Here we offer insights into selected scientific projects at the Institute.
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The Economics of Clinically Proven Treatment Strategies
for Attention-Deficit/Hyperactivity Disorder (ADHD)
Children and Adolescents with ADHD:
Cost Effectiveness Analysis Based Upon the NIMH MTA Study
The NIMH-initiated Multimodal Treatment of Attention-Deficit/Hyperactivity Disorder (MTA) Study has been the clinical landmark trial in ADHD research, including 579 children age 7-9.9 years with ADHD according to DSM-IV criteria, who were randomly assigned to 14 months of medication management (MM), intense behavioral treatment (BEH), both combined (COMB), or community care (CC). In order to evaluate the cost-effectiveness of clinically proven treatment strategies (neither placebo nor single drugs) for ADHD and Hyperkinetic Disorder (HKD/HKCD, a subgroup meeting ICD-10-based diagnostic criteria used in Europe) in five countries, using patient-level data from the MTA Study over 14 months. Methods: Medical resource utilization data came from the MTA, excluding its research component. Unit costs (year 2005) were calculated from a societal and from a third-party payer’s perspective for Finland, Germany, Netherlands, Sweden, United Kingdom, and USA. Corresponding to the primary study endpoint, treatment response was defined as “normalization” of core symptoms. Utility estimates were derived from expert estimates and parent-proxy-ratings.
Incremental cost-effectiveness ratios (ICERs) were determined for the total study population and subgroups with pure ADHD (without comorbidity), pure HKD, or HKD/HKCD. ICERs were also calculated using functional improvement as a secondary clinical outcome criterion.
For additional analyses, patient subgroups were defined by coexisting conditions: pure ADHD (n=184, ~32%), ADHD and internalizing (n=81, ~14%) or externalizing (n=172, ~30%) comorbidities only, or ADHD and both comorbidities (n=142, ~25%). Again, cost-effectiveness was evaluated calculating incremental cost-effectiveness ratios (ICERs) and cost-effectiveness acceptability curves (CEACs).
The four MTA treatment strategies were all clinically effective. From an economic perspective, MM dominated BEH and exhibited extended dominance over CC compared to a hypothetical “Do Nothing” alternative. Results were broadly similar for symptomatic “normalization” as well as for functional improvement, across jurisdictions and across subgroups with pure ADHD, HKD/HKCD, and pure HKD. Sensitivity analyses including probabilistic evaluations using non-parametric bootstrapping supported these findings.
The overall findings were however somewhat moderated by the presence of co-existing conditions. In terms of symptomatic improvement, MM still consistently represented the economically most attractive strategy (across jurisdictions and comorbidities). In terms of functional improvement, however, MM was most attractive at relatively low levels of willingness-to-pay, whereas BEH was more attractive at moderately higher levels of willingness-to-pay for patients with internalizing comorbidity, and COMB became more attractive in the presence of externalizing comorbidities.
Despite international differences regarding standards of care, diagnostic criteria, and unit costs, key findings for European jurisdictions were consistent with US results. Likewise, the observed pattern of cost-effectiveness by comorbidity was remarkably similar across jurisdictions. Although cost-utility estimates for this pediatric population should be interpreted with caution, results indicated acceptable to attractive cost-effectiveness of an intense medication management strategy.
Cost Effectiveness of Long-Acting Medications for ADHD
Modeling the cost-effectiveness of long-acting stimulant medications compared to immediate-release forms from the perspective of the UK National Health Service (NHS) illustrated the sensitivity of incremental cost-effectiveness ratios (ICERs) to treatment persistence rates. An economic model, originally developed by the Canadian Coordinating Office for Health Technology Assessment (CCOHTA), was adapted using data from clinical efficacy studies, treatment compliance research, and retrospective database analyses. Results consistently indicated acceptable to attractive cost-effectiveness ratios for long-acting (modified-release) compared to short-acting (immediate-release) drug formulations and as such provided economic support for European treatment guidelines.
Budget Impact of Novel Medications for ADHD
Over more than a decade, pharmaceutical spending for patients with attention-deficit-hyperactivity disorder (ADHD) had been escalating internationally. Thus, future expenditures for ADHD-related medications were estimated from the perspectives of the statutory health insurance (SHI; Gesetzliche Krankenversicherung, GKV) in Germany and the National Health Service (NHS) in England, respectively. Further to this, budgetary impact was predicted for individual prescribers (child and adolescent psychiatrists and pediatricians treating patients with ADHD in Germany).
Inputs for the budget impact model were derived from demographic and epidemiological data, a literature review of past spending trends, and an analysis of new pharmaceutical products in development for ADHD. Uncertainty was addressed by way of scenario analysis. Annual ADHD pharmacotherapy expenditures for children and adolescents were predicted to further increase substantially. Overall drug spending by individual physicians might increase even more dramatically (2.3- to 9.5-fold), resulting from the multiplicative effects of four variables, namely, increased number of diagnosed cases, growing acceptance and intensity of pharmacotherapy, and higher unit costs of novel medications.
Adult Patients with ADHD:
Cost Effectiveness Analysis Based Upon the COPMAS Study
Very little is known about the comparative cost effectiveness of treatment strategies for adult ADHD. The COMPAS Study is a randomized, double-blind, placebo-controlled multicenter study that has been initiated in Germany under the lead of the University of Freiburg (A. Philipsen). Patients were assigned to one out of four parallel treatment arms: (1) a structured disorder tailored psychotherapy (dialectical behavioral therapy, DBT) plus medication (methylphenidate), (2) DBT and placebo, (3) psychiatric counseling without specific behavioral interventions (clinical management) plus medication, or (4) clinical management and placebo. DBT and clinical management are administered weekly for the first 12 weeks and on a four-weekly basis thereafter, until the end of the one-year-observation period. An additional follow-up investigation is scheduled at 18 months after treatment termination. The COMPAS project promises, for the first time, robust insights into the clinical effectiveness and cost effectiveness of a disorder-tailored psychotherapy for adult ADHD. Assessment criteria include symptomatic improvement (Conners’ Adult Rating Scale), general psychopathology, clinical global impression, and a disorder-specific quality of life questionnaire. In order to facilitate cost utility analysis, health-related quality of life is also measured by means of the EQ-5D and SF-36.
The study has been supported by the German Federal Ministry of Education and Research (Bundesministerium für Bildung und Forschung, BMBF).
Analysis Plan (212.3 KiB)