Giving patients a voice in drug development
The patient perspective is important in all medical research, and particularly in drug development. This month, a public private research initiative called PREFER, is launched to assess when and how patient preferences on benefits and risks should be incorporated in decisions on medicinal products.
Drugs are developed for patients and there is an emerging consensus that they should be involved in determination of benefit-risk considerations during the life cycle of medicinal products. The patient voice is becoming increasingly important, not only for the companies that develop new therapies, but also for the authorities that assess, regulate and decide which drugs are effective, well tolerated and cost-effective for patients and the community.
Industry, regulatory authorities, health technology assessment bodies, reimbursement agencies and patient organisations are in agreement regarding the high value of patient preferences. However, there is little guidance on conducting and using such studies in pharmaceutical industry, regulatory and reimbursement decision environment. PREFER will provide a set of systematic methodologies and recommendations to assess, engage and include patient perspectives during the development, approval, and post-approval of new therapies.
PREFER is a five year project funded by the Innovative Medicines Initiative (IMI), to evaluate different preference elicitation methods and test them in a clinical setting.
“Almost all decisions taken in research, trials and approval somehow affect patients, which is why we involve patients directly as partners in the PREFER project. We have also created patient advisory groups to make sure the project gets input from a wider patient community.” says Mats G. Hansson, Professor of Biomedical Ethics at Uppsala University’s Centre for Research Ethics & Bioethics (CRB).
“We have put a lot of effort into building close collaborations between academic and industry researchers, health and technology assessment bodies, payers, regulators and patients to investigate how patient preference studies can best address everybody’s needs. We believe that PREFER will provide recommendations that can support the development of guidelines for industry, regulatory authorities and HTA bodies for all medicinal products” says Conny Berlin, Global Head of Quantitative Safety and Epidemiology at Novartis Pharma AG.
PREFER will conduct patient preference studies in three disease areas where patient and clinical research partners have expertise: cancer, rheumatoid arthritis and neuromuscular disorders. Industry partners will provide additional patient preference studies, covering disease areas from their fields of expertise.
About the public-private research collaboration
Uppsala University and Novartis lead a new public-private research project "Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER)" under the Innovative Medicines Initiative (IMI). IMI is a partnership between the European Union’s Horizon 2020 programme and the European pharmaceutical industry (represented by EFPIA, the European Federation of Pharmaceutical Industries and Associations).
Mats G. Hansson, Professor of Biomedical Ethics at Uppsala University’s Centre for Research Ethics & Bioethics (CRB) and Conny Berlin, Global Head of Quantitative Safety and Epidemiology at Novartis Pharma AG are leading this public-private research collaboration.
The academic research is supported by a 6 million € grant from IMI. The pharmaceutical industry contributes the same amount as financial or human resources. The 33 partners include: 10 academic research institutions, 16 pharmaceutical companies, 4 patient organisations, 1 Health Technology Assessment body and 2 small and medium sized enterprises, all adding their experience and perspectives to the project.
Contact and more information:
To find out more, please visit the PREFER website: www.imi-prefer.eu
Co-ordinator: Mats G. Hansson, Professor of Biomedical Ethics, Centre for Research Ethics & Bioethics (CRB), Uppsala University. E-mail: email@example.com, phone: +46 18 4716197, mobile: +46 763 412050
Project Leader: Conny Berlin, Global Head Quantitative Safety & Epidemiology Novartis. E-mail: firstname.lastname@example.org, phone: + 41 61 3248736, mobile: + 41 798451615
The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. The contents of this text reflects the PREFER project's view and not the view of IMI, the European Union or EFPIA.
Academic institutions: Uppsala University (Co-ordinator), Erasmus MC - University Medical Centre (EMC), Erasmus University Rotterdam, European Institute of Oncology, National Cancer Research Center G Paolo II, Bari, University Medical Centre Utrecht, University of Birmingham, University of Erlangen, University of Leuven, University of Newcastle upon Tyne
HTA-Bodies: Belgian Health Care Knowledge Centre (KCE)
Pharmaceutical Industry: Novartis Pharma (Project Leader), AbbVie, Actelion Pharmaceuticals, Amgen, Astellas Pharma Europe, AstraZeneca, Bayer Pharma, CSL Behring, Eli Lilly and Company, Janssen, Merck KGaA, MSD, Pfizer. Roche, Sanofi, Takeda Development Centre Europe
Patient preferences, consensus & standardised procedure
Despite growing interest, the use of patient preferences in decision-making is hindered by a lack of standardization. An article recently published in Patient captures stakeholder perspectives on patient preferences in decision-making.
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Measuring patient preferences
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PREFER: Patient Preferences in Drug Development
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PREFER Newsletter: New issue out!
A new issue of the PREFER newsletter is out! Want to receive updates from us in your inbox? Subscribe, or follow us on YouTube, LinkedIn, & Twitter.
Patient preferences in early drug development
If a pharma company aims to meet the needs of patients, patient preferences should be included at the very beginning: From phase 1/early development, through to the post-marketing phase of any medical product’s life cycle. A recent paper from Novartis, one of the IMI PREFER project partners, argues for aligning stakeholders in early drug development to ensure development plans best meet patient needs.
Talking about terminology: PREFER glossary
In a project like PREFER, a glossary helps researchers by providing a common taxonomy for our day-to-day interactions. We have agreed on definitions for a number of terms that we use in reports and publications. The definitions in the PREFER glossary are written for expert stakeholders (people in academia, industry, regulators and HTA bodies). We are also working to develop video that explains some of these terms in plain language.
Patient Preferences – the science of tomorrow
At DIA Europe 2019, Conny Berlin, Rocco Falchetto, Ardine de Wit, and Christine Radawski presented information on the importance of patient involvement, and the rapid evolvement of the science of patient preferences. The close interactions between patients, researchers and decision makers show how it is possible to establish a new approach to support medicinal development. The presentations focused on the work of the IMI PREFER project and highlighted an example where patients views could be extremely useful in regulatory decision making.
First phase complete in PREFER’s NMD case study
PREFER partners working on the neuromuscular disorders (NMD) case study, led by Newcastle University and MDUK, have now completed their qualitative data collection. Over the last couple of months, the team have carried out 14 interviews and 5 focus groups with NMD patients and caregivers across the UK.
Using psychological instruments to understand patients’ preferences
The interest in patients' preferences has grown in recent years. Regulators, HTA-bodies, payers, the pharmaceutical industry, and patient organizations are asking for patients to be involved in healthcare decision-making. Most of the attention on this topic has focused on how to elicit patients’ preferences, and not so much on why patients form specific preferences and why they take certain kinds of decisions. A new review article from PREFER maps the field.
PREFER at Patients as Partners Europe
Stakeholders from industry, patient advocacy organisations and policy makers came together at Patients as Partners Europe 2019. Couldn’t make it? Speaker presentations are now available online, and PREFER’s Berkeley Phillips was there. Find out what he has to say.
PARADIGM: Results from survey on stakeholder expectations on patient engagement
Patient engagement in the medicine R & D process is largely underutilized, despite the benefits of co-creation in delivering health solutions that meet patients’ defined needs and improve health outcomes. The PARADIGM project have surveyed stakeholders' expectations, needs and aspirations when it comes to patient engagement, and results are now available!
First patient interviewed in PREFER neuromuscular disorder case study
February 21 marks an important milestone for the PREFER project: The first patient interview in our neuromuscular disorders case study! This is a clinical patient preference study asking patients and caregivers what they think about different treatments.
First ever scientific advice for patient preference study from British HTA authority
The British National Institute for Health and Care Excellence (NICE) recently provided its first piece of advice on a patient preference study design. The advice was for Novartis, who are planning a patient preference study for people who are suffering from chronic obstructive pulmonary disease (COPD).
PREFER case studies at DIA Europe 2019
This year, PREFER is present at the DIA Europe meeting to discuss patient preferences and present our case studies. On February 6, PREFER chairs a session on patient preferences. The following day, Ardine de Wit, academic lead for PREFER's clinical patient preference case studies, will present our study portfolio in a session on patient involvement in clinical research. Don't miss us it if you are there!
Giving patients with Muscular Dystrophy a voice in drug development
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Interested in patient engagement? Subscribe to the PARADIGM newsletter!
Patient engagement is becoming increasingly important for the pharmaceutical industry, health technology assessment bodies and regulators. PREFER has signed a memorandum of understanding with the PARADIGM project (Patients Active in Research and Dialogues for an Improved Generation of Medicines). We encourage everyone who is interested in patient engagement to sign up to the PARADIGM newsletter!
Asking patients what they prefer
Decision makers need to understand how patients themselves value the risks and the benefits associated with the treatments of their disease. Karin Schölin Bywall's PhD project has received feedback from the PREFER project. To prepare her preference study, she decided to approach the patient group immediately.
Season's greetings from the PREFER consortium!
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Including patients in decisions about drugs
Companies and authorities sometimes involve individual patients and patient representatives in their decisions. By using preference studies, we can find out what larger groups of patients think about drugs. And how they weigh the good and the bad against each other. But what do patients think about being included in these decisions? In her PhD project, Karin Schölin Bywall, interviewed Swedish patients with Rheumatoid Arthritis to find out what they think about patient involvement in decisions taken by the authorities that approve drugs.
PREFER: First newsletter issued!
Want to receive updates from us in your inbox? Today, PREFER sent out our first external newsletter!
PREFER: Rheumatoid Arthritis Case Study
Rheumatoid arthritis (RA) is an autoimmune disease that causes painful joint inflammation. If it is not controlled, the inflammation can lead to deformity and loss of function. RA often runs in families. One of PREFER’s clinical patient preference case studies will focus on people at risk of developing RA in the future.
Patient preferences in HTA: ISPOR Europe 2018 Issue Panel
The PREFER project was well represented at the latest ISPOR Europe Congress in Barcelona on 10-14 November, 2018. We presented four posters with results from the project. And organised one Issue Panel on the use of patient preferences in Health Technology Assessment (HTA).
PREFER: first two years
The PREFER consortium met in Leuven on October 25-26 this year. Celebrating our achievements so far, and outlining strategies for years to come. We used the opportunity to plan the next big step: our upcoming clinical patient preference studies!
PREFER poster wins prize at IMI 10th Anniversary Scientific Symposium
PREFER was present at the Innovative Medicines Initiative’s 10th Anniversary Scientific Symposium in Brussels on October 22-23. At the meeting, Chiara Whichello, PhD student from the Erasmus University Rotterdam, won third prize for her poster on characterising and appraising patient preference exploration and elicitation methods in PREFER.
PREFER celebrates 2 years at annual meeting #PREFER2years
The PREFER annual meeting of 2018 is all about celebrating accomplishments. Hosted by KU Leuven and Janssen, the PREFER consortium meets 25-26 October to discuss our plan of action. The work we have done so far outlines what comes next: case studies, recommendations and further collaboration efforts.
Factors influencing the value of patient preference studies
Finding out what patients prefer is becoming increasingly important in Industry, HTA and regulatory decision-making. The PREFER project has looked at what the literature can contribute to our understanding of when and how to ask patients what they prefer.
PREFER: Open science, open access
The PREFER project is committed to open science. We have promised our funders, the Innovative Medicines Initiative, to ensure all our publications are available to those who do not have, or cannot afford, subscritpions to scientific journals. This is made possible through postprint publishing in the Uppsala University library's DiVA database, also known as green open access.
PARADIGM and PREFER connect to make patient engagement in medicines R&D a greater reality
Today, the two Innovative Medicines Initiative (IMI) consortia PARADIGM and PREFER issue a joint statement on the signing of a Memorandum of Understanding. The document aims to enhance the cooperation and collaboration between the two projects. For both projects, this represents an opportunity to avoid duplicate efforts and maximize results.
From the lab to you: How do medicines get approved?
Want to know how medicines move get approved in Europe? Watch this video from the European Medicines Agency (EMA) to find out!
PREFER and neuromuscular disorders
Cecilia Jimenez-Moreno, Research Physiotherapist and PhD leads the upcoming PREFER patient preference case study on neuromuscular disorders (NMD). She shares the task with Dr. Grainne Gorman. Both are involved in NMD research at Newcastle University
Whose preferences are we collecting?
PREFER looks at how to collect & share patient preferences and when to include them in medical product decision-making. Want to learn more about who we are asking? Watch our new video clip to find out who will tell us what patients prefer.
Patient engagement: what comes next?
In a recent guest column in Clinical Leader, Barbara Lopez Kunz, global chief executive of DIA writes about what is next in patient engagement. She lists PREFER among organisations that drive research and policy activities for guidance on patient-centricity in benefit-risk assessment and systematic incorporation of data on patient perspectives in product approval applications.
FDA issues draft guidance on Patient-Focused Drug Development
FDA has just issued a new draft guidance to inform patients and medical product developers on how to gather information on patients’ views and needs in a way that provides meaningful data.
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.
What Parkinson's patients want from treatment
Another successful public-private-patient collaboration provides new insight into what patients want from treatment. Listen to the PREFER webinar where patients in this Parkinson’s disease Project identified a new benefit they would trading off against a treatment’s burden.
Identifying methods and criteria for testing
Well-informed patients provide the most useful preference information. According to an article recently published in Value & Outcomes Spotlight, this raises questions about risk communication, educating patients and psychological variables that affect patients’ preferences.
Planning for patient preference studies begins
Right now, the PREFER consortium and external experts are meeting in Berlin. For two days, we will discuss and agree on methods and study design for patient preference ‘case’ studies in Lung Cancer, Rheumatoid Arthritis and Neuromuscular Disorders. If you are interested to find out more, follow us on twitter #IMIPreferBerlin18.
PREFER: the first year
Wow! This is the singular word that comes to mind after reading the first PREFER report to our funders, the Innovative Medicines Initiative. Seeing all the completed and ongoing work shows what public and private partners can achieve through hard work and collaborative efforts.
Watch PREFER on YouTube
Want to learn more about patient preferences and the PREFER project? Watch our videos on YouTube. Remember to like and share our videos, subscribe to our channel and click the little bell icon on Youtube to get post notifications when we upload new content.
Follow PREFER on twitter!
PREFER is now on twitter. Follow @IMI_PREFER to stay updated and receive invitations to webinars.
PREFER Annual meeting 2018
The next annual meeting of the PREFER project will be convened on October 25-26th 2018. We look forward presenting and discussing the project’s progress with members of the consortium and invited stakeholders.
Preference research from the patient perspective
Diagnosed with Rheumatoid Arthritis (RA) ten years ago, An de Groef describes her journey from diagnosis, through treatment, to remission. Sharing her story on how RA affects her life, and outlook on the choices available to her at different stages of the disease.
PREFER at ISPOR 2017
This week, four PREFER PhD students are presenting at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 20th Annual European Congress in Glasgow.
Data management in PREFER
Over the next five years, PREFER will gather large amounts of data from patients and other stakeholders. To ensure we handle data appropriately, PREFER has developed an initial data management plan that will evolve with time. Here, the PREFER data management team explains the thoughts behind it.
PREFER annual meeting 2017
The annual meeting of the PREFER project will be convened on October 26-27th 2017. We look forward presenting and discussing the project’s progress with members of the consortium and invited stakeholders.
Patient preferences in healthcare decision-making
There is a growing movement in the pharmaceutical industry, putting focus on the patient experience. The PREFER project is part of this movement. In a recent issue of CenterWatch Weekly, Bennett Levitan explains patient preference research and how PREFER intends to play a role.
PREFER PhD project on rheumatoid arthritis
In March this year, Karin Schölin Bywall started her PhD studies at Uppsala University. Her project is part of PREFER. It will look at how rheumatoid arthritis patient preferences can add value when regulators make decisions on drug development.
Editorial in Patient: Giving patients’ preferences a voice in medical treatment life cycle
In the latest issue of The Patient: Patient-Centered Outcomes Research, PREFER researchers write about the importance of this project. The list of authors includes PREFER members from academic institutions and industry, patient organisations and HTA bodies. The list of authors also includes a representative from EMA.