The Development of a Flexible and Easy to Tailor Disease Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma by Combining Trial and Real-World Data

Author(s)

de Groot S¹, Blommestein HM², Leeneman B², Uyl-De Groot C², Haanen JBAG³, Suijkerbuijk KPM⁴, Aarts MJB⁵, van den Berkmortel FWPJ⁶, Blank CU³, Boers-Sonderen MJ⁷, van den Eertwegh AJM⁸, de Groot JWB⁹, Hospers GAP¹⁰, Kapiteijn E¹¹, de Meza MM¹², Piersma D¹³, van Rijn RS¹⁴, Stevense-den Boer MAM¹⁵, van der Veldt AAM¹⁶, Vreugdenhil G¹⁷, Wouters MWJM³, Franken M¹⁸, van Baal PHM²
¹Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, ZH, Netherlands, ²Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, Netherlands, ³Netherlands Cancer Institute, Antoni van Leeuwenhoek, Amsterdam, Netherlands, ⁴University Medical Center Utrecht Cancer Center, Utrecht, Netherlands, ⁵Maastricht University Medical Center, Maastricht, Netherlands, ⁶Zuyderland Medical Center, Sittard-Geleen, Netherlands, ⁷Radboud University Medical Center, Nijmegen, Netherlands, ⁸Cancer Center Amsterdam, Amsterdam UMC, Amsterdam, Netherlands, ⁹Isala, Zwolle, Netherlands, ¹⁰University Medical Center Groningen, Groningen, Netherlands, ¹¹Leiden University Medical Center, Leiden, Netherlands, ¹²Dutch Institute for Clinical Auditing, Leiden, Netherlands, ¹³Medisch Spectrum Twente, Enschede, Netherlands, ¹⁴Medical Center Leeuwarden, Leeuwarden, Netherlands, ¹⁵Amphia Hospital, Breda, Netherlands, ¹⁶Erasmus MC Cancer Institute, Rotterdam, Netherlands, ¹⁷Maxima Medical Center, Eindhoven, Netherlands, ¹⁸Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, Netherlands

Presentation Documents

Europe21_DeGroot

OBJECTIVES: Although in the last decade many novel treatments have been introduced for advanced melanoma (unresectable stage IIIc/V) patients, evidence on the outcomes of treatment sequences is lacking. Our aim was to develop a flexible disease model for advanced melanoma to estimate long-term effectiveness of treatment sequences, to support clinical guideline development and reimbursement decision making.

METHODS: A semi-Markov model with a life-time horizon was developed. Transitions describing disease progression, next treatment and mortality were estimated from real-world data, as a function of time since start of treatment or disease progression, and patient characteristics. All transitions can be adjusted based on the relative effectiveness of treatments that were derived from a network meta-analysis. Additionally, the duration of treatment effect and time horizon can be changed to demonstrate outcomes under different assumptions.

RESULTS: The model distinguishes three active treatment lines for BRAF mutant melanoma and two active treatment lines for BRAF wild-type melanoma. Depending on treatment, mean life expectancy of BRAF mutant melanoma patients with a poor, intermediate or favourable prognosis ranged from 2.2 to 3.4, 5.6 to 7.0 and 9.9 to 11.3, respectively. Mean life expectancy of BRAF wild-type melanoma patients with a poor, intermediate or favourable prognosis ranged from 4.3 to 5.8, 5.8 to 8.2 and 7.8 to 9.7, respectively. The scenario-analyses illustrate how estimates of life expectancy crucially depend on duration of treatment effect and time horizon.

CONCLUSIONS: Our model is flexible and can be tailored to answer questions concerning (cost-)effectiveness. Treatments and sequences of treatments can be adjusted, as well as the duration of treatment effects and the transitions influenced by treatment effect. With this model, we show how to combine real-world data with data from clinical trials to benefit most from the advantages of both data sources, which can guide the development of future disease models.

Conference/Value in Health Info

2021-11, ISPOR Europe 2021, Copenhagen, Denmark

Value in Health, Volume 24, Issue 12, S2 (December 2021)

Acceptance Code

P71

Topic

Clinical Outcomes, Economic Evaluation, Health Policy & Regulatory, Methodological & Statistical Research

Topic Subcategory

Comparative Effectiveness or Efficacy, Cost-comparison, Effectiveness, Utility, Benefit Analysis, Reimbursement & Access Policy

Disease

Oncology

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Presentation (Paper)