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
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.