A word of acknowledgement from the coordinators
PREFER has not only accomplished everything that we set out to do in the beginning. Significant contributions to the science of bringing patient perspectives to the centre of medical decision making, with methodological rigor based on close-up stake holder input. Large clinical case studies in several disease areas and in multiple countries with innovative insights from psychology and the field of educational tools. Providing detailed recommendations for anyone wanting to set up a preference study in their organization.
The PREFER Recommendations
The PREFER project has developed recommendations for how and when it is best to perform and include patient preferences in decision making during the medical product life cycle. Supporting the development of guidelines for structured patient input into decision-making for the pharmaceutical industry, regulatory authorities, health technology assessment bodies and reimbursement agencies!
The PREFER Recommendations in brief
Want to know why, when and how PREFER recommends that pharma, regulators, HTA bodies and payers assess and use patient preferences in medical product decision-making? In time for the 28 April launch, we offer a summary of the key messages from the PREFER Recommendations!
Final PREFER webinar recording online! Patient preferences in diabetes
On 7 April, we explored the results of our diabetes patient preference study in the final instalment of the PREFER webinar series. We presented the results of a diabetes case study on patients' preferences for, and the trade-offs they make, when selecting what device to use to monitor their glucose. And discussed whether outcomes differ depending on what type of preference elicitation method is used, what kind of educational tool patients are presented with, the way patients are recruited, and patient characteristics/experiences.
Webinar on patient preferences in multiple myeloma now online!
Multiple myeloma is a cancer that forms in plasma cells, a type of white blood cells. In our 1 April 2022 webinar, we presented the results of a multiple myeloma patient preference study that aimed to understand the unmet needs, treatment outcomes and attributes (side-effects, symptoms, efficacy outcomes) that are most important to multiple myeloma patients.
Webinar recording online! Patient preferences for treatment of chronic pain
Osteoarthritis and Chronic Low Back Pain are two of the most common chronic pain conditions worldwide. To quantify patients’ perspectives on this unmet need, Pfizer and Lilly conducted a patient preference study in the US and UK. In this 10 March 2022 webinar, we describe the results of the study and the insights that contribute to the final PREFER recommendations.
Curious about what methods to use when? Recorded webinar now online!
The PREFER project case studies utilised different methods were to elicit patients' preferences. In this webinar from 8 March 2022, we share what methods to use when, for the different decision-points along the medical product life-cycle, based on criteria that were found important for methods selection and compare methods based on the obtained outcomes of the case studies.
Launching the PREFER Recommendations
On April 28 2022, the PREFER project will launch a set of recommendations for how and when it is best to perform and include patient preferences in decision making during the medical product life cycle. The recommendations are the result of a five-year effort from public and private partners. Patient stakeholders have been involved at every level of the project, co-creating a set of recommendations that can support the development of guidelines for structured patient input into decision-making for the pharmaceutical industry, regulatory authorities, health technology assessment bodies and reimbursement agencies!
Informing future preference research for Rheumatoid Arthritis
Rheumatoid arthritis (RA) requires long-term treatment to prevent and control disease progression. There is an increasing interest in identifying and treating ‘at risk’ individuals in order to delay or even prevent the onset of RA. But the treatment (preventive and otherwise) comes with potential side-effects. Understanding what patients and individuals at risk of RA prefer can help facilitate patient centred healthcare strategies and shared decision-making. PREFER researchers just published a systematic review to inform future preference studies in RA.
Future areas of patient preference research identified: Recorded webinar online!
We have spent the last 5 years exploring why, when and how to assess and use patient preferences in medical product decision-making. But within the constraints of a 5-year time frame and a finite number of case studies, we were not able to explore all relevant research questions. In this webinar, recorded on 7 February 2022, we review topics of future research.
PREFER at DIA Europe 2022: Enhancing patient engagement
Patient Engagement is diverse. The PREFER session at DIA Europe 2022 will approach two different facets of patient engagement, covering both to patients’ involvement beyond study participation, as research partners in designing and returning results to participants, and the interest of patients and participants to join in and/or stay in a study.
Webinar on patients’ preferences for antithrombotic treatment now online!
PREFER partners are contributing data from their own patient preference studies to the project. One of these case studies explored patient preferences for treatment following a myocardial infarction (a heart attack) and how they value the benefits and risks of antithrombotic treatment at the acute and chronic stages of heart disease. In this webinar recording from 28 January 2022, Cathy Anna Pinto & Tommi Tervonen describe the design of the study and its main results.
Watch back our webinar on patient preferences for lung cancer treatment!
One of PREFER’s core case studies explored patient preferences for lung cancer treatments, assessing which trade-offs between benefit and risks related to treatment alternatives that patients are willing to accept or not. In the recording from our 13 January webinar, we describe the design of the study, its main results, and lesson learnt in conducting the preference study with this vulnerable population of patients.
Recorded webinar on patient preferences in Rheumatoid Arthritis now online!
Rheumatoid arthritis is a long-term condition that mainly affects the joints and causes pain, swelling, stiffness and often fatigue. It is common and affects around 1% of the population. In most cases, patients begin having symptoms between 40 to 60 years of age, but it can also begin earlier, or later in life. On 2 December 2021, we organised a webinar presenting two patient preference studies focusing on treatments for rheumatoid arthritis and its prevention. The recordings are now available on YouTube.
Separate good and bad in Case 2 Best-Worst Scaling
Case 2 Best-Worst scaling, or BWS-2 for short, is one of the most popular methods for finding out what patients prefer. This is method is used to ask patients to rank different treatment attributes from best to worst. BWS-2 is a quite new method that preference researchers are still exploring. A recent PREFER study explores how the features of BWS-2 can lead to estimation problems when including both benefits and risks in scaling exercises.
Challenges and opportunities of choice modelling in health
In his dissertation, Vikas Soekhai explores different methods used to collect patients’ preferences. There is a variety of methods available. Vikas Soekhai identified 32 and has focused particularly on two of the most popular ones in his dissertation: Discrete choice experiments and what is called Case 2 best-worst scaling. All to provide insights into how best to elicit the preferences of patients.
Web-based preference studies: Why and why not?
Covid19 has resulted in the expansion of online, web-based data collection methods. These have several important advantages over face-to-face data collection. But there are several limitations to consider. A recent PREFER publication offers a comprehensive overview of both challenges and opportunities. And suggest introducing comparisons, as well as adapting to the needs of participants.
Chronic obstructive pulmonary disease: The importance of different symptoms
Understanding how patients value different symptoms is important for the development of patient-centered therapies. One of the PREFER clinical patient preference case studies looked at how patients with chronic obstructive pulmonary disease value different symptoms. The results were presented in a webinar on October 27. Did you miss it? The recording is now available!
Launching the public consultation!
PREFER project has developed a framework with points to consider when selecting methods for industry, regulators and health technology assessment bodies for how to use patient preferences as input in medical product decision making. We have asked 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. Today, EMA published the draft opinion for public consultation! We now invite you and other stakeholders to give your input by 25 November 2021!
Curious about the PAVING study? Recorded webinar available!
Decisions about whether to approve new and emerging gene therapies are impeded by the associated uncertainties, e.g. long-term outcomes. In this complex context, the PAVING study investigated haemophilia patients’ preferences for gene therapies. Learn about how the preference study was designed, what the results were, and what the impact might be.
Lung cancer patients’ perspectives on quality of life
New treatments like immunotherapy and targeted therapy have drastically increased the number of options available to lung cancer patients and their treating physicians. But with uncertainties about their varying benefits and side-effects, there are questions to answer about their impact on patients’ health-related quality of life. PREFER researchers have investigated what patients prefer, and what matters most to them.
Missed the PREFER framework webinar? Recording now available
Incorporating patient preferences into decision-making is an important part of patient-focused drug development. The lack of a clear, practical framework for measuring patient preferences was one of stakeholders' main concerns brought up during PREFER’s initial research. On 31 August, we introduced the PREFER framework for patient preference studies, with a particular focus on points to consider for method selection and the application of preference study results to inform regulatory and HTA decision-making. The webinar was recorded, and you can now watch it in full, or the parts that interest you the most.
Patients’ preferences for gene therapy in haemophilia
For innovative treatments and treatments for rare diseases, finding a way to include the patient perspective in decision-making can be crucial. With answers from 117 people with haemophilia, PREFER researchers present their results from their clinical case study about haemophilia patients’ preferences for gene therapy. They found that patient preferences vary greatly. And that informing patients about gene therapy can facilitate acceptance.
What is a patient preference? Find out in 5 different languages!
Patients choose medical products based on preferences. But what is a patient preference? Find out in your language! More language versions are coming, but so far, we have translated our video about patient preferences to Romanian, Spanish, Catalan, Greek, and French!
Learn about PREFER in 5 different languages!
Drugs are made for patients. And patients speak different languages. We have five new language versions of our project video! Speak Spanish, Italian, Slovakian, French, or Catalan? Learn about PREFER in your language!
Curious about the PREFER research agenda? Recorded webinar available!
On 1 July, we held a webinar presenting the PREFER research agenda and how it was developed using a series of qualitative and quantitative assessments and engaging stakeholders in planning, execution, and/or evaluation of patient preference research across the medical product lifecycle. The result of these assessments was a list of methodological questions of concern to these stakeholders. These questions were then used to guide the design of cases studies in PREFER, used to inform recommendations for designing and conducting patient preference research. In the process, we also identified the methodological questions PREFER will not be able to answer.
Patient preferences for multiple myeloma treatment
Both new treatments being developed and existing treatment options available to multiple myeloma patients are associated with uncertainties. There are many unanswered questions, especially regarding the long-term efficacy and side effects of these treatments. But we don’t yet know which of the unanswered questions are most important to patients. PREFER researchers have found out what patients think matters most. They share their findings in a recent Frontiers in Medicine publication.
Missed our patient involvement webinar? Recording now available!
Patients are key in the success of patient preference studies. In our webinar on 11 June, we discussed the role of patients as partners in patient preference studies and explained the value of involving patients at the stages of design and while conducting a patient preference study as well as at the level of communication results back to patients after. Now, the recordings are available.
Psychosocial factors might hold the key to understanding preference heterogeneity
That patients’ preferences differ is not surprising. But figuring out why is one of the main challenges of integrating the patient voice in decision-making across the medical product life cycle. Some differences might be due to clinical characteristics like age and medical history, and others to psychosocial factors like health literacy or illness perception. A recent PREFER publication underlines how measuring psychosocial factors in patient preference studies can provide valuable information to decision-makers. And provides recommendations and a checklist telling us how to do it.
Understanding unmet patient needs and expectations
Learning what patients prefer, what benefits they are after, and what risks they can and cannot tolerate is particularly important in the case of rare diseases. A recent PREFER publication reveals results from a large patient preference study targeting rare disease groups through an international collaboration between patient organisations. Focusing on the preferences of patients with Neuromuscular Disorders.
6 PhD’s furthering knowledge on patient preferences
Thanks to the efforts of six PhD students, we have been able to contribute to the science of patient preference studies beyond the scope of PREFER. In addition to their tasks in the project, they have explored PREFER research questions and methodology in their own patient preference studies, contributing valuable results to both PREFER and the preference research community. Ranging from patient preferences for gene therapy, biologics, new cancer treatments, and glucose monitoring, to how simulations can support our understanding of preferences and whether educational materials and framing of the attributes in a study will have an impact on results.
Patient preferences for treatment of neuromuscular disorders
Rare diseases are complex, uncommon, serious and debilitating conditions that often come with a poor prognosis. Neuromuscular disorders are multisystem and progressive rare diseases with few treatment options available. PREFER researchers set out to explore unmet health care needs and patient treatment preferences for two of them: myotonic dystrophy type 1 and mitochondrial myopathies. They are hoping that their findings can support decision-making in the early stages of drug development.
Time to discuss PREFER results!
We are getting close to issuing recommendations for when and how patient preferences can support decision-making for industry, regulators, health technology assessors and payers. Our clinical case studies are delivering results. And we have developed a framework for patient input to decision making that is currently under evaluation by EMA and EUnetHTA and are looking forward to a public consultation process later this summer. In the meantime, we invite everyone who is interested in patient preferences and the PREFER approach to discuss our work in a series of webinars this year, and the upcoming DIA workshop in June.
Leveraging patient preference studies for development and decision making: DIA/PREFER workshop 15-16 June
How much risk do patients find acceptable for a given benefit? Patient preferences can give us answers that can play a critical role in the development of medical treatments and throughout the lifecycle of a medical product. On June 15-16 this year, PREFER is organising a workshop together with DIA, where we will navigate through the patient preferences landscape. Starting from the PREFER project, we will explore patient involvement in patient preference studies and put the spotlight on the practical implications patient preference information can have in regulatory decision making. The workshop is open for everyone, free of charge!
Bringing patients’ views into medical approvals
Patients want to have a say in decisions that affect their health. But decision-makers have not had the tools to listen. Patient preference studies offers just that: a tool for decision-makers to collect, and for patients to give, representative and well-informed input. Karin Schölin Bywall’s dissertation reveals when and how including patient preferences in regulatory decision-making.
Miss our webinar on the value of patient preferences? Video now available!
On 26 April, the PREFER project organised the first in a series of webinars presenting and discussing results from the project. Want to know what a patient preference is? And how preferences are different from patient reported outcomes? You can now watch a recording of the presentations. The presentations covers the actors that make decisions throughout the medicinal product life cycle, and how they can benefit from patient preference studies. We also explain what we mean by patient preference sensitive situations.
PREFER newsletter out!
The PREFER project is well on the way to delivering results. We are organising a series of webinars to present and discuss results, a workshop together with DIA on patient preferences. There is also plenty of publications to look forward to. Didn't receive the newsletter today but want to be kept in the loop? Sign up!
Save the date for the DIA/PREFER patient preferences workshop 15-16 June!
Patient preferences can give us information that is critical for developing medical treatments. But they can also tell us how much risk patients think is acceptable for a given benefit. The pharmaceutical industry, regulatory authorities, HTA bodies, reimbursement agencies and patient organisations all agree that ‘patient preferences’ need to be part of decision making on benefit and risk. But how? When? And what are the regulatory requirements for preference studies? Join us online on 15-16 June to find out!
PREFER webinar 26 April on the value of patient preferences in the medical product life cycle
What is a patient preference and what is the difference between a preference and patient reported outcomes? Join us on 26 April in a webinar to listen and discuss. We will talk about the actors in the medicinal product life cycle and how they can benefit from patient preference studies, and explain what we mean by patient preference sensitive situations.
Working together to develop a preference study for treatments to reduce risk of developing RA
Figuring out what patients prefer can help inform decision making during drug development. But getting useful results requires rigorous preparation. An international team of PREFER researchers including academics, clinicians, pharmaceutical industry representatives and patient research partners have worked together to develop a preference study capable of informing decision making and also answering questions for the PREFER recommendations about the methods used to study patient preferences. In a recent BMJ Open publication, they describe how they designed their study to be informative for a wide range of stakeholders.
Do educational tools influence what patients prefer?
Stakeholders across the medical product life cycle are eager to find out what patients prefer. From the pharmaceutical companies that develop new treatments, to the authorities that decide if they should become available to patients, and at what price. But to be useful in decision-making, the patient preferences collected need to be informed. A recent Patient Education and Counseling publication explores the use of educational tools in patient preference studies, and finds that sometimes, they influence patients’ preferences.
Patient preference priorities: Questions to answer for patient-centric decision-making
Patient preference assessment is an increasingly popular approach to engaging patients in decisions across the medical product life cycle. But questions remain about how to incorporate scientifically valid preference measurements into decision-making. Especially for decisions about medical treatment, including development, regulatory and reimbursement decisions. PREFER partners have identified questions related to the knowledge gaps that are most crucial to decision-making stakeholders.
How to ask haemophilia patients about their preferences for gene therapies
Recent developments in gene therapy offer haemophilia patients the promise of permanent benefits or even a cure. But because these treatments are new, there are uncertainties about long-term efficacy and safety. This is a challenge for agencies that decide if a drug should be approved and what it should cost. PREFER researchers designed the very first patient preference study about what people with haemophilia think about gene therapies being brought to market.
PREFER: Patients and researchers in partnership
Giving patients a voice in the development of treatments means we first need to listen to them. The PREFER project develops recommendations for when and how that voice can be heard through a structured approach known as patient preferences. We rely on four patient organisations to make sure the patient voice is heard in the project. Together with universities and companies, they are part of developing recommendations for when and how patient preferences can be part of decision-making on whether or not to develop a treatment, if it should be approved, become available to patients, and what to do in case there are safety concerns after it is put on the market.
What matters most to lung cancer patients?
Several treatment options are available for non-small lung cancer, which is what 85 % of all lung cancer patients suffer from. With different benefits, risks, and uncertainties related to each treatment option, finding out what is most valuable to patients can help support decision-making. A recent PREFER publication reveals what treatment aspects lung cancer patients find most important and as a result will be included in an upcoming preference survey that investigates how patients make trade-offs between treatment options.
A Qualitative Protocol for a Patient Preference Study among Lung Cancer Patients
Lung cancer is the most prevalent cancer worldwide. And the deadliest. Treatments have different characteristics, all associated with a range of risks and benefits to patients. A new PREFER publication outlines the value of using a qualitative approach with advanced lung cancer patients to identify preferred treatment characteristics. With the aim of informing decision-making for the companies that develop medicines, regulators, payers as well as clinicians.
What do Multiple Myeloma patients prefer?
Right now, the PREFER project is recruiting patients with Multiple Myeloma to participate in an online survey. The survey will help us understand patient preferences in relation to the risks and benefits of myeloma treatments. The results may inform stakeholders on treatment outcomes, side-effects and symptoms that most significantly impact MM patients’ treatment attitudes and choices. It will also contribute to answering some of the PREFER project’s more methodological questions. In doing so, this study supports our goal of finding out when and how patient preferences should give input to decision making for the companies that develop medicines, and the authorities that approve them, and decide what becomes available to patients.
Rheumatoid arthritis patients’ preferences in regulatory decision-makings
There are many treatment options available for patients with rheumatoid arthritis (RA). Patients’ preferences for treatments vary significantly. Finding out what patients prefer can support regulatory decision-makers to make better decisions when deciding what treatments should be approved and made available to patients. A recent publication explores what RA patients prefer, and how different their preferences are.
Season's Greetings from the PREFER project
The PREFER project wishes you a happy new 2021! This year has affected us all in unexpected ways. Changing how we live, how we work, and project timelines. Despite the circumstances, our students delivered two PhD theses! We stayed connected, and submitted our framework to EMA and EUnetHTA for qualification advice. Want to stay in touch? Sign up to receive our newsletter!
Patient preferences throughout the medical product life cycle: Chiara Whichello’s PhD defence
Finding out what patients prefer is critical for the successful development, regulation and reimbursement or medical products. And for creating a patient centric decision-making within the medical product lifecycle. Before patient preference studies can be incorporated successfully into decision-making, stakeholders need more methodological clarity. Chiara Whichello’s PhD project has centered on this issue. On December 9, she defends her thesis.
Most haemophilia patients are positive towards gene therapies
Structured patient input can support decision-making across the medical product life cycle. This is where patient preference studies are useful. In a recent Haemophilia publication, PREFER researchers share their findings regarding haemophilia patients’ preferences for gene therapies. Despite the uncertainties about long-term efficacy and safety of the treatments, it turns out most of the patients are positive.
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.
Paving the way: First PREFER PhD defence
Eline van Overbeeke has investigated whether patient preference studies could help bridge the gap between new treatments and well-established decision-making structures. And bring gene therapies to market and patients sooner. She defends her thesis on 9 October 2020.
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.
What is the value of treatment effects according to Multiple Myeloma patients?
Patient preference studies provide evidence about what treatment effects are important to patients, how important they are to patients, and quantify the tradeoffs patients are willing to make between treatment effects. Understanding patient preferences may be especially valuable in the case of multiple myeloma, where the existence of treatments with different effects raises uncertainty about the value of these treatments effects to patients.
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.
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!
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: 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.
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.
New research initiative on patient preferences
The UK National Institute for Health and Excellence (NICE) launches two year study on patient health preferences.
ETHICS BLOG: How to listen to (the right) patient voices?
Ulrik Kihlbom, co-lead for the work to assess methodologies, writes about some of the challenges to finding the patients voice.
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.
Value in Health publishes theme section on patient preferences
The latest issue of Value in Health incudes a themed section on the incorporation of patient preferences into health care decision making.
PREFER kick-off 27-28 October
The kick-off meeting of the IMI project PREFER will be convened on October 27-28th 2016 at the Novartis Learning Center Horburg in Basel.
HTA and Payers Stakeholder Advisory Group formed
The needs and requirements of HTA agencies and Payers regarding patient preferences differ from those of the regulators. Both focus on risk-benefit assessment, but HTA bodies and reimbursement agencies are also interested in the relative effectiveness of a product. To make sure that their needs and requirements are taken into account, PREFER has formed a HTA and Payers Stakeholder Advisory Group.
Age, education and health literacy affects how people understand risk
We know that people have difficulties interpreting risk information correctly. This is a challenge for researches who want to know how people weigh possible risks against benefits. A recent study shows that age, education and health literacy affect the ability to understand risks. This means researchers need to have strategies in place for accurately measuring preferences regarding risk.
Patient advisory group formed
Patient input is crucial to PREFER's success. On September 1st, we formed the patient stakeholder advisory group to make sure the patient perspective is included at all levels of the project. The group is co-ordinated by the European Cancer Patients Coalition (ECPC),
The science of patient input
Margaret Anderson and Kimberly McClearly published a perspective paper on patient engagement in Science and Translational medicine earlier this year. According to them, creating a science of patient input is still in its early days.
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.
According to Karin Schölin Bywall, the PREFER project offers the chance to work with PhD students from other universities. PREFER also allows her to develop research questions with senior researchers from universities and the pharmaceutical industry. This collaboration also ensures stakeholder perspectives in her PhD project.
Willingness, preferences and medical products
What is it that makes patients decide to accept a treatment with serious side effects? And what would make them say no? Jorien Veldwijk explains how Discrete Choice Experiments can help find out what patients prefer.
Jorien Veldwijk is an expert in Discrete Choice Experiments, or ‘DCE’s” as they are often called. Her PhD thesis used DCE’s to look at preferences for and willingness to participate in different public health interventions. DCE’s are one of the methods that the PREFER project will look at. This method makes it possible to find out how people weigh benefits and risks against each other. She is public co-lead for PREFER’s clinical case studies, where the methods selected by the project will be tested in different patient groups.