Patient Advocacy: The Growing Voice of Healthcare
Timely and proper care after receiving a chronic illness diagnosis is extremely important in order to maximize and improve health outcomes. However, navigating the very intricate healthcare system can often feel like maneuvering through a maze with no exit in sight. Patients and their families suddenly become responsible for finding the right care providers, scheduling numerous doctors’ appointments, following complex treatment regimens, and even voicing their concerns to the industry and government decision makers. All of this can be laborious, difficult, and sometimes even close to impossible to manage without some assistance. Therefore, finding the right emotional and informational support at the time when everything seems to be crumbling into pieces becomes imperative. Sarah Johnson, Head of Patient Advocacy for the IQVIA Middle East and Africa region, Durham, NC, USA, points out that in the United Kingdom, healthcare professionals will often provide information about the available patient support organizations at the point of diagnosis, and even if they don’t, patients can usually find the right organizations or nonorganization-centered communities online.
Giving Voice to the Patient in Patient-Centered Care
Over the past few decades, patient advocacy groups such as the American Cancer Society and the Alzheimer’s Association have become increasingly more involved in disease awareness, care, and research. Most of us have likely heard of or even participated in a charity walk for breast cancer, diabetes, arthritis, or some other severe disease. These fundraising efforts help support patients and represent their interests in shaping healthcare towards more patient-centered decision making not only in physicians’ offices but also in clinical research and policy making. This switch to patient-focused care is long overdue. Suzanne Schrandt, JD, Founder and CEO of ExPPect, Arlington, VA, USA, and Chairperson for the ISPOR Patient Council, Lawrenceville, NJ, USA, points out that, “Despite being the only constant in their own care, patients have been the one missing player in system-level healthcare decision making for most of the modern healthcare system.” She adds that while clinician knowledge and insights are extremely valuable, they cannot replace the unique lived experiences of the patients themselves and their caregivers.
Lack of Patient Insight Should Be a Red Light
When it comes to advocating for patient-centered drug and medical device development and health policy initiatives, much of the work has been led by patients, their caregivers, the patient communities and patient advocacy organizations, explains Elisabeth Oehrlein, PhD, MS, Assistant Vice President for Research & Programs at the National Health Council (NHC). Patient advocates are often personally impacted by a disease, whether themselves, a family member, or a close friend. This drives them to challenge assumptions and reimagine healthcare and research, putting patient needs, priorities, and desired outcomes at the center. As one outstanding example, Oehrlein mentions the Parent Project Muscular Dystrophy (PPMD), an organization founded by parents of children with Duchenne muscular dystrophy (DMD), should be mentioned. DMD is the most fatal genetic childhood disorder with no cure, and it affects about 1 in 3500 boys worldwide. When in 2013 European Medicines Agency issued their first draft guidelines for muscular dystrophy drug development and approval, it soon became apparent to the DMD community that these guidelines did not appropriately address the needs of the patients. In response, PPMD and its partners created and submitted to the US Food and Drug Administration the first-ever, externally developed guidance for industry.1
“Despite being the only constant in their own care, patients have been the one missing player in system-level healthcare decision making for most of the modern healthcare system.” — Suzanne Schrandt, JD
Milestones like these illustrate the important role patients bring to the drug development table, not only as study subjects, but also as protocol, methodology, and policy codesigners. In fact, 2017 research conducted by Dr Bennett Levitan and his team showed that patient engagement can also benefit the manufacturers. The team estimated that patient input during the early stages of study design may help avoid research protocol amendments; improve enrollment, adherence, and retention; and consequently accelerate product launch by as many as 2.5 years and add a financial value that 500-fold exceeds the initial patient engagement investment.2
“The world of clinical development is changing, and the patient is taking center stage, quite rightly.” — Sarah Johnson
The biopharmaceutical companies are starting to realize these benefits, and in recent years, patient involvement across the drug development pipeline has gained more traction. “The world of clinical development is changing, and the patient is taking center stage, quite rightly,” Johnson points out and adds that it is important to ensure that the patient engagement happens in a systematic manner, in a way that supports and protects them. In order to provide autonomy and reduce the patient burden, it’s crucial to level the playing field among all involved parties. It’s easy to imagine a scenario in which a patient is invited to a stakeholder meeting to voice their experiences and concerns in a room full of industry, payer, and provider representatives. While most patient advocates do feel very passionate and encouraged to communicate their issues, being outnumbered can often feel intimidating and disempowering. Luckily, this is an easy fix, explains Schrandt, “It’s much easier to feel like you need to be silent when you’re just one person. Having multiple patients in the room really helps with the power dynamic and patients feel that not only can they speak up but that they should do it.”
“When it comes to advocating for patient-centered drug and medical device development and health policy initiatives, much of the work has been led by the patient communities and patient advocacy organizations.”
In addition, patient organizations frequently convene patients, researchers, and other experts to develop tools and resources to make it easier for patients and researchers to work together. For example, NHC-led working groups have developed educational resources to help patients feel prepared to engage in conversations around patient-focused drug development, value assessment, and real-world evidence. For researchers, recently developed resources include guidelines on applying patient insights when designing real-world research, a Patient Experience Mapping Toolbox, and a Patient Engagement Fair-Market Value Calculator to guide compensation. They also collaborate with organizations internationally, such as Patient-Focused Medicines Development (PFMD), to align resources.
Operational Transparency Builds Trust and Credibility
Such an undertaking understandably comes at a cost, and funding often appears to be the topic of contention for the patient advocacy organizations. PPMD, for example, has invested over $50 million in research and therapy development, and larger organizations such as the American Cancer Society have contributed billions of dollars to advance cancer research and improve patient quality of life.3,4 Since the majority of patient advocacy groups are nonprofit organizations, often a large proportion of their support comes from corporate sponsorships and is therefore viewed with skepticism by some. In 2017, Susannah L. Rose, PhD, and her team published findings of their investigative survey and showed that 67% of a national sample of patient advocacy organizations reported receiving funding from for-profit companies.5
"While there is no question that we still have a long way to go and many more milestones to reach, patient engagement in healthcare has currently gained momentum, and we must take advantage of it."
Since these organizations are becoming increasingly more influential stakeholders in healthcare decision making, concerns are being raised regarding potential conflicts of interest and independence. However, Oehrlein explains that with the right protocols in place, good governance practices, and high level of transparency these concerns can be ameliorated. For example, the NHC maintains ‘Standards of Excellence’ for patient organization members related to transparency, expenditures, and governance. That is not to say that every organization that positions themselves as patient advocates has noble intentions. As happens in every industry, on extremely rare occasions, a group identifying itself as a patient organization will instead be serving as a voice or lobbying body for a nonpatient, for-profit entity. Luckily, both experts agree that nearly everyone entering the advocacy field does so because of their passion, commitment, and willingness to improve care for themselves and future generations.
ISPOR Helps to Carry the Momentum Forward
ISPOR, too, has been involved extensively in improving patient engagement in health economics and outcomes research. Annual patient representative roundtables are being held worldwide with an aim to connect patients with other stakeholders. Additionally, through various short courses, webinars, and conferences, ISPOR works towards creating best practice guidelines and facilitates discussions between patients, the industry, and healthcare providers. While there is no question that we still have a long way to go and many more milestones to reach, patient engagement in healthcare has currently gained momentum, and we must take advantage of it. Patient voices must not only be heard, but they should also serve to lead and guide health innovation. After all, they are going to be the end-user of the product and should therefore be the priority.
1. Furlong P, Bridges JFP, Charnas L, et al. How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the US Food and Drug Administration. Orphanet J Rare Dis. 2015;10(1):82.
2. Levitan B, Getz K, Eisenstein EL, et al. Assessing the financial value of patient engagement: a quantitative approach from CTTI’s patient groups and clinical trials project. Ther Innov Regul Sci. 2018;52(2):220-229. DOI: 10.1177/2168479017716715
3. American Cancer Society. 2019 Annual Report. January 4, 2022. .
4. Parent Project Muscular Dystrophy. January 4, 2022.
5. Rose SL, Highland J, Karafa MT, Joffe S. Patient advocacy organizations, industry funding, and conflicts of interest. JAMA Intern Med. 2017;177(3):344-350. DOI: doi:10.1001/jamainternmed.2016.8443.
About the Author
Ilze Abersone, BS, MS is a research consultant for Vital Statistics Consulting, Hoboken, NJ, USA.