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.
An de Groef, postdoctoral researcher at KU Leuven, gave an inspiring keynote presentation at the PREFER annual meeting in October 2017. As both an academic researcher and patient, she offered a unique perspective on what is important in preference research from the patient point of view. On behalf of patients, she said, “We want to tell our story. We want someone to listen to us. I had good medical outcomes, but I had some other issues that were hard to talk about.” She pointed out that it is important to remember that patients are overwhelmed after receiving diagnosis. They receive lots of information and ideas about what their diagnosis entails, but do not yet know what it will mean for them personally.
An de Groef received her own RA diagnosis at the age of 18. She described the differences between her own thoughts and reactions then compared to 10 years later, now in remission. She emphasised the fact that patients’ preferences will vary depending on the stage of the disease. A person with a recent diagnosis may not have the same preferences as someone in a more chronic and stable condition. In addition, patients in stable conditions might have a different view of what is normal in terms of fatigue or pain than someone with a recent diagnosis. This is something researchers need to remember when they design a preference study.
Another important point to consider is how and where you recruit patients, whether in the clinic, or through patient organisations. Moreover, depending on where you find the patients, and where they are in their own ‘disease journey’ and personal maturity, researchers also need to think about how they collect information on preferences. Whether individual interviews or focus groups are the better choice depends on whether patients are comfortable sharing.
An de Groef is a physiotherapist and postdoctoral researcher at KU Leuven, Belgium. In the early years of her RA diagnosis, An de Groef was a patient research partner in a study on the outcomes patients with early stages of RA prefer in terms of treatment. This was also the start of her own academic research career.
While being a research partner gave her a voice as a patient, it also created some confusion. Research is unknown territory for most patients, and An de Groef suggested patient partners should have one contact person that they trust. Based on her own experience, An de Groef pointed out that it could be difficult to differentiate roles when you sometimes meet your doctor as patient, and other times as a research partner. She suggested clinical researchers should work with their research partner to make sure roles are clear. Perhaps brief job descriptions would help clarify roles and responsibilities, keeping expectations realistic on both sides.
Respect is also key to a successful collaboration between patients as research partners and clinical researchers. Patients might worry whether their contribution is valuable, and are sometimes are surprised when told they actually offer valuable input.
By Josepine Fernow
About patient input in the PREFER project
The PREFER consortium includes four patient organisation: the European Cancer Patients Coalition (ECPC), Muscular Dystrophy UK (MDUK), European Patients Forum (EPF), and International Alliance of Patients’ Organizations (IAPO). Together they form the PREFER Patient Advisory Group, coordinated by ECPC.
Patient Advisory Group members are directly involved in activities within all work packages of PREFER, sharing common expectations and ensuring that the methodologies identified correspond to the patient views, experiences and preferences. We also have a number of individual patients with rheumatoid arthritis on board through the clinical research partners at the University of Birmingham.
PARADIGM reports from PREFER annual meeting: Understanding patient preferences – the next frontier in PE
The PARADIGM project were present at the PREFER annual meeting. Want to know what EPF’s Camilla Habre (Project Officer) and Mathieu Boudes (PARADIGM Coordinator) have to say about PREFER? Here is a short report (originally posted on the PARADIGM blog onm October 31, 2019).
Right now in Basel: PREFER annual meeting 2019
Today and tomorrow, the PREFER consortium is meeting to discuss the second half of the project and how our patient preference case studies will support the project. We will also talk about how regulatory and HTA qualification and scientific advice for a framework for patient preference studies can help us have impact.
Want a summary of what we have published?
PREFER looks at how and when it is best to perform and include patient preferences in decision making during the medical product life cycle. We include patient stakeholders at every level of the project. The end-result will be recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies. Want to know what we have published so far? In this pdf booklet, you will find a list of our publications with abstracts and links to full text.
Critical need for better methodological understanding of preference methods
Before issuing recommendations on when and how to use patient preference studies for decision-making along the medical product life cycle, learning more about the opportunities and challenges faced by stakeholders is key. An article recently published in BMC Medical Informatics and Decision Making has done just this, and offers comprehensive lists of roles, reasons, concerns and requirements for patient preference studies.
Live webinar on patient preferences in European HTA
PREFER’s Nigel Cook is one of the featured presenters at this live webinar on patient preferences in health technology assessment (HTA) in Europe, the recent advances and future potential.
Optimizing patient preference studies
A recent publication from PREFER lists 18 factors and situations that should be considered when designing and conducting patient preference studies. To obtain valuable patient preference information for use in decision-making, knowing what challenges to look out for, and how to overcome them, is imperative. The paper was published in Frontiers in Pharmacology this week.
Positive feedback on PREFER mid-term review!
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.
Joint IAHPR-PREFER workshop and symposium
On Saturday 13 July 2019, the Academy of Health Preference Research (IAPHR) and PREFER will host a joint workshop on good research practices for health preference studies We will describe the basics for how to conduct a health preference study focusing on trade-offs between risks and benefits. The workshop material will build on the textbook that is currently under development by IAHPR members, incorporating the experiences of scientists working with PREFER.
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.
Celebrating Success: RA Case Study First Participants In!
The PREFER rheumatoid arthritis case study team are celebrating the successful start of recruitment and data collection. The first focus group to inform the development of a treatment choice survey was conducted on Wednesday 29th May 2019 with seven members of the public in Birmingham, UK. A second focus group is scheduled to follow a week later, followed by more focus groups with relatives of patients with rheumatoid arthritis.
Measuring patient preferences
Both industry representatives, academics, regulatory and patient groups suffer from a lack of knowledge of the range of methods used in patient preference studies. A recent publication provides insight into the many methods used.
PREFER: Patient Preferences in Drug Development
Mitochondrial patients and caregivers have been integral in helping guide the development of potential treatments for mitochondrial disease as part of the first phase of the PREFER study. But, what does this mean and what happens next? Dr Cecilia Jimenez-Moreno, the PREFER clinical case study lead based in the Wellcome Centre for Mitochondrial Research at Newcastle University, tells us more.
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
PREFER partner Muscular Dystrophy UK is commited to giving patients a voice in drug development. In PREFER, they work with pharmaceutical companies, academics, patients, healthy technology assessment bodies and regulatory authorities to ensure the rare disease patient perspective is present in the recommendations coming out of the project.
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!
Season's greetings from the PREFER consortium! And best wishes for 2019. We look forward to new challenges in the coming years. 32 partners representing academic research institutions, patient organisations, HTA bodies and the pharmaceutical industry, working together to include the patient perspective in medical product decision-making.
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.