Including the patient perspective
PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products.
Over the next five years, we will run patient preference studies in both academic and industry settings. Our experience will provide a better understanding of what will be a recommended best-practice approach to patient-preference studies. We will also show how patient preference studies can give valuable information to support decision making for regulators and HTA bodies.
PREFER is divided into four 'work packages'. The methodology work package looks at the concerns stakeholders have around the use of patient-preference studies. Based on what they find, they will make recommendations about what methodologies to use in case studies that the case study work package will design and carry out. After evaluating the case studies, Finally, the recommendations work package will take over and draft recommendations based on the work. The management work package will make sure this work is done on time.
Public private partnership
PREFER is a public-private collaborative research project under the Innovative Medicines Initiative (IMI). This means that in addition to academia and the biopharmaceutical companies, PREFER also includes patient organisations, Health Technology Assessment bodies and small and medium-sized enterprises.
IMI aims to boost biopharmaceutical innovation in Europe and to speed up the development of better and safer medicines for patients. Through its unique and innovative funding scheme, IMI supports research projects in the areas of safety and efficacy, knowledge management and education and training. This project is a joint undertaking between the European Union’s Horizon 2020 programme and the European Federation of Pharmaceutical Industries and Associations European Federation of Pharmaceutical Industries and Associations (EFPIA) with a 6 million Euro contribution from each side. PREFER is led by a co-ordinator from Uppsala University, and a project leader from Novartis. The project has been structured with equal leadership and participation from academia and industry in all areas of the project, underlining the collaborative and inclusive approach that guides our work.
Assessing methods
We start our work with a literature review, interviews and focus group meetings with patient organisations, physicians, regulatory authorities, health technology assessment bodies, industry experts and academics on their key concerns, needs, expectations and desires on the assessment and use of patient preferences.
Through clinical case studies
We are running three core PREFER patient preference studies looking at lung cancer, rheumatoid arthritis and neuromuscular disorders. Both academic and industry partners have added studies to the PREFER portfolio that will help us cover different disease areas, methods and research questions.
Developing recommendations
By the middle of 2019, we will have drafted recommendations and asked stakeholder advisory groups to test them to see how they work for other disease areas and decision points. By the middle of 2021 we expect to have a refined draft. Final recommendations will be presented in the autumn of 2021.
Patients
Advisory group on board
PREFER has four patient partners. ECPC coordinates the work together with MDUK, EPF and IAPO.
HTA and payers
Advisory group on board
This group is coordinated by the Belgian Health Care Knowledge Centre (KCE). Other members are CADTH, NIHDI, G-BA, LBI and EUnetHTA.
Regulators
Advisory group on board
PREFER's regulatory stakeholder advisory group has representatives from EMA, CHMP, SAWP, COMP and FDA through CBER.