus, various philosophies on patient voice compensation exist. In practice, patients are compensated differently by different organizations and for different types of research: Some research projects may pay patients a time-based rate, other activities may limit compensation to reimbursement of expenses, and yet others may not compensate beyond an expression of appreciation for the patients’ contribution.
A new paper “A Call-to-Action: Advancing the Conversation Around Fair Market Value of Compensation for Patient Insights Work” addresses what is happening today, the rationale behind it, and recommendations from and tools developed by patient organizations. It also challenges the members of the life science industry to strengthen collaboration among each other and with other vested stakeholders around a common approach to patient voice FMV.
The paper explores a variety of considerations around compensation for a patient’s time, including how differences among patients might provide important context for determining appropriate compensation. For example, some patients have broader understanding of medicine development or function as advocates for other patients with their disease. The paper also explores a variety of existing approaches to compensating research participants and how these approaches might be considered in the conversation around patient insights work. For example, a value-for-time concept may be agreeable to many organizations and thought leaders but can be challenging to benchmark. The non-profit space has established an FMV for volunteers for specified activities; however, the for-profit structure of the life science industry may require an entirely different lens.
The paper also highlights the complexities of involving patients from diverse regions and countries around the world, recognizing that each has (or doesn’t have) regulations in place to ensure fair compensation. Several professional associations — such as the National Health Council (NHC), the Association of the British Pharmaceutical Industry (ABPI), and Patient Focused Medicines Development (PFMD) — have developed guardrails, but these are also different from group to group, and none spell out a practical FMV instrument. (ABPI recently published a sourcebook that includes a section on FMV for patient insights work and also references many other ongoing multi-stakeholder collaborations in this space.) The paper reflects a call for alignment while also respecting anti-competitiveness laws and local influencing factors for rate determination.
The paper identifies some particular gaps in these compensation models, including whether they are perceived as fair and respectful by patients (this, in itself, is an example where patient voice is needed to inform how we gather patient voice) and whether compensation models result in systematic exclusion of socioeconomic, ethnic, gender identity, or other demographic groups. The paper also lays out some patient- and research-oriented considerations to be taken into account, which may influence the rate of compensation or compensation method. For example, research can be conducted in person or virtually via phone or computer and the desired participants may be in different geographic (urban, rural, remote) locations.
The paper is not intended to be prescriptive and concludes not with a specific algorithm or pay scale but with a call for industry and other stakeholders to continue to collaborate around this important topic. The goal should be a harmonized approach to compensation for patient insights work toward product development, which accommodates geographic, research type, and patient-specific differences. The call to action also begs all parties involved in this harmonization approach to continue joint design of all recommendations with patients, following the lead of such collaborative efforts as those of the European Federation of Pharmaceutical Industries and Associations (EFPIA) Patient Think Tank, the Workgroup of European Cancer Patient Advocacy Networks (WECAN), and the NHC.
While this is no small task, this paper intends to raise even more awareness of and involvement in the ongoing multi-stakeholder discussion and to build on the contributions of several other groups leading this charge in regions around the world. Our hope is that the strengthening dialogue and action will lead to the creation of a framework that reflects the value patients and their insights bring to the entire drug development process.
The author would like to thank Thérèse Johnsen of Novartis, Elizabeth Manning of UCB, and Carrie Corboy of Janssen Global Services for reviewing this article and providing insightful comments and helpful suggestions.
About The Author:
Roslyn F. Schneider, MD, MSc, FACP, FCCP, is principal of RozMD Patient Affairs Consulting LLC. She is a physician transforming healthcare quality and efficiency by partnering with patients who are experts in what they need and want. Schneider applies human-centered design to deliver on health and business outcomes and to create and communicate with stakeholder communities. Patient centeredness has been the common thread through her clinical practice and her academic and industry career, most recently in the global role she created in Pfizer's Chief Medical Office.