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1.
Objectives
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.Methods
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.Results
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.Conclusions
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. 相似文献2.
Background
It is unclear whether UK National Health Service (NHS) policies for orphan drugs, which permit funding of non–cost-effective treatments, reflect societal preferences.Methods
We conducted person trade-off (PTO) and discrete choice experiment (DCE) among 3950 adults selected to be representative of the UK general population. Experimental design was informed by surveys of patients affected by rare diseases, their caregivers, health care staff, and policymakers. Societal preferences were estimated in relation to treating a common disease, increases in waiting lists, or filling of vacant NHS posts. Results of the DCE were applied to recently licensed orphan drugs.Results
On the basis of equal cost, the majority of respondents to the PTO (54%; 95% confidence interval [CI] 50–59) chose to allocate funds equally between patients treated for rare diseases and those treated for common diseases, with 32% (95% CI 28–36) favoring treating rare diseases over treating common diseases (14%; 95% CI 11–17), which this reduced to 23% (95% CI 20–27) when rare disease treatments were 10 times more expensive. When framed differently, more respondents prioritized not increasing waiting list size (43%; 95% CI 39–48) than to treat rare disease patients (34%; 95% CI 30–38).Discussion
The DCE indicated a greater preference for treating a common disease over a rare disease. Respondents agreed with five of 12 positive appraisal recommendations for orphan drugs, even if their list price was higher, but preferred the NHS not to fund the remainder.Conclusions
The general public does not value rarity as a sufficient reason to justify special consideration for additional NHS funding of orphan drugs. This has implications regarding the appropriateness of operating higher thresholds of cost-effectiveness. 相似文献3.
Sandra Nestler-Parr Daria Korchagina Mondher Toumi Chris L. Pashos Christopher Blanchette Elizabeth Molsen Thomas Morel Steven Simoens Zoltán Kaló Ruediger Gatermann William Redekop 《Value in health》2018,21(5):493-500
Background
Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated.Objective
To develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges.Methods
Challenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search.Results
Research-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers.Conclusions
Identifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money. 相似文献4.
Background
The number of authorized orphan and non-orphan medicines for rare diseases has increased in Europe. Patient access to these medicines is affected by high costs, weak efficacy/safety evidence, and societal value. European health care systems must determine whether paying for expensive treatments for only a few patients is sustainable.Objectives
This study aimed to evaluate patient access to orphan and non-orphan medicines for rare diseases in 22 European countries during 2005 to 2014.Methods
Medicines for rare diseases from the Orphanet list, authorized during 2005 to 2014, were searched for in the IMS MIDAS Quarterly Sales Data, January 2005 – December 2014 (IQVIA, Danbury, CT). The following three measures were determined for each country: number of available medicines, median time to continuous use, and medicine expenditure. A medicine was considered available if uninterrupted sales within a 1-year period were detected.Results
From 2005 to 2014, 125 medicines were authorized and 112 were found in the search. Of those, between 70 (63%) and 102 (91%) were available in Germany, the United Kingdom, Italy, France, and the Scandinavian countries. These countries were also the fastest to enable continuous use (3–9 mo). Only 27% to 38% of authorized medicines were available in Greece, Ireland, Bulgaria, Romania, and Croatia, which took 1 to 2.6 years to begin continuous use. A country’s expenditure on medicines for rare diseases in 2014 ranged between €0.2 and €31.9/inhabitant.Conclusions
Patient access to medicines for rare diseases varies largely across Europe. Patients in Germany, Scandinavian countries, Switzerland, France, and the United Kingdom can access larger numbers of medicines in shorter time. 相似文献5.
Objectives
To identify challenges that affect the feasibility and rigor of economic models in rare diseases and strategies that manufacturers have employed in health technology assessment submissions to demonstrate the value of new orphan products that have limited study data.Methods
Targeted reviews of PubMed, the National Institute for Health and Care Excellence’s (NICE’s) Highly Specialised Technologies (HST), and the Scottish Medicines Consortium’s (SMC’s) ultra-orphan submissions were performed.Results
A total of 19 PubMed studies, 3 published NICE HSTs, and 11 ultra-orphan SMC submissions were eligible for inclusion. In rare diseases, a number of different factors may affect the model’s ability to comply with good practice recommendations. Many products for the treatment of rare diseases have an incomplete efficacy and safety profile at product launch. In addition, there is often limited available natural history and epidemiology data. Information on the direct and indirect cost burden of an orphan disease also may be limited, making it difficult to estimate the potential economic benefit of treatment. These challenges can prevent accurate estimation of a new product’s benefits in relation to costs. Approaches that can address such challenges include using patient and/or clinician feedback to inform model assumptions; data from disease analogues; epidemiological techniques, such as matching-adjusted indirect comparison; and long-term data collection.Conclusions
Modeling in rare diseases is often challenging; however, a number of approaches are available to support the development of model structures and the collation of input parameters and to manage uncertainty. 相似文献6.
Julia Walter Matthias Vogl Martin Holderried Christian Becker Alina Brandes Moritz F. Sinner Wolf Rogowski Jens Maschmann 《Value in health》2017,20(6):769-776
Objectives
To compare complication rates, length of hospital stay, and resulting costs between the use of manual compression and a vascular closing device (VCD) in both diagnostic and interventional catheterization in a German university hospital setting.Methods
A stratified analysis according to risk profiles was used to compare the risk of complications in a retrospective cross-sectional single-center study. Differences in costs and length of hospital stay were calculated using the recycled predictions method, based on regression coefficients from generalized linear models with gamma distribution. All models were adjusted for propensity score and possible confounders, such as age, sex, and comorbidities. The analysis was performed separately for diagnostic and interventional catheterization.Results
The unadjusted relative risk (RR) of complications was not significantly different in diagnostic catheterization when a VCD was used (RR = 0.70; 95% confidence interval [CI] 0.22–2.16) but significantly lower in interventional catheterization (RR = 0.44; 95% CI 0.21–0.93). Costs were on average €275 lower in the diagnostic group (95% CI ?€478.0 to ?€64.9; P = 0.006) and around €373 lower in the interventional group (95% CI ?€630.0 to ?€104.2; P = 0.014) when a VCD was used. The adjusted estimated average length of stay did not differ significantly between the use of a VCD and manual compression in both types of catheterization.Conclusions
In interventional catheterization, VCDs significantly reduced unadjusted complication rates, as well as costs. A significant reduction in costs also supports their usage in diagnostic catheterization on a larger scale. 相似文献7.
Patricia M. Danzon 《Value in health》2018,21(3):252-257
Objectives
To analyze how value-based pricing (VBP), which grounds the price paid for pharmaceuticals in their value, can manage “affordability” challenges, defined as drugs that meet cost-effectiveness thresholds but are “unaffordable” within the short-run budget.Methods
Three specific contexts are examined, drawing on recent experience. First, an effective new treatment for a chronic, progressive disease, such as hepatitis C, creates a budget spike that is transitory because initial prevalence is high, relative to current incidence. Second, “cures” that potentially provide lifetime benefits may claim abnormally high VBP prices, with high immediate budget impact potentially/partially offset by deferred cost savings. Third, although orphan drugs in principle target rare diseases, in aggregate they pose affordability concerns because of the growing number of orphan indications and increasingly high prices.Results
For mass diseases, the transitory budget impact of treating the accumulated patient stock can be managed by stratified rollout that delays treatment of stable patients and prioritizes patients at high risk of deterioration. Delay spreads the budget impact and permits potential savings from launch of competing treatments. For cures, installment payments contingent on outcomes could align payment flows and appropriately shift risk to producers. This approach, however, entails high administrative and incentive costs, especially if applied across multiple payers in the United States. For orphan drugs, the available evidence on research and development trends and returns argues against the need for a higher VBP threshold to incentivize research and development in orphan drugs, given existing statutory benefits under orphan drug legislation. 相似文献8.
9.
Background
Conditional reimbursement of new health technologies is increasingly considered as a useful policy instrument. It allows gathering more robust evidence regarding effectiveness and cost-effectiveness of new technologies without delaying market access. Nevertheless, the literature suggests that ending reimbursement and provision of a technology when it proves not to be effective or cost-effective in practice may be difficult.Objectives
To investigate how policymakers and the general public in the Netherlands value removing a previously reimbursed treatment from the basic benefits package relative to not including a new treatment.Methods
To investigate this issue, we used discrete-choice experiments. Mixed multinomial logit models were used to analyze the data. Compensating variation values and changes in probability of acceptance were calculated for withdrawal of reimbursement.Results
The results show that, ceteris paribus, both the general public (n = 1169) and policymakers (n = 90) prefer a treatment that is presently reimbursed over one that is presently not yet reimbursed.Conclusions
Apparently, ending reimbursement is more difficult than not starting reimbursement in the first place, both for policymakers and for the public. Loss aversion is one of the possible explanations for this result. Policymakers in health care need to be aware of this effect before engaging in conditional reimbursement schemes. 相似文献10.