Finding out what patients with Neuromuscular Disorders prefer!
PREFER is conducting a patient preference study about what kinds of treatment patients with two hereditary neuromuscular disorders prefer. Together with eleven patient organisations, we are now actively recruiting respondents for an online survey. Anyone over 18 with either myotonic dystrophy type 1, mitochondrial disorders, or caregivers of a person with one of these conditions is welcome to participate!
We are proud to announce the launch of the online survey for the PREFER Neuromuscular Disorders (NMD) Case Study! We ask patients and caregivers about how they decide between the risks and benefits of different treatments. The results will help us understand how and when to patient’s preferences should be included in decision-making: from medicine development, through approval and reimbursement decisions by authorities, bringing products to market and monitoring safety after.
There is very little research on the preferences of patients with Myotonic Dystrophy and Mitochondrial Disorders. There is no cure, and few treatments. These are progressive and debilitating rare diseases, where patient preference information can give us a better understanding of the kinds of unmet health needs patients experience.
Direct engagement of members from academia, industry and patient organisations has been the biggest drive of this case study. This also makes it a promising example of how we can conduct patient-centric research.
Rare disease affect a small portion of the population. Patients with these conditions are few and spread across the globe. To be able to reach enough patients to get the answers we need, we need international collaboration. We are very proud to say that 11 patient organisations have joined forces with the PREFER project to support the dissemination and delivery of this study. With their help, we will be able to say something about what patients with myotonic dystrophy type 1, and mitochondrial disorders think is important, and help further the research on rare diseases!
The survey is in English, which is why we are engaging with patient organisations from English speaking countries only. We thank them all for their support!
- Muscular Dystrophy Canada
- Myotonic: My cause, my cure (US)
- Myotonic Dystrophy Support Group (UK)
- Muscular Dystrophy New Zealand
- United Mitochondrial Disease Foundation (US)
- Lily foundation (UK)
- Mito Canada
- Muscular Dystrophy Association (US)
- Cure DM CIC (UK)
- Mito Foundation (Australia)
- Muscular Dystrophy UK
Are you affected by or caring for someone with Myotonic Dystrophy and Mitochondrial Disorders? You might be eligilble for our survey! Have a look at the PREFER survey on Neuromuscular disorders.
By Anna Holm & Josepine Fernow
PREFER partner MindBytes shares learnings from the project!
MindBytes’ Scientific Integration Expert, Connor Buffel, reports the company’s participation in PREFER has allowed them to build long-term relationships with new partners, which helps them broaden their reach. Thanks to the large scale of the PREFER project, they have also been able to both validate and fine-tune the tools that they brought to the project. This year MindBytes got an order from a pharmaceutical company that they credit to their membership in the PREFER team.
PREFER's patient input to decision making under evaluation by EMA and EUnetHTA
Patient preferences reflect which aspects of health treatments matter to patients individually and why patients choose a particular treatment (medicine or medical device) over others. We measure these preferences in a structured way, using particular methods. The PREFER project has developed a framework for industry, regulators and health technology assessment bodies for how to use patient preferences as input in medical product decision making. We are now asking the European Medicines Agency and EUnetHTA to assess our framework and issue a public opinion on how useful our approach is from the regulatory- and health technology assessment perspective!
Right now in cyberspace: PREFER annual meeting 2020
Today and tomorrow, the PREFER consortium meets to discuss the final stage of the project and go through initial results from our case studies. We are also looking at what the outputs from the project will be and understand the EMA/EUnetHTA qualification concept of the framework for patient preference studies.
Patient preferences in HTA and payer decision-making: challenges and opportunities
Patient preferences can inform health technology assessment (HTA) and payer decision-making. But application and integration may differ between countries. A recent PREFER publication explores how HTA and payer representatives think patient preferences can be implemented in their own countries.
15 ways to listen to the patient voice
Listening to the patient voice and understanding their benefit-risk trade-offs has the potential to improve patients’ healthcare options. Still, patient preference information is under-utilised in decision-making throughout the medical product life cycle. A recent Health Policy publication explores at which industry, health technology assessment (HTA) and regulatory decision points could use patient preference information and become part of the decision making process, allowing patient’s voices to be heard.
Preference methods to support decision making
A recent publication identifies 13 patient preference elicitation and exploration methods that are can support medical product decision making. The study has characterised and appraised different methods. The result is a comprehensive overview based on empirical evidence of the usefulness of different methods for decisions at different stages of the medical product life cycle.
Testing preference elicitation methods in clinical case studies
Our researchers will test different methods for preference elicitation in clinical case studies. We will evaluate what patients think is relevant about their disease and its impact. We will look at which treatment options they prefer and their willingness to accept trade-offs between benefits and risks of their treatment. These methods will be evaluated at different decision points in the drug development process.
Patients’ and physicians’ preferences don’t always align
Despite having a joint objective, patients' and physicians' preferences for treatment options sometimes differ. Patients are more inclined to want acute symptoms reduced, whereas physicians are looking for treatment options that offer more long-term disease management. A recent publication from Novartis examines physicians’ preferences for treating patients with liver disease, more specifically non-alcoholic steatohepatitis.
Patient input for more valuable patient preferences
Patients want to be involved in research about them. In a paper recently published in Frontiers in Medicine, PREFER researchers explore how patients want to be involved in patient preference studies, how they want it to work in practice and where they think their contribution will add most value to a patient preference study.
PREFER in 2020: Looking forward to results from clinical patient preference studies
To be able to recommend when and how to involve patient preferences in medical product decision making, we need to do patient preference research. This year, the PREFER project has launched three core clinical patient preference case studies in three disease areas: Lung cancer, rheumatoid arthritis and neuromuscular disorders. In addition, both academic and industry partners have added studies to the PREFER portfolio that will help us cover different disease areas, methods and research questions.
Lessons learned from the joint IAHPR-PREFER symposium
Thought leaders and experts discussed ways of facing the growing demand for patient preference information at the “Patient preferences in the medical product life cycle” symposium in Basel this summer. An event organised jointly by PREFER and the International Academy of Health Preference Research (IAHPR). In a recent commentary, panellists report on key messages and lessons learned from this event.
Patient preferences in dry eye disease
This month, two papers on patient preferences in dry eye disease were published in BMJ Open Ophthalmology. The first paper presents the consolidated data and methodology. The second paper examines country differences and preferences of patients who are affected to different degrees.
Valuable patient preferences from stakeholder collaboration
Stakeholder collaboration increases the value of patient preference studies, and the acceptance of their results. A paper recently published in Frontiers in Pharmacology shows that this is a key element of successful patient preference studies.
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 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.
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!
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!
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!
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.
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!
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.