“What’s the patient perspective?”
That is a question often heard in health care circles lately. Perhaps the question is asked most visibly in the US Food and Drug Administration (FDA) “Voice of the Patient” series within its Patient-Focused Drug Development initiative
. FDA, however, is not alone. Many involved in health care are now asking that question, including drug and device manufacturers, researchers, legislators, policymakers, health care providers, and payers. Getting the perspective right can be the difference between getting a treatment approved for use and covered for payment.
Accurate and encompassing patient perspectives can be difficult to discern because they don’t exist just to be found, but rather must be synthesized from three primary sources: 1) patients and caregivers, 2) disease specific patient groups, and 3) umbrella advocacy groups. I will describe how these sources contribute to the synthesis of a patient perspective.
While what I describe applies to patient groups in both rare and common diseases, the distinction among the three sources of patient perspectives are more pronounced in rare diseases. Rare diseases are inherently bottom up in that rare disease patients generate and push a pure, empirically derived experience. In contrast, the patient perspectives for common diseases are more likely drawn up from the top and through a filter of preconceived ideas driven by managers, researchers, and scientists. (click here for more on this subject
The Patient and Caregiver
A rather obvious point of getting patient perspectives is to understand their experiences as they describe them, and to understand their preferences as they rank them. With this realization spreading and with medical paternalism eroding, many organizations and institutions are rushing to patients for their views. The patients tell us about the everyday challenges of living with particular diseases, looking for diagnoses, finding treatment centers and practitioners, and getting insurance coverage. They tell us what outcomes matter to them and what they are willing to risk for the outcomes they seek.
Alas, the patient and caregiver input is never uniform. It will vary as disease manifestations vary, as treatment responses vary, and as human experiences and the way they are described vary. Thus, the primary perspective should derive from a good number of direct encounters with patients and caregivers.
The Patient Group
Important input on patient perspectives is available from careful observations of individuals with specific diseases. This aggregated view covers the range of issues patients encounter and can involve medical, social, financial, and emotional dimensions of their experiences. Also appreciated from this approach is the variability among individual patients and caregivers, which can identify and characterize important aspects of a disease experience that an individual patient often cannot. Well-functioning patient advocacy groups are probably the best sources for the aggregated patient experience.
Well functioning patient advocacy groups have definitive vision and mission statements, clear organizational structures, effective communications capabilities, and a critical mass of followers. Ideally, these groups are established 501(c)(3) nonprofit charities. Their very missions are to represent the interests and address the needs of patients with certain diseases. In serving their missions, they become highly familiar with the broad range of patient perspectives and can characterize and articulate them well. Some groups systematically collect, organize, study, and report patient experiences through registries or other types of natural history study programming.
Getting input from rare disease patient groups is generally not as daunting as getting individual patient input just because there are so few groups for a given rare disease. However, there are certainly situations where many groups for a given rare disease exist, and there can be important variability among them. They can have different priorities, policy positions, management approaches, and capabilities. Some groups collaborate with one another, some compete; some groups are cordial with one another, some antagonistic. Patient groups can provide reliable and comprehensive input, but when there are many patient groups for a given disease, they can also present contradictory views that leave observers baffled and stymied. Those who seek patient perspectives from individual patient groups will be wise to find out how many groups exist and to get the perspectives of as many as possible. This is also where the umbrella groups come into play as well.
The Umbrella Group
Individual patient groups have many common objectives and needs. Several groups have formed over the years to address these common needs. These groups serve patients mostly through their support of individual patient groups, and as such, they are sometimes referred to as “umbrella groups”. Examples of umbrella groups that concern themselves with rare diseases include the National Organization for Rare Disorders, National Health Council, Genetic Alliance, Global Genes, and FasterCures.
Umbrella groups interact mostly with individual patient groups, so they have a grasp of patient perspectives across a wide range of diseases. They can contribute to patient perspectives that concern individual groups as a whole. Yet, there is variability among these umbrella groups also. For example, while some overlap in what they do for individual patient groups exists, most of the umbrella groups focus on a certain scope of activities that could fall under categories such as legislative and regulatory needs, capacity building, training and education, fund raising, and direct patient support. These groups also have their own ideas about priorities and policies, which are in concert with one another on some matters and not on others. As can be seen among individual rare disease patient groups, the umbrella groups often collaborate on major issues that affect the rare disease community broadly, but occasionally compete with one another on policy matters or funding opportunities. Thus, umbrella groups can contribute to forming patient perspectives, but no one particular umbrella group should necessarily be seen as the sole source of insights.
From Many, One
Whoever seeks the rare disease patient perspective will do well to keep in mind what Michel Foucault said about being an effective observer:
The only normative observer is the totality of observers: the errors produced by their individual points of view are distributed in a totality that possesses its own powers of indication.
Anyone looking for the rare disease patient perspective will have to become in effect a “normative observer” of a rare disease. This observer will account for views available from patients and caregivers, rare disease patient groups, and rare disease umbrella groups, and will also acquire input from the many constituents of each source. The right patient perspective is not just discovered, or found, or stumbled upon; it’s synthesized. That’s a lot of work. But, there are consequences to getting the perspective wrong, and rewards for getting it right.