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1.
Background
It has been suggested that differences in health technology assessment (HTA) processes among countries, particularly within Europe, have led to inequity in patient access to new medicines.Objectives
To provide an up-to-date snapshot analysis of the present status of HTA and reimbursement systems in select European countries, and to investigate the implications of these processes, especially with regard to delays in market and patient access.Methods
HTA and reimbursement processes were assessed through a review of published and gray literature, and through a series of interviews with HTA experts. To quantify the impact of differences among countries, we conducted case studies of 12 products introduced since 2009, including 10 cancer drugs.Results
In addition to the differences in HTA and reimbursement processes among countries, the influence of particular sources of information differs among HTA bodies. The variation in the time from the authorization by the European Medicines Agency to the publication of HTA decisions was considerable, both within and among countries, with a general lack of transparency as to why some assessments take longer than others. In most countries, market access for oncology products can occur outside the HTA process, with sales often preceding HTA decisions.Conclusions
It is challenging even for those with considerable personal experience in European HTA processes to establish what is really happening in market access for new drugs. We recommend that efforts should be directed toward improving transparency in HTA, which should, in turn, lead to more effective processes. 相似文献2.
Alessandra Ferrario 《Value in health》2018,21(7):809-821
Objectives
First, to quantify the median time from European Union (EU)-wide approval to first use (launch) for a sample of cancer medicines and number of launches in Belgium, Estonia, Scotland, and Sweden as of June 2015. Second, to assess whether longer times to launch or lack of launches affected medicines with high or low expected additional clinical benefit. Third, to identify possible determinants of the probability of a cancer medicine to be launched.Methods
Correlation between time to launch and a set of variables hypothesized to affect launch was tested using a complementary log-log model for a sample of 46 cancer medicines that obtained EU-wide marketing authorization between 2000 and 2014.Results
In median, for a sample of 24 cancer medicines that obtained marketing authorization between 2010 and 2014, the expected time from EU-wide marketing authorization to first use of a medicine was shortest in Sweden, 3.1 months, followed by Scotland (9.3 months), Belgium (14.8 months), and Estonia (27.8 months). Median times to launch were longer for the entire sample of 46 cancer medicines that obtained marketing authorization between 2000 and 2014. In the all-country model, medicines with shorter times to submission for reimbursement, local manufacturers headquarter (or local sales representative), and a Food and Drugs Administration priority review or a combination of expedited approval programs and medicines launched in Scotland and Sweden were associated with a higher hazard of launch. Longer times since EU-wide approval initially correlate with an increased hazard but as time further elapses they negatively affect the hazard of launch.Conclusions
Median times from marketing authorization to first use of cancer medicines were shorter for medicines launched between 2010 and 2014 versus sample-wide (2000–2014). In Estonia, more medicines than in the other countries were not yet launched at the end of the observation period. There was no correlation between Prescrire and European School of Medical Oncology Magnitude of Clinical Benefit Scale ratings of added clinical value and time to launch. 相似文献3.
Objectives
Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient access to ultra-orphan drugs between countries. Furthermore, we describe how reimbursed and non-reimbursed orphan drugs differ with respect to pharmacoeconomic properties.Methods
We studied patient access to eight high-priced inpatient ultra-orphan drugs in nine countries. In addition, we determined whether differences with respect to cost per patient, budget impact and cost-effectiveness existed between orphan drugs with a positive and negative reimbursement status.Results
Reimbursement status was available for 78 orphan drugs, of which 56 (72%) were positive. Large differences were observed between countries; while two countries had a positive status for two out of nine ultra-orphan drugs, four countries had positive status for all drugs it assessed. A number of drugs were reimbursed only after price negotiations and/or through specific orphan drug policies. The average cost per patient, budget impact and incremental cost-effectiveness ratios were lower for ultra-orphan drugs with a positive reimbursement status than for those with a negative status, although only cost-effectiveness ratios were statistically significant.Conclusions
Large differences in patient access to ultra-orphan drugs were observed between countries. Future research should examine if similar findings can be seen in other countries and with other orphan drugs, and it should also determine which other factors play a role in reimbursement status of orphan drugs. 相似文献4.
5.
Georg Isbary Thomas R. Staab Volker E. Amelung Charalabos-Markos Dintsios Christof Iking-Konert Sonja Mariotti Nesurini Miriam Walter Jörg Ruof 《Value in health》2018,21(6):698-706
Background
In oncology clinical trials, crossover is used frequently but may lead to uncertainties regarding treatment effects.Objective
To investigate the handling of evidence from crossover trials by the European Medicines Agency (EMA) and the German Federal Joint Committee (G-BA).Methods
For oncology medicines with early benefit assessments before January 2015, presence of crossover, clinical data, EMA requests for additional data, and G-BA benefit ratings/evidence levels were analyzed from manufacturers’ dossiers, G-BA appraisals, European Public Assessment Reports, and original publications.Results
Eleven of 21 benefit assessments included crossover trials. Significant intergroup differences (P < 0.05) in overall survival (OS) were noted in 7 of 11 trials with and 7 of 10 without crossover. For 6 of 11 medicines with crossover, these were demonstrated before crossover. Treatment effects generally worsened with increasing proportions of crossover. The EMA requested additional data more frequently if crossover was performed, particularly if no OS data were available before crossover. The G-BA granted a considerable benefit to 73% of medicines with crossover and 40% of those without. Evidence levels were intermediate for 50% and 75%, respectively. None of the medicines received the highest evidence level.Conclusions
In G-BA appraisals, oncology medicines with crossover received better additional benefit ratings, but were assigned lower evidence levels, than those without. The five medicines with crossover after progression were assigned lower evidence levels than the six medicines with crossover after demonstration of superior OS, indicating that the way in which crossover is implemented may be one factor influencing the assignment of evidence levels by the G-BA. 相似文献6.
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7.
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8.
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9.
Anna Essén Isabella Scandurra Reinie Gerrits Gayl Humphrey Monika Alise Johansen Patrick Kierkegaard Jani Koskinen Siaw-Teng Liaw Souad Odeh Peeter Ross Jessica S. Ancker 《Health Policy and Technology》2018,7(1):44-56
Objectives
Patient-accessible electronic health records (PAEHRs) are being implemented at international scale. Comparing policies and systems could allow countries to learn from each other to address global and nation-specific challenges. We compare national PAEHR policy (hard and soft regulation) and services in 10 countries.Methods
PAEHR policy and system documentation was gathered from Australia, Denmark, Estonia, Finland, France, the Netherlands, New Zealand, Norway, Sweden and the United States. A basic analytic model for policy analysis was used to delimit our focus to policy content, followed by an inductive thematic analysis across countries, in which we clustered initial themes into a set of categories of PAEHR service “approaches” related to three specific content areas.Results
Although all 10 countries ensured some patient rights to access medical records, policies and systems were highly variable, as were the technological processes arising from these. In particular, three policy areas showed great variability. Depending upon country of origin, a patient would encounter differences in: login procedures (security), access to own and other patients’ data during adolescence (user rights), and types of medical data made available to the patient (data sets).Conclusions
Individuals encounter very different access rights to their medical data depending on where they live. Countries may be able to develop improved policies by examining how other nations have solved common problems. Harmonizing policies is also an initial step likely to be needed before cross-national PAEHRs could be possible. 相似文献10.