Patient experience data (PED) is an increasingly important cornerstone in successful intervention development. Scientifically rigorous, easily interpretable and actionable PED can help researchers and clinicians make pivotal decisions during the development and commercialization of interventions. But the field of PED research is a complex one, and it can be difficult to decide when and how to collect the right PED from the right population.

The Patient Centered Solutions (PCS) team at IQVIA has issued a new book exploring this topic in depth. Using Patient Experience Data to Evaluate Medical Interventions brings together articles written by a unique combination of experienced physicians, medical researchers, social scientists and strategy consultants with a passion for patient-focused intervention development. The book aims to provide a robust overview of how PED can be generated during intervention development, and how it can be utilized to inform decision-making at the regulatory, payer, healthcare professional and patient levels.

Using Patient Experience Data to Evaluate Medical Interventions is available on the Amazon Kindle store, where it has been ranked as the #1 new release in Medical Research (May 2023). Note: It is not necessary to use a Kindle device to read a Kindle book. The free Kindle app is available for iOS, Android, PC and Mac.

Below is an excerpt from the book’s introductory chapter, written by editor Matthew Reaney, Ph.D. Dr. Reaney is Senior Director, Global Science and Analytics Lead & Head of the Patient Centered Solutions Institute at IQVIA.

The relevance of PED extends beyond patient-level healthcare and into the development of new interventions. Indeed, understanding PED in intervention development is an important precursor to patient-focused healthcare provision. The goal of intervention development has long been to maximize health and well-being, but it is only recently that developers and sponsors of intervention development have recognized the importance of the patient experience in achieving this goal. For an intervention to improve health and well-being, it must be respectful of and responsive to the general preferences and priorities of patients (recognizing social and cultural differences that may exist in societies), meet patient-defined unmet needs, and appeal to patients; or else people will not accept it or adhere to it. If we cannot offer patients the best possible experience, health and well-being cannot be maximized. To know whether and how we are doing this, we must engage in rigorous, focused and generalizable PED research during the development of the intervention.

PED is defined variably and somewhat inconsistently in the context of intervention development, with the U.S. government offering the clearest definition: data that is collected by any person with the intention to provide information about patients’ experiences with a disease or condition, including the impact of the disease or condition or related therapy or clinical investigation, and patient preferences with respect to treatment of the disease or condition.¹

If we cannot offer patients the best possible experience, health and well-being cannot be maximized.

Patient-focused medicines development (PFMD)² and patient-focused drug development (PFDD)³ initiatives have spearheaded the drive towards the generation of PED inside and alongside clinical trials (i.e., during the development of an intervention). These initiatives highlight numerous important research questions which may be important to answer through PED to determine the value of the intervention during development. For example, PED can be used to identify unmet needs that are important to focus on when developing a new intervention and help us to design patient-focused trials in which to test the new intervention. PED is particularly important in the modern fast-paced world of intervention development, as novel treatments with new mechanisms of action (e.g., gene therapy, CAR-T, COVID-19 vaccines) seek to change the treatment paradigm, and as novel settings for collecting data emerge (e.g., decentralized clinical trials). A variety of different PED research methods, including literature reviews, interviews and focus groups, administration of clinical outcome assessments (COAs), preference surveys, and the use of digital health tools, can be used to generate PED. When used appropriately, these offer a systematic, data-driven approach to understanding the patient which can help developers and sponsors create an intervention that offers more value to patients. And interventions that offer more value to patients are more likely in turn to offer greater rewards to intervention developers and sponsors through approvals for use, reimbursement, and greater clinical use of the intervention.

While this breadth of PED clearly offers great opportunities to hear from patients during intervention development, it may also be daunting and confusing for intervention developers and sponsors. It may not always be clear how to collect the right PED from the right population at the right time to maximize its value. Nor how to ensure that PED is scientifically rigorous, generating well-defined, reliable, valid and interpretable insights that can be considered representative and generalizable. This book aims to help, by providing an overview of how PED can be generated in a robust manner during intervention development, and how it can be utilized to inform decision-making at the regulatory, payer, healthcare professional and patient levels. It provides expert insight and examples from across multiple disease and therapeutic areas including diabetes, rheumatology, movement disorders, oncology, central nervous system (CNS) disorders, and rare disease. The contents can be broken down into four main parts:

• Section 1: Generating Patient Experience Data
  These chapters outline different approaches for generating PED, providing methodological guidance to answer various questions.
• Section 2: Understanding Patient Experience Data
  These chapters describe approaches to understanding and contextualizing PED.
• Section 3: Using Patient Experience Data
  The book provides information on evidentiary standards and use of PED with various stakeholders.
• Section 4: Developing a Patient Experience Data Strategy
  The final chapter of the book offers a roadmap for intervention sponsors and developers interested in generating, understanding and using PED during intervention development.

In summary, PED is increasingly important in intervention development. It can help developers and sponsors make decisions about which interventions will most likely meet unmet needs, be perceived as valuable by patients, and through acceptance and adherence live up to the promise of maximizing health and well-being. It can further help developers and sponsors make investment decisions and generate data that can drive approval, placement and uptake of novel interventions. This book provides you with a comprehensive discussion of approaches to PED research in intervention development, some of which are well-established and others of which are emerging. It also provides information about the key stakeholders for PED and how to best share PED with them. I hope that it will help you ask and answer questions that are relevant for your own patient-focused intervention development, and take action in generating robust PED as relevant – whether you are seasoned in PED research or new to the field.

To read more of the IQVIA Patient Centered Solutions book, access Using Patient Experience Data to Evaluate Medical Interventions on the Amazon Kindle store. If you are interested in PCS’s training modules on this material, please contact us to secure a tailored Patient Experience Data Corporate Training Program.

References

1. United States Congress (2016). Title III, Section 3002(c) of the 21st Century Cures Act. https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf. Accessed April 12, 2023.
2. Patient Focused Medicines Development. https://patientfocusedmedicine.org/. Accessed April 12, 2023.
3. U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER). Patient Focused Drug Development. https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-centurycures-act. Accessed April 12, 2023.