The following is a selection of current research projects in which HTA research group is involved. A short summary is provided for each project:
What is the right balance between the costs and benefits of good treatment? This study compares two groups of patients with hay fever and house dust mite allergy. One group will continue their standard treatment of tablets, nose spray and/or ear drops. The other group receives, in addition, a highly diluted solution of their allergen. This treatment is known as immunotherapy. No one is given fake medicine. This approach makes it possible to study whether the drawbacks to immunotherapy outweigh the advantages.
The advantages are long-term decrease in allergic complaints, resulting in the patient needing less medication, feeling better at home and at work and not being off sick as often.
The drawbacks are the high cost of the treatment, the length of the treatment (monthly injections for 3-5 years) and potential side effects. Immunotherapy is therefore a good treatment, but it is certainly not ideal. That is why this study uses the responses to Internet questionnaires to explore whether immunotherapy is worth the money and the trouble involved.
If someone lives longer thanks to preventive or curative treatment, it is very likely that this person will consume medical care in his or her additional years of life. Take John, for instance, who underwent a successful heart transplant when he was 60 years old. Thanks to the heart transplant, John will not die at 60, but at 75, and he will consume medical care during these 15 additional years. This medical care in the additional years is often referred to as ‘indirect medical costs’ in specialist literature.
A tool kit has been developed for including indirect medical costs in economic assessments in a standardised way. This tool kit is called PAID 1.0: Practical Application to Include future Disease costs. The goal of this project is to expand the methodology of PAID in order to quantify the uncertainty regarding the estimation of indirect medical costs. The result will be PAID 2.0.
Drugs can have a positive or negative impact on a patient’s quality of life. In the Netherlands, drugs that provide a very significant improvement in quality of life are allowed to be more expensive than drugs that do not. Sometimes, however, no research at all has been conducted to obtain information about the patients’ quality of life. This makes it difficult to determine whether a drug is worth the money.
We used three methods – mapping, direct valuation and ranking – to determine the best way of replacing missing data on quality of life.
All three methods were found to work. The preferred method depends on the user’s goal. The mapping technique is preferable for use in analyses of drug cost-effectiveness. This technique is best suited for replacing the missing quality of life data.
Updating parameters of decision-analytic cost-effectiveness models: a systematic comparison of methods.
After the introduction of a new drug, various trials are often conducted to compare the drug with other drugs. These trials often reach (slightly) different conclusions. This project is about bringing together these various sources of information. This process is called meta-analysis, or data synthesis. We will compare a number of frequently used meta-analysis methods.
A major problem in health care is that there is often no direct evidence, only indirect evidence: drug A is compared with drug B, and drug B is compared with drug C, but there is no direct evidence regarding drug A compared with drug C. There are also various methods involved here, which we will compare.
The study mainly looks at what the different methods of meta-analysis mean for weighing up the costs (e.g. higher price for the drug) and benefits (e.g. living longer) provided by the new drug compared to the old drug (i.e. the cost-effectiveness).
Hospitals are facing increasing numbers of COPD patients suffering bouts of acute shortness of breath. In the GO-AHEAD project, patients were therefore discharged after just three days in hospital. They then received four days of home care. In this sub-project, we explore the reasons why the parties concerned choose either hospital or home care.
Patients, carers and care providers completed a questionnaire. They were asked to choose between three treatments: standard hospitalisation or two combinations of shorter hospitalisation followed by home care. The characteristics of these treatments were always different: the frequency of home visits by the nurse, the co-payment, the burden on the carer, the number of different nurses, the risk of rehospitalisation and the nurse’s level of training.
What are the benefits?
We can see exactly which factors determine the preference for hospital care or home care. The results are useful for doctors, hospitals, insurers and other organisations that are involved in the decision-making about treatments.
COMED is a highly ambitious project whose primary objective is to tangibly improve decision-making processes in European healthcare systems, by establishing a rigorous evidence base for some of the most pressing issues on the health policy agenda. We aim to advance scientific knowledge by improving existing and developing new research streams for cost and outcome analysis of medical devices. At the same time, we aim to provide immediately applicable policy advice and tools that will enhance the use of economic evidence in decision-making. Striving for methodological excellence and immediate applicability in decision-making processes is a great challenge for scientific research. COMED will fully embrace this challenge over the course of a three-year, intensive research effort to develop novel concepts and innovative approaches.
COMED’s ambition is to develop scientifically rigorous recommendations on what, when and how real world evidence sources can and should be used for the evaluation of medical devices. COMED will develop new methods for assessing patient-reported outcomes for mHealth technologies. COMED will provide a comprehensive understanding of the main drivers of clinical practice variations across Europe, differentiating between warranted and unwarranted differences. COMED will promote research-driven policy advice and tools for the early assessment and conditional reimbursement of medical devices. The project will expand the geographic scope of evidence generation from a single jurisdiction to collaborative evidence generation throughout Europe. It will have a substantial impact by on public health in Europe, by providing scientifically robust evidence for a wide range of key stakeholders, including policy makers, patients and the wider public.
More information will follow.
HEcoPerMed stands for “Healthcare- and pharma-economics in support of the International Consortium for Personalised Medicine – ICPerMed". The project responds to the demand for economic models that evaluate treatments made possible through innovations in personalised medicine. HEcoPerMed also seeks to identify funding and reimbursement mechanisms that provide financial incentives for the rapid development and uptake of such innovations. HEcoPerMed goes beyond current assessment and payment models in order to serve the need of personalised medicine for more comprehensive cost-effectiveness estimates – incorporating patient and societal perspectives – and for sustainable affordability of cutting-edge health innovations.
PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products.
Over the next five years, we will run patient preference studies in both academic and industry settings. Our experience will provide a better understanding of what will be a recommended best-practice approach to patient-preference studies. We will also show how patient preference studies can give valuable information to support decision making for regulators and HTA bodies.
The main objective of this HORIZON 2020 project is to clinically validate a personalised medicine tool that predicts the most effective treatment option in myeloma patients. Currently, there are a range of single/combination novel agents and chemotherapy drugs available and in development for myeloma, the second most common type of blood cancer. As myeloma is a very heterogeneous disease, not every treatment is suitable for each patient. There is therefore an urgent need for a diagnostic tool that supports treatment decision-making in order to improve patient health outcomes, increase the value of new medicines to healthcare systems and to reduce healthcare costs.
Optogenerapy represents an innovative and effective therapeutic delivery with an impact on slowing the disease progression and increasing the Multiple Sclerosis patients’ quality of life.
The novelty lies in the continuous delivery of IFN-ß, overcoming current limitations of short drug half-life in vivo, adverse immune reactions, and pain and irritation at the site of local injection.
A prototype will be validated in relevant animal models using GLP and ISO testing procedures. All prototype manufacturing processes will be in agreement with GMP, PAT, and manufacturability requirements such as upscaling, cost price and technology transferability. Regulatory requirements will also be taken into account for technology selection and design trade-offs.
ProgrammE in Costing, resource use measurement and outcome valuation for Use in multi-sectoral National and International health economic evaluAtions: PECUNIA aims to tackle the healthcare challenges of an ever-growing and rapidly ageing population in the EU by developing new standardised, harmonised and validated methods and tools for the assessment of costs and outcomes in European healthcare systems.
Comparing and exploiting data across different countries and sectors, PECUNIA aims to provide direct comparable solutions to improve chronic and mental healthcare in all EU health systems.
PREVENTOMICS project builds a new paradigm in preventive personalised nutrition exploiting the potential of omics sciences, especially metabolomics, and changes in habits as drivers of development.
During the project, PREVENTOMICS will deliver personalised nutrition tools accessible to everyone that promote changes in current habits and achieve the improvement of personal health in a sustained and lasting manner.
The personalised plans for nutrition and lifestyle habits to improve the health of people are based on individual traits such as physical and behavioural traits, lifestyle, genotype, preferences and physical condition and will reach the recipients in in the form of behavioural prompts, thanks to the use of Information and Communications Technology (ICT) tools.
The applied research carried out by PREVENTOMICS will help to sustain new healthy lifestyle habits, increase user satisfaction and confidence in a more personalized diet and promote greater adherence to the Mediterranean diet as a healthy dietary pattern in line with the recommendations of the European Food Safety Authority (EFSA).
Within our consortium we have developed an academic infrastructure for the harmonized production of MSC. In the RETHRIM proposal this will be combined with our clinical expertise to conduct the first Europe-wide placebo controlled randomized phase III trial using MSC regenerative therapy for the treatment of steroid-resistant visceral GvHD. Central to the RETHRIM project is the clinical trial for which 150 patients will be recruited. All MSC products will be extensively
analysed using molecular and functional markers, in order to develop a potency signature for the product and for the prediction of response. We also intend to collect data from additional quality of life, health technology assessment and ethical studies. We will apply for an Orphan Drug Designation in Europe and this may serve as a stepping-stone for the further commercialization of the MSC product, once a positive outcome has been obtained.
The objective of the SPCCT project is to develop and validate a widely accessible, new quantitative and analytical in vivo imaging technology combining Spectral Photon Counting CT and contrast agents, to accurately and early detect, characterize and monitor neurovascular and cardiovascular disease.
The project will therefore provide a complete tool (acquisition system and specific probes) dedicated to CV imaging. It will finally contribute to:
- Improved early diagnosis of atherosclerosis, prevention of acute event (MI, stroke) and personalized preventive treatment;
- Improved management of patient presenting with an acute CV event and clinical validation of treatment efficiency;
- Sustainability and harmonization of healthcare systems, as costly disorders of heart failure and stroke-related disability would be better prevented and efficiently treated;
- Economic growth in the EU diagnostics sector, through the development of new targeted contrast materials for SPCCT by SMEs.