PREFER stakeholder feedback session at DIA Europe


PREFER organised a lively and interactive discussion with stakeholders at the DIA Europe meeting in Basel this April. Patient representatives, participants from industry, academia, HTA and regulators appreciated the lively and interactive discussions.

Eline van Overbeeke, PhD student
Eline van Overbeeke reports from DIA Europe

The session allowed people outside PREFER to express their thoughts and needs. We received positive, technical, constructive, and critical feedback that will help us decide how to conduct preference studies, inform us on how results can be used and how our recommendations should be developed. We look forward to returning to this forum to present how our work develops!

Until now, patient preference studies have not been given a high priority by top management in industry. This prompted one participant to ask whether PREFER is trying to structure something that does not exist. Several people pointed to the need to build trust in preference methods before this kind of studies can be integrated in drug development and evaluation.

We received some critical questions and concerns related to how patient preference studies are going to be conducted in PREFER. A patient representative asked how PREFER intends to interact with patients. This led to a question on how PREFER and the recently launched IMI project PARADIGM complement each other. Other questions raised included: How do we assure that we really measure what is relevant to patients? How do we make sure that the “right” patients are recruited? And that all kinds of patients are represented? Some of these questions are currently being investigated by PREFER, while others still need to be addressed. On the question of ensuring that what is measured in the studies is relevant to patients, PREFER representatives stated that it is very important to first get an understanding of the patients’ needs by conducting qualitative studies (e.g. interviews) before moving on to quantitative studies. Following the topic on what patients to recruit, a question was raised on when to conduct preference studies, and whether to include them within clinical trials where patient selection criteria might be very narrowly defined.

Regulators highlighted that there might be a difference in preferences, also known as preference heterogeneity, between patients who have experienced treatments and those who have not and that this should be taken into account in defining the patient sample. However, not all patient representatives agreed with this statement. Besides investigating preference heterogeneity among patients, participants also pointed to the need to compare patient preferences to what caregivers and physicians think, as these preferences are often very different.

The importance of communicating with patients was stressed and a discussion took place around the current lack of educational tools for patients, which, if developed, could help them understand the context of the study and make informed choices. It can be difficult for patients to choose between several different treatment options and it is important that surveys are kept short, as people get tired, which is also known as respondent fatigue. An HTA representative was concerned about how preferences important for HTA as well as those important for regulators could be captured in one study, without having too many questions that result in respondent fatigue.

Most participants agreed it is important to look at preference methods in the context of when they are used, meaning that the criteria for selecting methods would be different, depending on when they are conducted during in the product life cycle. This means that the methods should be linked to the actual decisions that are being made about the product. Ideally, methods should also be adapted to make sure patients are educated in a fitting way.

On the use of results from patient preference studies, a question was asked on how we can prepare for the unexpected. For example, What do we do if patients turn out to be less willing to take risks (what is known as risk-averse) than we might think? In addition, there were concerns among attendees on how to translate or generalise results from the study patients answering the preference questions to the larger group of patients. One attendee also suggested that measuring patient preferences via digital tools might be useful to inform future assessments in cases where medical products are granted ‘early access’ through conditional marketing authorization or early market access.

Participants also wanted to know how the PREFER recommendations could be used and whether they will provide guidance on how to compare different patient populations with each other. Participants and PREFER representatives all agreed that decision-making is a key concept in defining the scope of PREFER, and should be considered in a broad context and should not only cover regulatory decision making. Regulators recognized the efforts of PREFER in already reducing the possible methods to twelve and mentioned that the European Medicines Agency (EMA) scientific advice working party (SAWP) is ready and waiting to look at any selected preference methods PREFER wishes to submit for an EMA method qualification procedure. This would enable formal assessment by the SAWP of the acceptabililty of a specific method for use in assessing patient preferences. One regulator stated that the broader (across several purposes and / or disease areas for example) the submitted proposal is, the more challenging the assessment will be for the EMA. The submitted method should be fit for a specific purpose as outlined in the EMA qualification guideline.

In summary, much was learned and taken away for incorporation into PREFER activities.  Moving forward, PREFER will work proactively with stakeholders to keep them up-to-date with the project’s progress.

By Eline van Overbeeke

At the workshop, PREFER was represented by Conny Berlin, Juhaeri Juhaeri, Jürgen Kübler, Susan Bhatti, Sheila Dickinson, Isabelle Stoeckert, Nigel Cook, Mireille Muller and Eline van Overbeeke

More patient voices in PREFER