Exploring Person-Centred Value-Based Health Care: Insights from Pharmaceutical Industry Leaders

As we gear up for the 2024 Australasian Forum on Person-Centred Value-Based Health Care, we are excited to present a three-part interview series that delves into the critical importance of focusing on people's values, goals, and preferences within the pharmaceutical industry. This series features candid conversations with three distinguished leaders who bring diverse perspectives and rich experiences to the table:

Adam Heathfield (AH), Senior Director at the Healthcare Innovation Center, Pfizer? ?

Cécile Huyghe Garassus (CHG), International Developed Markets Innovative Access Director, Pfizer ?

Elizabeth de Somer (LdS), CEO of Medicines Australia. ?

These interviews underscore the collective commitment within the pharmaceutical industry to advance Person-Centred Value-Based Health Care. By focusing on what truly matters to people, we can pave the way for a more responsive, effective, and humane health care system.? Stay tuned for this enlightening series and join us at the 2024 Australasian Forum to continue the conversation on transforming health care through Person-Centred Value-Based Health Care approaches.?

Interview with Adam Heathfield,?Cécile Huyghe Garassus and Elizabeth de Somer?

Could a greater understanding of people's values, goals, and preferences influence a pharmaceutical company’s priorities for research and development??

AH: Companies already consider both technical opportunities and unmet needs when prioritizing research and development efforts. While we may identify technical possibilities, it's essential to determine if there's a genuine demand for intervention in a specific disease area. This involves understanding what patients truly value and what current treatments may lack in delivering. We shouldn't solely rely on scientific knowledge but also delve into patients' broader values, life aspirations, and treatment outcome goals. This deeper understanding becomes particularly vital in later development stages to ensure that medicines genuinely deliver value to patients and align with their goals and preferences.?

Could values, goals and preferences be used to measure the effectiveness of medicines in clinical trials? ?

AH: I find the idea intriguing. Particularly in the early stages before a medicine is approved, it's important to establish clear tests for safety and efficacy. This initial focus allows us to grasp how a medicine functions and interacts more broadly. Robust endpoints are essential for designing studies that can effectively address these questions. It's also beneficial to consider specific patient populations and their goals and how these could be captured in randomised controlled trials (RCTs) for registration and in post-approval studies. Importantly, value for patients will extend beyond just the medicine itself to encompass their entire care pathway. Exploring different care packages could help patients with specific preferences achieve their goals. However, comparing diverse care packages may present challenges in terms of randomisation and control. Despite these complexities, conducting experiments including values, goals and preferences could provide valuable insights that mere speculation might not provide.?

If you could redesign the research and development (R&D) process to place greater emphasis on people’s values, goals and preferences, what changes would you make??

AH: I believe there's potential for improvement in industry’s R&D approach by incorporating diverse perspectives on outcomes and goals. This would have the potential to not only influence HTA considerations but also how physicians and health systems use our medicines. Including values, goals and preferences seems ambitious, but conducting more pilots could demonstrate the feasibility of these changes for all stakeholders, ultimately driving reforms in R&D processes.?

LdS: In Australia, there's a belief from patient organisations that incorporating the patient perspective could enhance the design of clinical trials. They emphasise the importance of understanding the disease from the patients' viewpoints. While scientific endpoints and markers are necessary, there's a call to consider what truly matters to patients when demonstrating a clinically meaningful difference or value. These aspects are sometimes overlooked because we typically approach trial design from a scientific and statistical standpoint, rather than considering the lived experiences of patients. However, there's a global shift towards a more community-centred approach, with patients advocating for more involvement in the process. This shift may require us to adapt and consider alternative methods of clinical development that include the patient perspective more prominently. What's essential is to develop methodologies that are not only acceptable for regulatory submissions but also for health economics assessments. Currently, there are outdated methods in place where quality of life measures and other vital patient endpoints are simply mapped to existing models. This practice diminishes the true value of these products. Therefore, there's a pressing need to modernize our methodologies, particularly in measuring quality of life.?

AH: There are some important examples from Duchenne muscular dystrophy where the patient community made it clear that some of the normal clinical measures used in that condition didn't fully capture what mattered to them. For many patients, maintaining upper body function was crucial for daily tasks like using a computer or communicating. This insight led to the creation of a new measure of "performance of the upper limb," which has been integrated into studies. It would be wonderful to see more examples like this in the future.?

CHG: I believe most of us are struggling with similar challenges, trying to make sense of the ongoing changes. It's not that we're unwilling to consider patient goals and preferences; it's more that we're uncertain about how to effectively integrate them. Our industry heavily relies on statistics to derive meaning from vast amounts of data, and we can't feasibly address every individual case separately. Instead, we must generate evidence of safety and efficacy that applies to broader populations. This poses a significant challenge. While there have been attempts to address these issues, such as the legislation in France two decades ago that aimed to involve patients more in medical decisions, translating these intentions into practical actions remains a major question mark.?

AH: Perhaps there's a way to deliver somewhat tailored solutions without addressing each case individually, by identifying groups of patients with similar goals and preferences, forming cohorts. We could explore which group of patients would benefit most from a particular intervention based on their specific goals or treatment context. Currently, decisions can appear quite blunt, offering only one option for a mass system, even though there are usually multiple potential solutions. It's challenging to cater to individuals with a one-size-fits-all approach, but maybe there’s a balance between that and validated approaches for particular cohorts and groups.?

Secure your?ticket for the 2024 Australasian Forum on PCVBHC

Change starts with collaboration. Join us and other leaders from across the world as we shape the future of health care. The 2024 Australasian Forum on Person-Centred Value-Based Health Care is happening in Melbourne on the 2nd and 3rd of September 2024. Don’t miss this fantastic opportunity to build a more person-centred, value-driven health care system for all.?

We look forward to welcoming you to Melbourne in September 2024!

Book your place now