From the lab to you: How do medicines get approved?

Collecting patients’ preferences is a way to systematically listen to the patient voice in drug development. The companies that develop medicines and the regulators that decide if they should be approved and reimbursed all have a series of decisions to make. Knowing what patients would prefer can support their decision-making. There are many different methods to collect patient preferences and all have different characteristics, but which characteristics do stakeholders find important? A recent PREFER publication reveals the answer.

Rheumatoid arthritis, or RA for short, is a chronic inflammatory joint disease causing pain, swelling, stiffness and fatigue, as well as joint damage and disability. There is currently no cure for RA, and long-term treatment with immunomodulatory drugs is usually required. Early treatment, within a few months of symptom onset, improves patient outcomes. Clinical trials are currently investigating whether short-term treatment in people who are at risk of developing RA can delay the onset of the disease or lower the chance of it developing altogether. With increasing interest in preventive treatment in RA, PREFER researchers have explored why people at risk of RA may or may not consider taking medication for this purpose.

The role that patient preference information can play received recognition in the recent report from the CIOMS working group XI: Patient involvement in the development, regulation and safe use of medicines. The report devotes a section to patient engagement in patient preference studies, emphasising the importance of including patient perspectives in medical product decision-making. 

Rheumatoid arthritis (or RA for short) is a chronic disease that affects the joints and causes pain, stiffness and swelling. There are a number of ongoing clinical trials of treatments to prevent the development of RA. A survey of over 2,900 people just published in the journal Rheumatology provides evidence that most people would be willing to accept at least some side effects in return for an effective preventive treatment.

The PREFER project developed a framework with points to consider for methods selection. We asked the European Medicines Agency (EMA) and EUnetHTA to assess this document and issue a public qualification opinion on how useful the PREFER approach is from the regulatory and HTA perspective. In April 2022, the EMA committee responsible for human medicines, CHMP, issued a positive qualification opinion. We are now sharing lessons learned from the process and some of implications for the future. 

On 28 April 2022 the PREFER project launched recommendations on why, when and how to assess and use patient preferences in medical product decision-making. During the launch, we presented take-home messages from the PREFER Recommendations. Missed the event? Don’t worry! A recording is now available!

We are pleased to announce that Rosanne Janssens from KU Leuven won Best General Podium Research Presentation at ISPOR 2022, the annual conference for the Professional Society for Health Economics and Outcomes Research (ISPOR conference. The presentation was entitled PPreferences for Multiple Myeloma Treatment: A Stated Preference Survey Using Discrete Choice Experiment and Swing Weighting, presenting results from one of the PREFER project’s clinical patient preference case studies.

The PREFER project is officially ending today, but our legacy continues! On 9 June at 16:00 CEST, we invite you to discuss the future impacts of the European Medicines Agency's (EMA) qualification opinion on our framework and points to consider for patient preference studies. The online event is open to everyone and free to attend! 

After receiving the positive qualification opinion from the European Medicines Agency's (EMA) committee responsible for human medicines CHMP, we updated our recommendations. The new 1_1 version was published on 30 May, with minor edits, pointing to the feedback we received. 

The PREFER project developed a framework with points to for method selection that can be used by industry, regulators and health technology assessment bodies for how to use patient preferences as input in medical product decision making. We asked the European Medicines Agency and EUnetHTA to coordinate the assessment of our framework and its points to consider for method selection and issue a public qualification opinion on how useful the PREFER approach is from the regulatory perspective. Join us on 9 June, when we present and discuss the opinion in an online event at 16:00 CEST. 

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 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!

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!  

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. 

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.

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.

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.

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!

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. 

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. 

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.

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. 

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.

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.

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. 

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.

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. 

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!

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.