Follow The Leader: Why Clinical Development Must Transition From Pharma-Led To Patient-Led

By Philip Winkworth and Kelsey Jakee, PA Consulting

“Patient-led drug development” has matured in recent years from an industry buzzword to a maxim practiced in pockets of pharma organizations each day. Yet, much more can be learned by looking through the patient lens, deeply considering their needs, and focusing on their quality of life. In recent years, companies have galvanized their efforts to develop additional methods for gathering patient input, to redesign clinical programs that improve patient experiences, and to track surrogate measures of “patient-centricity” at key stages.

We have seen signals from patients, payers, regulators, and technology companies like Google and Amazon, all of which indicate the need for urgency in fully embracing patient-led development. Right now, innovative strategies are being piloted within the industry; however, it is essential for pharma to further embed these practices. How can companies that have only taken tentative steps to embed new, patient-led approaches during clinical development embrace this strategy and maintain momentum? We have compiled insights from patients, regulators, payers, tech companies, and the life sciences industry below that demonstrate why ensuring development becomes patient-led is paramount to enabling success.

Signals From Patients

The information age’s increased public availability of data has allowed patients to be far more engaged, knowledgeable, connected, and empowered than at any point in history. This impacts not only patients’ own experiences with their diseases, but also the sophistication with which they approach a wide range of decisions, purchases, and time investments in everyday life. Like most consumers, patients are accustomed to the growing movement of “quantified self” — logging all manner of aspects of daily life such as food consumed, mood, etc. — and utilize mobile apps to perform everyday tasks on their behalf. It is with a more holistic, better-informed, and increasingly efficient perspective that many patients are evaluating their options and benefit-risk trade-offs to decide whether to participate in trials and which approved therapies to request from their physicians.

To remain relevant in both the development and commercial realms, companies must understand patients’ desired outcomes from products or services used to treat a disease, and their overall health and lifestyle goals. They must incorporate services that reduce burden as part of clinical trial design, understand communication and engagement preferences, and fit tools and services neatly and unobtrusively into patients’ lives to support adherence. They also must evaluate the information needs of patients along the continuum of engagement with a clinical trial, product, or service — providing patients curated content most suitable to their journey. Where direct benefit to patients’ lives is demonstrated — as perceived by each individual patient — companies will find an intrinsic link to improved patient retention, accelerated product development timelines, and potential revenue.

Indications From Regulators And Payers

Both regulators and payers are beginning to use patient-led approaches to better understand and determine the value of potential treatments. The FDA has in recent years leveraged its own patient advisory forums to solicit patient input on a variety of topics. In many cases, input is gathered regarding patients’ overall experiences with a disease and decision criteria for treatment several stages ahead of an expected drug application. Furthermore, regulators are increasingly likely to request evidence of patient input in clinical trial design during the application review process. Quality of life and related measures are becoming significant alongside clinical endpoints. In addition, outcomes-based pricing is becoming more desirable for payers and insurers, with examples now found in a sizable proportion of health plans. It follows that demonstrating a positive and holistic impact on patient lives, in addition to clinical endpoints, will be key to continued success with those influencing market access.

Innovative Approaches From Tech Companies

Technology advancements are enabling a new age of clinical development that places greater control and insight into the hands of both patients and trial sponsors. Smartphones, health trackers, and wearables generate an exponentially greater volume of and more personalized data, creating novel endpoints and real-world evidence. As modern methods for validated data collection become available, patients, sites, and sponsors will all benefit from the increased use of remote monitoring. Similarly, accelerating the adoption of technologies that deliver more rapid and more varied patient data during clinical trials will allow sponsors to compete for targeted patient groups with the Big Tech firms that heavily invest in healthcare and life sciences.

Being patient-led also means stripping away obstacles that limit patient involvement or autonomy. Capitalizing on available technologies designed to limit patient burden will reduce the stress of participating in clinical trials and provide support for patients to stay enrolled. Reducing the number of reasons for patients to drop out of clinical trials will subsequently lessen the proportion of trials that fail to enroll even a single patient.

Organizations suffering from risk-induced inertia may not embrace new ways of working or apply novel technologies fast enough to keep up. Whether through partnering/acquisitions (e.g., Roche and Flatiron Health), internal innovation, or a strategic balance of both, the industry must select a path or risk being driven off the road.

Observations From The Pharma Industry

Patient-led development is already showing tangible results for companies across areas of portfolio planning, trial design, recruitment, and patient engagement throughout the life cycle of trial participation. In portfolio planning and trial design, companies are beginning to use more robust methods of patient engagement, forming advisory boards, focus groups, and strategic partnerships with advocacy groups. These approaches are generating a high “return on engagement” from cost savings/avoidance and future trial performance. In recruitment, platforms that reach patients directly, rather than through a complex web of healthcare providers or enrolling sites, are becoming more widespread, with major pharma companies not only recruiting patients for their own trials, but also suggesting other trials if patients are eligible.

In the U.K., the National Institute for Health Research developed Join Dementia Research to enable patients and researchers to connect with each other, resulting in 5,000 volunteers registering their interest in clinical studies after just one month. (As of January 2019, the number of volunteers registered was 38,651 and 11,082 had enrolled in dementia studies.) Though active online patient communities are not new, researchers are increasingly taking the lead online to connect patients to trials more directly. For rare diseases, designing trials with patients from the outset and “baking in” their needs and priorities can significantly increase the rate of trial recruitment and reduce dropouts. For Big Pharma, the approach demonstrates potential faster trial recruitment, shorter lead times in development, and associated cost savings with the added commercial advantage of being first to market. Finally, we have also seen how collaborations between pharma and tech companies can lead to site-less trials. This can dramatically improve the experience for patients, allowing them to be “seen” at a meta-site on a mobile app or device. The power of these patient-led approaches demonstrates the potential to reduce patient burden, operational costs associated with traditional site-dependent approaches, and the timelines of recruitment and overall development.

The Road To Success Is Following The Patient Leader

Medicinal and scientific innovation has traditionally served as the root for industry revolution and progress. Failure to adopt and slowness in adapting have often been reasons for companies’ lack of growth. Today, human-centric innovation has moved to the forefront of the industry’s ongoing evolution.

Future success for pharma will be achieved when companies have:

• understood patients’ desired outcomes and designed medicines and drugs around them
• demonstrated to patients, regulators, and payers a medicine’s positive and holistic impact on patients’ lives and well-being
• used and incorporated technology to limit patient burden and ease trial participation
• learned from and continuously improved pilot approaches to patient-led development.

About The Authors:

Philip Winkworth is a U.K.-based life science expert at PA Consulting. He is experienced in product and technology landscaping, portfolio optimization, product launch, and life cycle management. Follow him on LinkedIn at https://uk.linkedin.com/in/philip-winkworth-121a6716.

Kelsey Jakee is a U.S.-based life science expert at PA Consulting. She is experienced in R&D strategy and transformation, patient-centered drug development, and operating model design. Follow her on LinkedIn at https://www.linkedin.com/in/kelseyjakee.