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Treatment Sequencing in Multiple Myeloma: modeling the disease and evaluating cost-efficacy vs. cost-effectiveness

Projectomschrijving

Samenvatting na afronding

Patiënten in de dagelijkse praktijk kunnen verschillen van patiënten in klinische studies. Dit project onderzocht de impact van verschillen op de kosteneffectiviteit. De effectiviteit uit klinische studies is vergeleken met de effectiviteit in de dagelijkse praktijk voor multiple myeloom behandelingen die al langer in Nederland gebruikt werden. Voor sommige behandelingen was de effectiviteit vergelijkbaar, maar niet voor alle behandelingen.
Met een ziektemodel zijn de verwachte kosten en effecten berekend voor bestaande en verwachte behandelmogelijkheden. Het model geeft een goed inzicht in de effecten, kosten en kosteneffectiviteit van het toevoegen van een nieuwe behandeling aan het huidige behandelpalet.

Resultaten

Resultaten laten zien dat nieuwe behandelingen effectief zijn maar ook hoge kosten met zich mee brengen ten opzichte van de huidige behandelingen. De kosteneffectiviteitsratio van bestaande behandelingen was hoger (minder gunstig) op basis van de effectiviteit in de dagelijkse praktijk dan de ratio op basis van effectiviteit uit klinische trials.

Samenvatting bij start

De (kosten-)effectiviteit van medicatie wordt bepaald door onderzoek in klinische trials. Daarin bekijken onderzoekers verschillen tussen verschillende behandelingen. Maar bij de hematologische aandoening multipel myeloom gaat het niet om de effecten van enkele behandelingen. Het gaat vooral om de effecten van de verschillende manieren waarop behandelingen op elkaar kunnen volgen. Om die effecten goed te kunnen vergelijken ontwikkelden onderzoekers een speciaal gezondheidseconomisch model. Maar het is nog niet duidelijk, wat dit theoretische model precies zegt over de (kosten-)effectiviteit van verschillende opeenvolgingen van behandelingen in de klinische praktijk. Sinds kort worden de gegevens over patiënten met multipel myeloom systematisch samengebracht. Die verzameling van klinische data maakt onderzoek mogelijk naar de klinische praktijk. Dit onderzoek wil met behulp van gegevens uit deze databank onder andere:
  • onderzoeken in hoeverre het ontwikkelde model de klinische praktijk weerspiegelt
  • het model - indien mogelijk - zodanig verbeteren, dat het de (kosten-)effectiviteit in de klinische praktijk zo goed mogelijk voorspelt.

Producten

Titel: The Relationship of Response on Time to Next Treatment Based on Evidence from Two RCTs in Newly Diagnosed Stem Cell Transplantation Ineligible Multiple Myeloma Patients
Auteur: Chrissy van Beurden-Tan, Hedwig Blommestein, PhD, Sonja Zweegman, MD, PhD, and Pieter Sonneveld, MD, PhD
Titel: Systematic Literature Review and Network Meta-Analysis of Treatments for Relapsed/Refractory Multiple Myeloma Patients
Auteur: Chrissy van Beurden-Tan, Margreet Franken, PhD, Hedwig Blommestein, PhD, Carin Uyl-de Groot, PhD, and Pieter Sonneveld, MD, PhD
Titel: THE PROPORTIONAL ODDS MODEL IS MORE EFFICIENT THAN THE MULTINOMIAL LOGISTIC MODEL FOR NETWORK META-ANALYSES OF ORDERED OUTCOMES
Auteur: Bouwmeester W, van Beurden-Tan C, Bennison C, Heeg B
Titel: Time to Next Treatment Is Response Dependent and Treatment Independent Based on Evidence from RCTs in Stem Cell Transplantation Eligible Multiple Myeloma Patients
Auteur: Chrissy van Beurden-Tan, Philippe Moreau, MD, PhD, Laura Rosiñol, MD, Phd, Walter Gregory, PhD, MD, Michel van Agthoven, PhD and Pieter Sonneveld, MD, PhD
Titel: COST-EFFECTIVENESS OF INDUCTION TREATMENT WITH BORTEZOMIB ADDED TO THALIDOMIDE AND DEXAMETHASONE IN NEWLY DIAGNOSED MULTIPLE MYELOMA PATIENTS ELIGIBLE FOR AUTOLOGOUS STEM CELL TRANSPLANTATION IN GERMANY
Auteur: van Beurden-Tan C;Rosiñol L;Diels J;Wirth D;Chirita O5;Lahuerta JJ;Gaugris S;Marsh S;San Miguel J;Spencer M;Treur M, Bladé J
Titel: REVIEW OF META-ANALYSIS METHODS FOR MULTINOMIAL DATA
Auteur: Bennison C;van Beurden-Tan C;Buyukkaramikli C; Heeg B
Titel: TREATMENT SEQUENCING SURVIVAL MODEL FOR PATIENTS WITH MULTIPLE MYELOMA INELIGIBLE FOR STEM CELL TRANSPLANTATION (SCT)
Auteur: Bart Heeg, Michel van Agthoven, Chrissy van Beurden-Tan, Johan Liwing, Ulf-Henrik Mellqvist, Torben Plesner, Floris Logman, Johan Aschan, Hermann Einsele, Martin Kropff, Maarten Treur, Mirjam Barendse, Paul Richardson, Antonio Palumbo, Hareth Nahi, Pieter Sonneveld

Verslagen


Samenvatting van de aanvraag

In multiple myeloma (MM) an evaluation of the effect of different treatment sequences is more important than the evaluation of one treatment in one treatment line. There is a scarcity of health economic models in this disease, which hampers the evaluation of cost-effectiveness in this disease. We have developed a disease model and health economic model that gives account for the distinctive assets of MM and that models the disease reliably (Heeg et al. 2010). An unresolved question though is the relationship to data from daily clinical practice. This question however is becoming more and more important in the light of the desire to evaluate the performance of drugs in daily clinical practice. Last year, an observational registry of patients with MM (PHAROS: Population based HAematological Registry for Observational Studies) has been established in the Netherlands. This registry, in combination with the mentioned model offers an opportunity to explore the topic of the relationship between cost-efficacy and cost-effectiveness further. In this project, we will focus on three steps: First, on the basis of a comparison with the PHAROS dataset, we will investigate the level of representativeness of the current model (which is based on published clinical trial data) for the treatment in daily practice. This is very important, as current procedures for reimbursement of expensive hospital drugs require the availability of a t=0 model that gives an estimation of the drug’s cost-effectiveness at a time (market introduction of the drug) at which only published trial data are available. The question how representative a cost-efficacy model can be for cost-effectiveness is therefore a very valid one from a policymaker’s point of view. It is likely that the comparison with data from the PHAROS database will show the required improvements that are needed to make the model valid for an improved prediction of daily practice costs and effects. Second, on the basis of the previous step, we will further improve the t=0 health economic model by incorporating risk factors, prognostic factors, treatment modifying variables, links with cytogenetic research and decision making for choice of therapy and change of treatment in individual patients. This is an important step in the sense of adapting the model such that the validity is improved further, and hopefully as a second result to also improve the validity to daily clinical practice further (the latter question however remains the biggest endeavour here). Also, we will try to expand the model to the population of MM patients that is eligible for stem cell transplantation (currently not included). The updated model therefore has two goals: to further improve the ability to model multiple myeloma, and ultimately: to predict cost-effectiveness from cost-efficacy. An important remark here is that this endeavour should be accompanied by a further study into the question whether “cost-effectiveness in daily practice” is a feasible concept at all. An earlier pilot study performed by the iMTA showed that the variety of treatments in MM is too wide to draw valid conclusions about cost-effectiveness in daily practice. A further study into this matter will define the boundaries of the updated model. It is not impossible that a conclusion will be that there are strict limits regarding the kind of evidence that can be incorporated in the updated model anyway, e.g. randomized controlled trial data only in order to maintain internal validity as opposed to the use of daily practice data. Thirdly, we will attempt to model the disease, such that the starting point of the modeling work is explanatory variables related to the disease progression rather than aggregated outcomes of clinical trials or observational data. This would make it easier in the future to have a pre-evaluation of cost-effectiveness of new treatments in multiple myeloma even before the daily practice data are available. In summary, this project aims to contribute to the understanding of the representativeness of efficacy for effectiveness and what this implies for cost-effectiveness for new (expensive) drugs. On that behalf, an existing health economic model that gives account for treatment sequences rather than individual treatments will be further optimized. By means of the knowledge gathered, this project will also contribute to the understanding whether “cost-effectiveness in daily practice” is a feasible concept at all.

Onderwerpen

Kenmerken

Projectnummer:
152001020
Looptijd: 100%
Looptijd: 100 %
2012
2017
Onderdeel van programma:
Gerelateerde subsidieronde:
Projectleider en penvoerder:
Prof. dr. P. Sonneveld
Verantwoordelijke organisatie:
Erasmus MC