Season's Greetings from the PREFER project

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! 

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.

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.  

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.

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. 

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! 

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!

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.

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.

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. 

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.

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.

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. 

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.

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. 

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!

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.

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.

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!

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!

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.

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. 

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 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. 

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.

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.  

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.

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. 

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.

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. 

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!

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. 

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. 

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. 

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. 

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!

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 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. 

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. 

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.

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.

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.

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!

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.

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. 

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.

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.

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.

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.

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).