Harnessing Patient-Centered Science to Improve Health Outcomes and Commercial Pharma Success

The term patient centricity has become a mantra for the pharmaceutical industry. However, life sciences companies should think about the need to move beyond abstract commitments to “patient-centricity” and rather embrace patient-centered science as a foundational approach across all stages of the product lifecycle. A crucial component of this entails actively listening to the most important stakeholder—the patient—and using scientifically rigorous patient-relevant data to advance therapeutic innovation.

Understanding the patient perspective, from the patient themselves, has become an ever-evolving and must-have in order for life sciences companies to enhance research strategies, to drive advances in drug development, and to achieve commercial success. This patient-centric initiative is driven by several trends:

• Today, patients are more invested and engaged in their own health than ever before. Patients want to understand how they can improve their health and prevent disease. They crave information including access to their own health data, and many patients are willing to share their health data.¹ However, while credible and accessible information is important; bad health information used in an improper way can be highly detrimental.² Many people turn to Wikipedia for information—it was the eighth-most-visited website in 2020³ and a recent systematic review highlighted it as ranking highly in Google searches for health information and as a resource accessed frequently by the public for this aim.⁴ A study analyzing Wikipedia articles of 10 medical conditions, including diabetes, found statements on the webpages discordant with the latest medical research at the time in 9 of the 10 entries.⁵
• Technology has become a powerful enabler of the patient perspective through a constantly growing array of personal health technologies, such as smart phones, sensors, and other remote technologies. Just think about all the devices on the market that track everything from heart rate to stress levels. Patients have an abundance of their own personal data they can use to inform their health decisions. However, this technology is rarely being utilized to its full potential in clinical research⁶ and is not currently being used in routine healthcare.⁷
• Science has deepened the understanding of and ability to advance more individualized and tailored treatment options enabling patients to make shared decisions with their providers. There have been significant advancements in understanding treatment effects, identifying responders, and incorporating patient needs, perspectives, and priorities into treatment decision-making, but the tools are not always there to support it.
• Regulators and payers increasingly recognize the value of applying a patient perspective in research and decision-making, although this is done inconsistently.

Challenges in the adoption of patient-centered science

To generate patient-relevant research data that can be used to support regulators, payers, healthcare professionals, and patients in making well-informed and reliable decisions, it is essential that scientific approaches are taken. Patient-centered science is evolving as an evidence-based approach for the life sciences industry, which provides a more rigorous scientific underpinning to adopting a patient perspective than a lofty declaration of patient-centricity.

However, adopting patient-centered science has its challenges for life sciences companies. Such challenges are reflected in the current communications of clinical endpoints and in engagements with patients around data:

• Endpoints: In the past, life sciences companies have relied on endpoints that don’t always resonate with patients. As an example, in diabetes HbA1c—a 3-month average marker of blood glucose control—is used as the traditional indicator that clinicians rely on as part of the diagnosis and clinical decision-making, but the blood glucose markers that patients rely on for their own decision-making are fundamentally different—with fluctuations and daily control key. Further, behavioral modifications needed for successful diabetes control are rarely built into the endpoint, so while we aim to understand the impact of treatment on glucose control, it is possible that some of the effect is in fact a result of behavioral changes.
• Health literacy: Many people don’t have the ability to understand and respond to health information. A 2015 survey suggested that 43% of English adults possess insufficient literacy skills and 61% possess insufficient literacy and numeracy skills to routinely understand health information.⁸ This pertains to both the collection of quality data directly from patients (which inherently assumes a level of health literacy to reliably understand and respond to the questions asked about their experiences) and the dissemination of relevant data to patients.
• Communications challenges: Communicating clinical information is not as easy as it sounds. This is increasingly challenging with the rapid evolution of complex science, specifically in the areas of genomics and precision medicine. There are boundless opportunities for life sciences companies and health care professionals to provide clinical and scientific information to a patient in a way that is relevant, understandable, and actionable. However, the traditional approach by clinicians to lead clinical decision-making is only slowly being replaced by shared decision-making where the patient and physician partner around the decision-making.
• Cultural and ethnic bias: The role of cultural and ethnic bias has also been poorly understood, which has limited the understanding, acceptance, and adoption of therapies and lifestyle modifications among minority populations. It is only recently that the medical community and the life sciences industry has started to address the cultural barriers and health disparities that affect various, diverse populations.

So how do we all work to advance patient-centered science?

Several strategic steps should be undertaken to advance patient-centered science:

• Listen to patients: The traditional “push” communications approach where patients are told what to do by the provider or the life sciences company should be replaced by a new paradigm based on listening. This should be done within a scientific research framework as market research and individual case studies can be informative, but neither comprehensive nor generalizable. Fundamentally, the medical ecosystem needs to recognize that the patient is the one suffering from the condition and therefore has unique perspectives, insights, and experiences that should impact all aspects of the patient journey. It’s a learning opportunity for all parties.
• Expand the understanding of endpoints: Traditional clinical endpoints based on the pathology, physiology, and biology of disease need to be expanded with endpoints that relate to functional ability (sleep, mobility, etc.), and the emotional and psychological patient experience.
• Utilize technology: In the era of patient-focused drug development there is a greater use of patient-reported outcome measures (PROMs) in clinical trials. Electronic technology has a key role to play in capturing timely and patient-relevant information to inform study endpoints. There are several areas where technology can be used to enhance patient-focused drug development: Capturing qualitative data from patients; using digital health technology tools; employing reactive technology-enabled clinical outcome assessments; and generating passive patient experience data.

• Rethink the application of patient-centered science: Patient-centered science should be incorporated and adopted across all aspects of the product lifecycle where life sciences companies make decisions about endpoints. This will include the application of Clinical Outcome Assessment (COA) and Patient Experience Data (PED) platforms that intelligently automate capturing patient experiences and information about priorities, preferences, and needs, as well as experiences with treatment, care, and outcomes, through robust and reliable electronic measures, providing real-time insights that inform clinical trial progress and demonstrate results. (See Exhibit. Example scenarios across the product lifecycle where companies may make patient-centered science investment decisions).
• Overcome internal fragmentation: Life sciences companies should aim to overcome the internal fragmentation and silos that impede the ability to think holistically about the patient experience as an integrated approach across the enterprise. This can be a challenge as it may conflict with traditional ways of working in pharmaceutical organizations. It all starts with a few small steps. One example is directly coordinating patient-centered solutions services with the CROs to accomplish more dynamic patient perspectives in clinical trial design, development, and execution.

What does good patient-centered science look like in practice? Albireo Pharma case study

The critically important role of patient-centered evidence was demonstrated in IQVIA’s work with Albireo. Patient-centered science was applied to define clinical endpoints and to secure regulatory approval of a novel drug for the treatment of a rare liver disease in children.

A global rare liver disease company focused on the development of novel bile acid modulators to treat rare pediatric cholestatic and adult liver diseases, Albireo has developed a novel bile acid modulator, odevixibat (A4250), for progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease in children.

In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a negative impact on the daily lives of patients and caregivers. Until last year, there was no approved drugs for PFIC. Only surgical options that include biliary diversion surgery (BDS) and liver transplantation have been available, and without them, most PFIC patients do not survive past the age of 30.

Supported by the global Phase 3 PEDFIC studies, on July 16, 2021, Albireo received European Marketing Authorization for Bylvay® (odevixibat), the first drug approved for the treatment of PFIC in patients aged 6 months or older. Four days later, on July 20, 2021, FDA followed up with a similar approval of Bylvay (odevixibat) in the U.S. for the treatment of pruritus in patients 3 months of age and older with all types of PFIC.

“Until now invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” said Emily Ventura, leader of PFIC Advocacy and Resource Network and mother to a PFIC patient. “As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult—the burden is unimaginable with our kids suffering physically, emotionally, and developmentally.”

Albireo harnessed patient-centered science from the very beginning of the development program for Bylvay, and its experience illustrates the points previously discussed.

Listen to patients: Through semi-structured interviews with patients and/or their parents and clinical experts Albireo was able to understand the unique experience of PFIC patients and to develop a measurement tool for the pruritus symptoms and impacts (e.g. sleep disturbance) which was presented to regulatory authorities.

Expand the understanding of endpoints: Albireo selected a truly patient-centric primary endpoint by measuring both a pruritus improvement endpoint, and a serum bile acid endpoint as primary endpoints in its pivotal trials.

Utilize technology: Through a well-designed, patient-friendly eDiary, patients and/or parents reported their experience of pruritus and other aspects of PFIC, twice daily throughout the trial. Albireo’s strong engagement with parents ensured that completion rates for the eDiary were high throughout treatment.

Overcome internal fragmentation: Albireo implemented an end-to-end patient-centric strategy that began in Phase I and continues in patient support programs and real world studies.

IQVIA has been the scientific partner for Albireo throughout this journey. We look forward to seeing how this information is shared with HTA/payers, patients, and HCPs to support reimbursement decisions/formulary placement, and inform clinical care.

Achieving benefits from patient-centered science

Capturing the patient perspective can generate more relevant, valuable insights that can help drive better outcomes for life sciences companies and, at the end of the day, achieve what we should all put front and center: improved patient outcomes. Companies doing this can claim to be truly “patient centric.” Benefits of capturing the patient perspective include:

• Improved understanding of the patient experience across the product lifecycle by listening to patients
• Enhanced data transparency and quality by enabling patients to better understand the importance of evidence-based endpoints in clinical trials—technology-enabled endpoints can further increase reliability and validity of the data
• Patient-informed clinical trial designs which maximize recruitment and retention
• Harmonized clinical, commercial, and access decisions across the organization
• Reduced study implementation timelines that accelerate access to new therapeutic innovations

These benefits of “patient centricity” can only be fully realized with an emphasis on good scientific principles and practices. If these data are to inform pivotal decisions in pharmaceutical/biotech companies, among regulatory and payer organizations, and in HCP-patient communications in routine clinical practice, they need to be well informed, reliable, non-misleading, and easy to interpret. As such, patient-centered science is emerging as a pivotal effort in medicine along the entire product lifecycle, redefining “patient-centricity” as the generation of patient-relevant and scientifically sound data which is both representative of, and generalizable to, the target population for the treatment under investigation. In this way it is translating the voice of the most important stakeholder—the patient—into relevant data to advance therapeutic innovation. Now it needs to become the mainstay, if not the only way in which patient centricity can be claimed.

Mary New, Vice President, EMEA Lead, Patient Centered Solutions, Jean Paty, Vice President, Patient Centered Solutions, and Matthew Reaney, Senior Principal Scientist, Patient Centered Endpoints; all with IQVIA

References

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