Budgetary Impact and Cost Drivers for Rare and Ultrarare Diseases.
Value in Health (2017): DOI 10.1016/j.jval.2017.10.015.
To review recent studies reporting health care expenditures (budgetary impact) for orphan medicinal products (OMPs) in Europe and to contribute to our understanding of the cost drivers of nononcological OMPs by means of an empirical analysis in Germany. A systematic search for relevant studies on rare diseases was conducted in PubMed and Embase (until December 2016). In addition, annual treatment costs of nononcological OMPs in Germany were analyzed with respect to five explanatory variables: total prevalence of disease, prevalence with added benefit, availability of alternative treatments for the same indication, extent/probability of treatment benefit, and evidence for a treatment effect on mortality. A total of nine studies with specific estimates of the budget impact of OMPs for a total of 11 countries were identified; one study addressed specifically ultrarare diseases. Annual per-capita spending for OMPs ranges from €1.32 in Latvia to €16 in France. Per-patient annual treatment costs vary between €27,811 and €1,647,627 in Germany. On the basis of the German data set, the regression analysis shows that log prevalence has a significant inverse relationship with log annual treatment cost. In this model, doubling the prevalence leads to a 43% decrease in annual treatment cost. Despite per-patient annual treatment costs ranging up to several hundreds of thousands of euros for some OMPs, per-capita spending for OMPs is relatively small. In this study an inverse relationship between prevalence and annual treatment costs was found.
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Woran bemisst sich Effizienz im Gesundheitswesen? Zur Klärung fachwissenschaftlicher Begriffe und Kriterien.
Amos International 11 (2017) 1: 22-31.
The word efficiency has a positive connotation. Those who are not familiar with economic theory often do not realize that the technical meaning of the term differs markedly from common usage. This may cause misunderstandings and problematic consequences when economists make influential statements on efficiency and inefficiency in the health care system. An instructive case in point is the debate on efficient conduct and the so-called “rule of rescue”. To understand the underlying issues it is necessary to know about the expectations towards and objectives of health care systems. For “efficiency” can by definition be an instrumental objective only, secondary to the (primary) effectiveness criterion.
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Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases.
Orphanet Journal of Rare Diseases 12 (50), 2017: DOI 10.1186/s13023-017-0601-9.
The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field.
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Conventional Health Economic Evaluation Fails to Capture Social Value of Interventions for Rare and Ultra-Rare Disorders.
Drug Information Association (DIA) Global Forum, October 2016: 13-14.
Orphan drug legislation provided for a broad range of incentives for research and development (R&D) into interventions for the prevention and treatment of rare and ultra-rare disorders. These measures have contributed to a stream of new medications, some of which rank among “the most expensive drugs in the world”. In times of economic austerity, health care policy makers need to address whether these interventions offer “value for money”. Decision makers struggle with the absence of accepted validated tools how to determine – and how to quantify – the social value of such interventions.
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Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement.
Journal of Market Access & Health Policy 4 (2016): 33039; 1-9.
In order to critically appraise the problems posed by the systematic valuation of interventions for ultra-rare disorders (URDs), an international group of clinical and health economic experts was convened in conjunction with the Annual European ISPOR Congress in Berlin, Germany, in November 2012. Following this meeting and during subsequent deliberations, the group achieved a consensus on the specific challenges and potential ways forward. The group concluded that the complexities of research and development for new treatments for URDs may require conditional approval and reimbursement policies, such as managed entry schemes and coverage with evidence development agreements, but should not use as justification surrogate end point improvement only. As a prerequisite for value assessment, the demonstration of a minimum significant clinical benefit should be expected within a reasonable time frame. As to the health economic evaluation of interventions for URDs, the currently prevailing logic of cost-effectiveness (using benchmarks for the maximum allowable incremental cost per quality-adjusted life year gained) was considered deficient as it does not capture well-established social preferences regarding health care resource allocation.
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Comparison of Methylphenidate and Psychotherapy in Adult ADHD Study (COMPAS) Consortium: Effects of Group Psychotherapy, Individual Counseling, Methylphenidate, and Placebo in the Treatment of Adult Attention-Deficit/Hyperactivity Disorder: A Randomized Clinical Trial.
JAMA Psychiatry, 72 (12), 2015: 1199-1210.
Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder with high prevalence in adulthood. A prospective, multicenter, randomized clinical trial of 18- to 58-year-old outpatients with ADHD assessed the effects group psychotherapy, individual counseling, methylphenidate, and placebo in the treatment of adult ADHD. Patients received either methylphenidate or placebo for 1 year. The primary outcome was the change in the ADHD Index of the Conners Adult ADHD Rating Scale from baseline to the end of the 3-month intensive treatment (blinded observer ratings). Secondary outcomes included ADHD ratings after 1 year, blinded observer ratings using the Clinical Global Impression Scale, and self-ratings of depression. 433 patients were centrally randomized, and 419 were analyzed as randomized. After 3 months, the ADHD Index all-group baseline mean of 20.6 improved to adjusted means of 17.6 for GPT and 16.5 for CM, with no significant difference between groups. Methylphenidate (adjusted mean, 16.2) was superior to placebo (adjusted mean, 17.9) (difference, −1.7; 97.5%CI, −3.0 to −0.4; P = .003). After 1 year, treatment effects remained essentially stable. Descriptive analyses showed that methylphenidate was superior to placebo in patients assigned to GPT (difference, −1.7; 95%CI, −3.2 to −0.1; P = .04) or CM (difference, −1.7; 95%CI, −3.3 to −0.2; P = .03). Thus, a highly structured group intervention did not outperform individual CM with regard to the primary outcome. Psychological interventions resulted in better outcomes during a 1-year period when combined with methylphenidate as compared with placebo.
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Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe.
Expert Review of Pharmacoeconomics & Outcomes Research, 15 (1), 2015: 171-179.
Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012–2021) and a pan-European payer’s perspective, based on prevalence data for UODs for which patented drugs are available and/or for which drugs are in clinical development. A total of 18 drugs under patent protection or orphan drug designation for non-oncological UODs were identified. Furthermore, 29 ultra-orphan drugs for non-oncological diseases under development that have the potential of reaching the market by 2021 were found. Total budget impact over 10 years was estimated to be e15,660 and e4965 million for approved and pipeline ultra-orphan drugs, respectively (total: e20,625 million). Conclusion: The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.
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