Myeloma Patients Europe (MPE) is involved in a Horizon 2020 project called MMpredict. The project consortium is aiming to develop a personalised medicine tool that can predict the most effective treatment strategy for myeloma patients.
To inform the project, MPE is running a survey with myeloma patients across Europe to understand their quality of life and how this is impacted by treatment side-effects. The survey also asks about patients’ attitudes towards genetic testing and risk stratification (e.g. understanding whether a patient has “high” or “standard” risk myeloma).
The project is explained below in more detail, alongside information on how the survey results will be used to inform the outcomes of the project. For more information, please visit the website www.mmh2020.eu or email MPE Policy Manager Kate Morgan at email@example.com.
Horizon 2020 MMPredict
What is Horizon 2020?
Horizon 2020 is a large-scale EU Research and Innovation programme which has around €80 billion of funding available over seven years (2014-2020). It funds a wide range of different projects relating to science, innovation and tackling societal challenges. To receive funding through the programme, multi-stakeholder consortiums work in partnership to develop a project proposal, which is reviewed by the European Commission. MMpredict was approved by the European Commission and started in November 2016. Over the duration of 42 months, the consortium will distribute the allocated funding proportionately to consortium members to carry out their assigned tasks.
What is the MMpredict project?
The MMpredict project is working towards the development of a tool which can help myeloma doctors predict the most effective treatment for their patients. The aim is to ensure that each patient receives the most suitable treatment regimen with the highest effectiveness and the fewest side-effects, right from the start of the treatment.
How will this tool be developed?
SkylineDx, a member of the project consortium, has already developed and validated a diagnostic device called the MMprofiler™ which can determine the level of risk of a myeloma patient by classifying them into “high” or “standard” risk groups. Patients with “high” risk myeloma do not normally respond as well to treatment and are likely to relapse more quickly than patients who have “standard” risk myeloma. This classification is done using gene expression profiling (GEP), a technology in which the activity (or “expression”) of specific genes is measured in tissue samples – creating a patient-specific picture. The GEP of an individual patient represents their biology and can give important clues on response to drugs.
The aim of the project consortium is to build upon this approach by developing a tool which can help myeloma doctors predict the most effective treatment for patients. This will be done through using predictive biomarkers, which are measurable indicators of response to treatment and disease progression. Assessing this within patient tissue samples should give researchers information on the treatment or treatment combinations which are most effective in each individual patient based on their own myeloma subtype. Correlations between predictive biomarkers and the effect of treatment or treatment combinations will then be processed into a treatment decision matrix, which should enable myeloma doctors to determine personalised medicine strategies.
What is the rationale behind the project?
Being able to predict myeloma patient responses to treatment would be beneficial to patient outcomes. Whilst outcomes in myeloma are gradually improving, there remains largely a one-size-fits-all approach to the treatment and care of patients. High-risk myeloma patients are typically associated with poorer outcomes; however, treatment approaches do not yet differ to patients who have standard risk myeloma. MMpredict should enable a more personalised treatment approach in distinct groups of patients.
In addition to this, myeloma treatment pathways in some European countries are becoming increasingly complicated and expensive. Individualised approaches to treatment will increase value-for-money of medicines as doctors should be able to predict when a patient will respond. Predictive approaches such as the one being explored by the MMpredict project consortium are therefore important to healthcare systems across Europe.
What is the MMpredict project consortium?
The project consortium is the partnership of organisations that are involved in delivering the work of MMpredict. This partnership ensures that a range of complementary skills and expertise is involved in the project.
The consortium partners are:
- SkylineDx B.V. (Netherlands)
- University of Turin (Italy)
- Erasmus University Medical Center (Netherlands)
- Erasmus School of Health Policy & Management (Netherlands)
- Myeloma Patients Europe AISBL (Belgium)
The work of the project consortium is coordinated by SkylineDx, who have appointed a project officer for the duration of the grant.
What is MPE involvement in the study?
The primary role of MPE is to ensure the patient voice is reflected in the project through conducting a survey on the quality of life of patients and patient preference on treatment. It is important that individualised approaches to the treatment of myeloma patients should also take account of the preferences of patients and the impact treatments have on quality of life. In addition to the survey, MPE will be communicating about the project with members and other external audiences.
Are there any similar studies in progress?
Yes. Given the importance of this research area, there are several other studies looking into predictive tests for treatment response in myeloma and predictive models for individual treatments. Below are examples of other organisations undertaking work in this area.
- The Medical Prognosis Institute, Denmark, created a tool called the Drug Response Predictor (DRP®) which has been shown to have the potential to provide a personalised medicines tool in myeloma and other cancers
- Quest Diagnostics has developed a range of diagnostic tools in myeloma including a tool to help doctors better understand a patient’s response to daratumumab (Darzalex®) treatment
- The University of Arkansas Medical School (UAMS) developed tools to effectively predict the outcome for of myeloma patients related to their risk profile – these are known as MyPRS™ and MyPRS Plus™. These tools were bought by Quest Diagnostics in 2016 to explore further
- Cellworks and the University of Florida, Gainesville alongside other partners highlighted the utility of computer modelling and digital drug simulations in the prediction of treatment responses in myeloma patients
About the MMPredict Survey
What does the MMpredict survey ask?
The survey will ask myeloma patients about their quality of life and their experience of different treatments received for their myeloma. This is done using standardised questionnaires that are used routinely to measure health-related quality of life (HRQoL) in patients – the EuroQol EQ-5D-5L and the EORTC QLQ-C30. HRQoL refers to the impact that health status has on a person’s ability to complete normal daily activities. The survey also asks about patients’ attitudes towards genetic testing and risk stratification of patients (e.g. understanding high or low-risk myeloma).
How will the results of the survey be used?
The survey results will be used to develop the outcomes of MMpredict. By combining the responses of all participating patients, the aim is to have a better understanding of the most relevant quality of life issues for patients and relate these to specific treatments or disease characteristics. This will provide valuable insights to help the consortium understand the kind of diagnostic tool that could be valuable for patients and their doctors when choosing treatments.
Which countries are involved in the survey?
The survey is focusing on assessing the views of myeloma patients in six European countries: the United Kingdom, the Netherlands, Germany, Belgium, Italy and France. The survey has therefore been translated into relevant languages. These countries have been chosen because of the model of their healthcare system (e.g. how their reimbursement systems work) and the numbers of myeloma patients present in the population. The results will therefore be analysed primarily based on responses from these countries.
However, the project consortium is also seeking responses from as many myeloma patients in Europe as possible – so the survey is open for patients of all nationalities.
Is the survey confidential?
Yes. The information collected through this questionnaire will be anonymous and confidential. Moreover, any collected data will be restricted for the use of this study only and will not be shared with or given to anyone, except for the authorized research staff.
Where I can find further information?
If you have any further questions about the study, please email MPE Policy Manager Kate Morgan at firstname.lastname@example.org
A very big thank you to AMM-online, Myelom Gruppe Rhein-Main/Leukämiehilfe Rhein Main, Hematon and Myeloma UK for their involvement in the survey so far. You can find further information and contact details of our members here.