Guest Column | February 18, 2020

Extension Of The Patient: Expanding The Role Of Caregivers In Biopharma Development

By C. Grace Whiting, National Alliance for Caregiving, and Schiffon L. Wong, EMD Serono


The importance of friend or family caregiver input has been recognized by the FDA as important for informing drug development and regulatory decision-making. The FDA has included family caregivers in a series of meetings, of which approximately 50 have been held to date, known as the Patient-Focused Drug Development initiative, or PFDD. These forums allow the agency to hear directly from individuals, including caregivers, with personal experience about what it is like for patients to live with their medical conditions, including their experience of the symptoms of their conditions, the effects their conditions have on their day-to-day lives, their unmet medical needs, and the burdens of available therapies.

As the patient-engagement movement increases in force, the roles for caregivers during medical product development have been illuminated. Some key roles include documenting the natural history of disease in an everyday setting, offering perceptions of benefit-risk trade-offs, and providing input regarding material development to facilitate initiating and adhering to therapy.

Biopharmaceutical companies have begun to realize the value of a more patient-centric operating model to improve drug discovery, development, and delivery. This is true for health conditions where patients may have an “informal” unpaid caregiver, typically a family member, friend, or neighbor, who assists with everyday activities. A caregiver may be critical to enabling a patient’s participation in a clinical trial. For medical product developers, the role of the caregiver is increasing as innovation advances. Caregivers may provide input on clinical trial concepts, protocol design, informed consent creation, and may also improve clinical trial recruitment and retention.

Caregivers Offer A Reservoir Of Information

While the caregiver’s direct or interpreted observations about the patient are valuable, there may be circumstances in which the caregiver’s own experience is relevant to medical product decisions. In conditions with cognitive impairment and in pediatrics, a caregiver may even be asked to “step into the shoes” of a patient, which requires them to have a well-supported, healthy relationship with the person for whom they care. The burden of disease may indirectly impact the health and well-being of the caregiver, especially as a disease advances. For example, caregivers may need to understand how to administer a medicine or treatment, thus making caregiver-relevant labeling and support services valuable to support caregivers to adequately perform medical or nursing tasks. Further, a treatment that requires frequent visits to a hospital or doctor’s office may impact a caregiver’s quality of life by causing strain in their career or finances.

Given their role as a member of the healthcare team, caregiver preferences are often very important to understand. Products or services that reduce disease and treatment burden for both the patient and caregiver may have greater perceived benefit and value to patients, caregivers, clinicians, and even payers. Operational considerations that reduce the burden for both patients and caregivers are important as well.

The report Paving the Path for Family-Centered Design: A National Report on Family Caregiver Roles in Medical Product Development, which resulted from a meeting of 50 professionals from a range of disciplines, organizations, and therapeutic areas of interest, proposes that caregiver input may lead to improved patient options and care. Medical product developers need to be thinking about a holistic integrated patient journey that includes the caregiver. In clinical trials, we often enroll “dyads” of patients with one or more caregivers. The report highlights that caregivers are an enormous and largely untapped reservoir of information about the conditions their care recipients experience, and they should play an important role in therapeutic product development.

The report explores the different relationships between the caregiver and the care recipient. The working definitions for caregivers — observer, reporter, surrogate, proxy — offer insight into distinct potential roles of caregivers. Consideration of these respective roles is a factor in unlocking the value of the caregiver’s inclusion in medical product development. While many may recognize the role of a caregiver in providing emotional support to someone who is managing disease or disability, there are many objective observations a caregiver can make that impact the safety and efficacy of a particular therapeutic. For example, a caregiver can report on a patient’s gait, adherence to medication, changes in behavior, or even physical signs or symptoms that the patient may not notice. Likewise, a caregiver can provide insight into the social and behavioral determinants of health that may help improve clinical trial recruitment, such as language or cultural barriers.

Toward Caregivers As Complementary Partners

While we’re enthusiastic about opportunities to more fully engage caregivers in medical product development, there are challenges and ethical considerations to navigate.

Discerning the potential contributions of each caregiver in each context requires consideration. Research from the American Association of Retired Persons (AARP), the National Alliance for Caregiving, and academic institutions has shown that many caregivers are often asked to assist with medical/nursing tasks without prior preparation. The more complex the care, the more likely that a caregiver has not been instructed on how to help with nursing activities — and is “flying blind” when conducting tasks such as administering medications, giving injections, or changing wound dressings. As such, we need to be cognizant of the risk of increasing strain on caregiver resources of time, energy, and emotional reserves.

It is imperative to balance a patient’s privacy with the caregiver’s full knowledge of medical history, current condition, and treatment decisions; in some cases, a more limited view of the total picture may compromise the caregiver’s ability to participate effectively.

EMD Serono has included caregivers in clinical trial patient advisory boards for more than five years to seek input from patients, caregivers, and patient organizations to help strengthen our clinical development process, and caregivers have been a part of all 10 patient advisory boards developed since the end of 2016.

We encourage key policies and agency engagements to include the development, validation, and broad dissemination of “regulatory-grade” language that articulates ways in which roles for unpaid caregivers could be differentiated from those of patients. Relatedly, defining, funding, and executing a prioritized program of research to undergird practice enhancement is essential.

Where Do We Go From Here?

As America ages, health systems are increasingly relying on friends and family members to provide care. Innovators have an opportunity not only to recognize the caregiver’s foundational role in the care delivery system but to identify how to better leverage the unique input, insights, and support a caregiver can provide as part of a therapeutic regime. We must build a pathway for friends and relatives to provide care without sacrificing their own quality of life and that better recognizes and values their unique perspectives, always protecting the patient’s autonomy and voice. Improving the care that people living with illness or disability receive depends on it.

We need to keep expanding our conceptualization of clinical benefit and societal value and recognize the caregiver as an extension of the patient who is impacted by clinical benefit from a societal and healthcare outcome standpoint.

About The Authors:

GraceGrace Whiting, J.D., is president and CEO of the National Alliance for Caregiving (NAC). She has contributed to several national reports on caregiving, and directed the development of NAC’s Paving the Path for Family-Centered Design: A National Report on Family Caregiver Roles in Medical Product Development. You can connect with her on LinkedIn.


GraceSchiffon Wong is executive director of global evidence & value development at EMD Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany in the U.S. She is also involved with Embracing Carers, a global initiative led by EMD Serono in the U.S. and Canada, in collaboration with leading caregiver organizations around the world to increase awareness, discussion, and action about the often-overlooked needs of carers. You can email her at or connect with her on LinkedIn. To learn more about Embracing Carers, visit