Positive feedback on PREFER mid-term review!

2019-09-10

This summer, PREFER has gone through a thorough mid-term review by the Innovative Medicines Initiative (IMI). We are happy to report that independent evaluators concluded PREFER is an ambitious project addressing relevant clinical and methodological research questions, that aims to show how patient preference studies can support decision-making. In the report, IMI states that the work we have provided so far is of high scientific quality.

According to the review report, the first results are promising. Although the real impact of the project will not be seen until after our empirical clinical patient preference case studies have been completed, IMI writes that the project is well designed and organised, and has all the necessary components to achieve the impact we expect. A project like PREFER is built on input from many different stakeholders, and the review describes our interactions with them as regular and efficient.

Mats G. Hansson, PREFER coordinator
Mats G. Hansson, PREFER coordinator

We are pleased with the overall positive review and look forward to using the input from our evaluators to build a better, stronger, and more impactful project, says Mats G. Hansson, Coordinator of PREFER and Professor of Biomedical Ethics at Uppsala University.  

The work in PREFER is organised in steps or work packages. The package on methodology has been completed, and we are now moving to a phase where we conduct clinical patient preference case studies, which will be followed by the development of recommendations from the entire project. PREFER also received praise for the strategy we developed for the project and the way we carry out the day-to-day management, communication and stakeholder coordination. IMI took note of the special task force we set up to develop a joint qualification from the European Medicines Agency (EMA) and the EUnetHTA network. Two qualification requests are being made - one for a general, systematic approach to collecting patient preferences; a second for one specific study design, or method, used to collect patient preferences.  IMI called this task force a “remarkable contribution to the project”

Conny Berlin, PREFER project leader
Conny Berlin, PREFER project leader

PREFER is a public-private partnership and we have worked hard to ensure that our different perspectives create synergies. I am very happy to see that the reviewers noted the collaborative, scientific and productive spirit of PREFER, says Conny Berlin, Project Leader for PREFER and Global Head of Quantitative Safety & Epidemiology at Novartis.

According to IMI, our methodology package delivered a precise and systematic approach to support the methodological foundations of the project. IMI noted that there are some questions worthy of extra attention, for example how to identify bias and the source of that bias. This is a challenge for all projects working with patients. On the one hand, we need input from patients who understand the questions we ask. On the other hand, patients who are more educated about their disease might express different preferences than others. Moreover, in a large European project, we also need to consider the fact that cultural differences could influence the input we receive from stakeholders. We also face the challenge of making sure we do not over-simplify a complex socio-psychological process, and IMI recommended that we present our strategy to handle this potential risk.

We are now actively collecting data and in the middle of carrying out our main patient preference studies. We are involving patients with rheumatoid arthritis in Germany and the UK, lung cancer patients in Italy and Belgium, and patients with neuromuscular disorders in the UK. Alongside these studies, PhD students and postdocs in the project are also conducting patient preference studies that will give input to PREFER, and some of our partners from the pharmaceutical industry are conducting studies as well. We are also collecting information from previous preference studies that can give input to the project and have decided to prolong this work to ensure we have as much data as possible to support our recommendations.

Even though we haven’t collected all the data yet, the work to develop recommendations has already started. These recommendations provide the main impact to stakeholders from the project.  The task force for an EMA and EUnetHTA qualification of a framework and one of the methods used in PREFER is aimed to ensure the project has an impact among industry, regulators, health technology assessment bodies, payers and in the end also patients. IMI also acknowledged that our work to develop training modules will contribute to impact from the project. We still have work to do to ensure we manage to engage other stakeholders, like general practitioners and other primary health care providers (nurses, physiotherapists and pharmacists), but overall, the review report described the outputs so far as impressive.

We are grateful to IMI and the group of reviewers to look at all our reports in the first half of the project. It is thousands of pages and we appreciate the effort made. We are happy for the very positive review and very grateful for the critical read and recommendations for us going forward. The work will very much improve, thanks to the efforts of the IMI review team!

By Josepine Fernow

Assessing PREFER methodology

The European Medicines Agency (EMA) is a European regulatory agency that issues qualification opinions of novel methodologies for medicine development. The opinion is issued by a group of experts from the committee for Medicinal Products for Human Use (CHMP) and will be posted publically on the EMA website. 

About EMA qualification

The European Network for Health Technology Assessment (EUnetHTA) is a European network that creates, facilitates and promotes sustainable Health Technology Assessment (HTA) cooperation in Europe. They have a similar process where they conduct assessments, which are posted publically on the EUnetHTA website.

About EUnetHTA assessments