The Proxy: The Magic Bullet of Healthcare Market Research

As healthcare innovation continues to advance, the focus is expanding beyond common conditions to develop new treatment options and improve the quality of life for individuals diagnosed with rare diseases. This shift underscores the importance of accessing insights from these unique populations, their caregivers, and healthcare providers.

One of the most challenging aspects of market research in the healthcare sector is recruiting participants from the rare disease cohort. To meet required quotas, researchers often need to introduce flexibility within audience requirements, leading to the inclusion of proxy participants.

When carefully selected and well-defined, proxy participants can significantly contribute to the success of a study. Conversely, poorly chosen or inadequately defined surrogate participants can undermine the legitimacy of the insights and compromise the integrity of the data. In this article, we will explore best practices for including representative populations within the rare disease patient sector.

Rare Disease Impact on Research

While rare conditions pave the way for medical advancements through research, the more niche a condition, the less accessible those diagnosed become for participation in market research, particularly for studies requiring in-person involvement. A rare condition is defined as one that affects fewer than 200,000 individuals in the United States and fewer than 1 in 2,000 people worldwide. When conducting patient-centered market research, understanding the incidence of the targeted condition is crucial for designing an effective recruitment strategy.

For in-person qualitative research, it is advisable to incorporate proxy patients into the recruitment approach from the outset, provided the research is not a clinical trial and does not involve treatments or medication administration. By pre-defining acceptable surrogate patients as a flexible component of the recruitment strategy, researchers can ensure a predictable pace, schedule, and budget, minimizing the need for additional approvals and unexpected accommodations during the research process.

The Power of the Proxy

When evaluating the impact of including representative patients in a rare condition study, there are four key elements to consider:

• Prevalence: Pre-identifying and approving surrogate populations to represent rare cohorts can significantly elevate a project's incidence rate (IR). This increases the likelihood that the project will meet its defined quota(s) within the specified timeframe and budget. 
• Accessibility: Reasonably budgeted in-person research requires clusters of patients in defined central locations. For rare conditions, clustering is challenging, if not impossible, without requiring patient travel. Due to the underlying diagnosis qualifying them for the research, patient travel is often unrealistic. 
• Budget: Rare condition in-person research is costly. Without the inclusion of surrogate patients, researchers face two options: research team travel or patient travel. Funding for rare disease research is often limited due to the perceived low return on investment for therapies prescribed to a small patient population, coupled with the high cost of drug development. 
• Stamina: The most researched rare conditions are often in the specialties of oncology, neurology, and pulmonology. These diagnoses are frequently accompanied by decreased stamina due to disruptions in energy production, muscle function, and nerve transmission. Consequently, patients may decline market research study opportunities, especially those that are longer or multi-step, to preserve and protect their energy and health. 

Defining Your Proxy

Following the recommended approach to include representative patients in the study’s recruitment plan, it is crucial to evaluate and prioritize the negotiable versus non-negotiable qualifiers related to your study participants. Whether focusing on patient journey, preference research, or device/software usability, consider what can be replicated by a population with higher prevalence but similar pathways in terms of symptomatology, provider pool, and treatment experience. From there, acceptable surrogate patients can be defined. Examples of successful patient proxies in qualitative market research:

• Supplementing Tardive Dyskinesia patients with those diagnosed with Essential Tremor. 
• Recruiting stroke-induced aphasia patients to meet quotas for Landau-Kleffner Syndrome. 
• Utilizing cirrhosis patients with a diagnosis of Chronic Obstructive Pulmonary Disease (COPD) to recruit for Alpha-1 Antitrypsin Deficiency studies. 

Considerations & Recommendations

Proof of Diagnosis: When recruiting rare and representative patients, it is advisable to require participants to provide proof of diagnosis, typically in the form of an EMR sent via secure transfer to the recruitment partner.

Decentralize: Whenever possible, conduct the research remotely. This approach increases the pool of rare patient participants available while keeping costs low. For small device testing, consider shipping the prototype to the patient with prepaid return arrangements.

Simplify: Availability and stamina are prevalent concerns. Keep the research simple by eliminating long engagements, multi-step, or multi-day study methodologies.

Transportation: Make transportation easy. Depending on the required diagnoses and/or the age of the participants, consider arranging transportation or providing an extra incentive for a family member or friend to assist with transportation.

Over-Recruit: The recommended over-recruitment rate for remote rare patient research is 20%, increasing to 25% for in-person research. Cancellation rates are higher among the chronically ill due to the unpredictability of their health on the study date and limited scheduling options. To ensure quotas are met within the designated timeline, a 25% over-recruitment is recommended.

Your Recruitment Partner

A successful rare patient recruitment effort is fundamentally rooted in the quality of your recruiting partner. Researchers are advised to seek out recruitment vendors who possess clinical expertise, as this fosters collaboration and the design of an achievable rare patient recruitment strategy. It is important to choose a partner with an established presence in the industry, affiliations with rare patient groups, and a proven track record of success in the subspecialty of rare patient research.

In summary, the inclusion of proxy participants in rare disease market research is a powerful strategy that can overcome the inherent challenges of recruiting these unique populations. By carefully selecting surrogate patients, decentralizing research efforts, simplifying study designs, and collaborating with an experienced recruitment partner, researchers can gather meaningful insights that drive healthcare innovation.

These practices not only enhance the quality and reliability of the data but also contribute to the development of effective treatments and improved quality of life for individuals affected by rare diseases. As we continue to push the boundaries of medical research, thoughtful and strategic recruitment approaches will remain essential in advancing our understanding and addressing the needs of these special populations.