NICE have published nine HST guidance, all with positive recommendations, a median of 20 months (range 7-38) after European MA. An additional 11 HST guidance are in development with MAs for a median of 14 months (range: 0-53) with six having draft guidance issued, all being "not recommended". Of the 20 HSTs with NICE guidance published/in-development, 16, 14, 8, 6, 2, and 6 were assessed by HAS, G-BA, NCPE, SMC, TLV, and ZIN, respectively. Of these, 22/30 (73%) and 7/22 (32%) of assessments made by clinical-effectiveness and cost-effectiveness HTA bodies received positive outcomes, respectively, with median delays between European MA and positive appraisal outcomes of 7 and 37 months, respectively. Conclusions: Although NICE HST appraisals have more positive recommendations and have faster time to recommendations following European MA than other costeffectiveness HTA bodies, time to positive recommendation is still substantially delayed compared to clinical-effectiveness HTA bodies. In 2018, a new SMC appraisal framework was introduced, whereby ultra-orphan therapies would be made available for $3-years while additional evidence is collected pending a final SMC appraisal. This could potentially prove a more suitable best-practice model.
Conclusions: Most medicines receiving an initial Orphan Designation in Europe maintain this at market authorization. For the ones that do not, this is due to withdrawal of the orphan designation by the sponsor, caused by the disease prevalence and the significant benefit criteria no longer being met. Although orphan drug legislation has incentivized drug development for rare diseases in Europe, most of these drugs received an earlier FDA approval and overcoming the frequently greater reimbursement/payer hurdle in Europe can further add to this access discrepancy.
Objectives: To incentivize innovation and investment, the US and EU have developed favorable regulations for orphan drugs. Given the low number of patients and the urgency to make treatments available, having robust data at launch is unlikely. As a result, markets such as Germany have revised HTA rules to provide increased leniency when reviewing orphan drugs for reimbursement decisions. Many approved orphan drugs have oncology indications. With this research, we wanted to determine if oncology orphan drugs can achieve higher prices with the same or lower quality of evidence as non-oncology orphan drugs. Methods: We built a database of all drugs launched in orphan indications in the US and Europe since 2012. The database tracks: the launch indication, follow-on indications, date of first approval, population size of each indication, the HTA assessment in France and Germany (as proxy for the quality of the evidence) and the price in France, Germany and the US. We conducted a data analysis by market using a linear regression model, with the prevalence-adjusted price (price per patient multiplied by expected population size) as a dependent variable and the quality of the evidence (measured via HTA ratings) as the independent variable. We controlled for all other variables. Results: From the analysis it emerges that in the FR and the UK, oncology orphan drugs have achieved similar prevalence-adjusted price points as nononcology orphan drugs despite less robust evidence or proven benefit at launch. In the US, the level of evidence at launch was not correlated with price. Conclusions: Markets that relay on evidence driven HTAs to negotiate price may be more lenient on orphan drugs approved for oncology indications. Non-oncology orphan drugs may need to generate more evidence at launch than oncology drugs to secure optimal price and market access in the EU.
was to define which criteria and HTA tools were applied in the decision-process on the health technology assessment process for high-cost drugs for rare genetic diseases and compare the Brazilian policies in the area with European Countries policies. Methods: All the processes carried out by CONITEC since its foundation in 2011 were evaluated those with medicines for rare genetic diseases selected, the outcomes of these processes (included in SUS or not included) and HTA tools applied. Results: Cost-benefit analysis and cost-effectiveness were the most frequent analysis applied. The indirect costs were not applied in the analysis and epidemiology was not central for the 'inclusion or not' decision. There were important differences between the evaluation processes in Brazil and in European countries. Conclusions: Despite the heterogeneity difficulties in establishing a health policy for each rare disease, it is important to create rational models to deal with this challenge. Definition of criteria and thresholds could be beneficial to the process.
Objectives: Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm of joints and tendon sheaths. The TGCT Observational Platform Project (TOPP) assessed the health-related quality of life (HRQOL) of patients with TGCT. Methods: TOPP is an observational prospective study conducted in EU and US on adult patients with an histolgically confirmed diagnosis of TGCT, enrolled between November 2016 and March 2019. Patients were followed up for two years. Patient demographics, disease and treatment history, health care resource utilization and patient reported outcomes were collected. Patient were stratified by severity (moderate versus severe) based on magnetic resonance imaging parameters determined by investigators. EQ-5D-5L questionnaires were administered to patients at baseline, 6 months Follow UP (FU), 18 months FU and 24 months FU. A baseline snapshot was taken from patients available for this analysis. Results: 183 patients were enrolled (median age: 44 years), of which 155 completed EQ-5D-5L. 34.2% of patients experienced moderate or severe disability in mobility, 23.8% had at least slight or moderate problems in self-care, 34.2% suffered moderate or severe problems performing usual activities, 45.8% had moderate or severe pain or discomfort, and 17.4% demonstrated moderate or severe anxiety or depression. Mean (SD) EQ-5D index score was 0.75 (0.21) and mean (SD) visual analogue scale (VAS) score was 69.1 (20.57). For patients with a moderate disease extent mean index score was 0.76 and VAS score was 70, while for patients with a severe disease extent, mean index score was 0.75 and VAS score was 68.7. EQ-5D-5L index score for TGCT patients varied from 0.62 in Germany to 0.84 in France and VAS score varied from 60.38 in Germany to 75.5 in US. Conclusions: Patients with TGCT in TOPP registry reported a relatively low HRQOL compared to the general population. No significant difference according to disease severity and country was observed.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.