Where is the patient’s preference?

Published by Søren Esben Hansen, December 9, 2020

On October the 29, 2020 I participated in a webinar as a part of EMA’s 25 Anniversary Symposium “New Approaches in Patient-focused Cancer Drug Development”. As the patient advocate Jan Geissler (Patvocates) claimed in his presentation, “a patient preference framework needs to be integrated into the early stages of drug development and onwards. Later in the drug’s life cycle, the approval bodies can only say yes or no to specific drugs.” I.e. this documentation cannot be acquired, if the RCT (Randomized Clinical Trial) is not designed to account for it.

A discussion of patient preference evidence as a trending issue

His contribution to the panel discussion reminded me of the opening scene to Monty Python’s movie “The Meaning of Life” (1983). Two doctors and two nurses get ready to assist a childbirth in the hospital’s delivery room. They find that something is missing and call for assistance of people and machines but still sense that something is not right. The patient! Where is the patient? Finally, the mother to-be is found behind a machine and brought into labor on the hospital bed. No questions asked or preferences identified.

The winding road to introducing patient preferences into the generation of evidence

The reason I bring up British satire from the past century, is not to imply that the patient is seen a passive stakeholder in the Nordic healthcare systems. Today there are many examples of patient-engagement, patient-involvement and patient-centricity on the part of patient organizations, health authorities, hospitals, healthcare professionals and pharmaceutical companies. Often in collaboration.

This patient-involvement most often takes place in the post-launch environment. In contrast, the patient preferences are rarely at the heart of drug development and trials in the pharmaceutical industry.

There are many good reasons why this has been the case. Patient priorities and preferences are subjective parameters that traditionally have not fitted into the doubled-blinded and controlled trials with evidence and ideals of replicability. However, the current trends around patient preference evidence does not challenge those ideals. Instead, they offer a supplement to traditional endpoints in building evidence and documentation for the outcome of medicines.

International approval bodies are paving the way

The ability to standardize and use patient preference evidence may still be early days. However, in international approval bodies (so-called HTA boards), the idea has circled for quite some time. Since 2016, the FDA (U.S. Food and Drug administration) has advocated for the value of patient preference documentation and published guidelines on the use of such data in their approval processes. This signals a paradigm shift in drug development by calling for patient preference evidence when approving medicines.

EMA (European Medicines Agency) and NICE (National Institute for Health and Care Excellence in the UK) are also requesting patient preference evidence but have not yet published specific guidelines.

In the whitepaper “The Value of Patient Preference – How Nordic Pharma Affiliates Can Utilize Patient Preference” (2020) [written by Amanda G. Hertz, Arun Micheelsen and myself, ed.] we document that the number of published patient preference studies were on the rise even before 2016.. We point to the fact that pharma HQs must begin to generate such evidence, and thus offer our recommendations on how different roles and capabilities in pharmaceutical companies can prepare for and utilize patient preference evidence, in market access processes. Also, we argue that the commercial organizations which are best at activating these resources will gain a competitive advantage.

You can access the whitepaper here.

All patients are different

The paradigm of patient preference rests on the idea that all patients have different needs. This is even true for patients with the same disease and similar demographics. Different disease stages, medical histories and social and psychological resources can result in quite different scenarios, needs and reactions to a treatment plan. In that light, it is particularly useful to evaluate patients’ perceived risk-benefit profiles when medical treatments are being designed and offered via preference eliciting methods.

In the developmental phase, evidence can be generated and documented according to patients’ self-reported preferences, choices and needs relative to a specific drug’s outcomes and safety profile. This means that a relevant patient population is asked to give input to a ranking exercise; e.g. via “discrete choice” models where key trade-offs are assessed and weighted in the light of individual importance. This helps to understand the preferences of different sub-populations in the trade-off between e.g. toxicity profiles, efficacy, drug administration and length of progression free survival.

These priorities can then be compared to how the same parameters are ranked by physicians, nurses and payer. The output of such a gap analysis is to identify possible overlapping interests and ambitions as well as where they deviate for patients and healthcare professional (HCP) groups.

Insights on everyday life of patients can inspire new approaches

My personal experiences with patient dialogue around needs and experiences lie both in the pre-launch and post-launch domains and has been conducted in collaboration with commercial roles in pharmaceutical companies such as market research, marketing and sales.

This work has typically been centered around a mapping of the “patient journey” and identification of pain points and possibilities when going from a pre-diagnosis stage to where patients are today. It establishes an everyday context around the course of therapy and helps to bring forward miscommunication, practical specifications for treatment and devices, unmet needs and aspirations on the part of the patient and relatives. This helps to uncover possible opportunities that can be turned into actions in a brand plan. In this way, a pharmaceutical company can help facilitate dialogue between patient life and the clinical world of hospitals, private practices and general practice.

In my years as a market researcher, I have interviewed more than 200 patients within a range of indications in the cardio-vascular space, virology, oncology, neurology and dermatology. In a recent piece of work, patient experience helped a pharmaceutical company mediate between stakeholders’ perspectives: To a group of cancer patients, it was more important to get information about the reduced risk of cancer recurrence in a specific treatment than prolonged overall survival. Prior to the research study, overall survival had been a key message from HCPs’ communication of the treatment plan to patients. This information on patient perspective proved to be useful for HCPs in their dialogue with the patients.

Early days but time to get started

If you work in the pharmaceutical industry, you may think, it is still too early to invest in implementation of patient preference evidence. You may even think it is a passing trend. However, it is my assumption, that pharma HQs may be running a little too late, if they have not yet made long-term plans to accommodate the paradigm shift seen in approval bodies such as the FDA.

Value-based healthcare systems like the Nordic are required to both deliver and document added value to patients. This places the patients’ experiences and preferences at the heart of public healthcare. In Nordic HTA boards, patient organizations represent the interests of patients and in this work, they need tools to measure and monitor the value of treatment and therapy. Patient preference evidence may just be such a measuring tool, since it works by the standards of international HTA boards and helps to systematize patient priorities. In turn, it has the potential of adding competitive edge to pharmaceutical companies that can deliver patient preference evidence in their drug development and market activation.

published by

SØREN ESBEN HANSEN


Senior Advisor

seh@dlimi.com

+45 20 570 670