The reimbursement decision speed for oncology new drugs in China and its determinant factors
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ABSTRACT:
SUBMITTER: Zhu X
PROVIDER: S-EPMC10643204 | biostudies-literature | 2023 Jan
REPOSITORIES: biostudies-literature
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Project description:IntroductionIndirect treatment comparisons (ITCs) evaluate novel treatments compared to appropriate comparators when direct evidence is unavailable or infeasible. The objective of this study was to highlight the prevalence of different ITC methods in oncology drug submissions and to provide insights into how ITCs have been used in recent regulatory approval, reimbursement recommendations, or pricing decisions across various regions and diverse assessment frameworks.MethodsA targeted literature review was conducted to identify assessment documents for oncology drug submissions that included ITCs. This included hand searches of the websites of four regulatory bodies and four health technology assessment (HTA) agencies with varying assessment frameworks across North America, Europe, and Asia-Pacific.ResultsA total of 185 documents were included for synthesis. Documents were retrieved from all four HTA agencies and the European Medicines Agency (EMA), the only regulatory body with eligible records. Within these, 188 unique submissions included a total of 306 supporting ITCs of various methods. Authorities more frequently favored anchored or population-adjusted ITC techniques for their effectiveness in data adjustment and bias mitigation. Furthermore, ITCs in orphan drug submissions more frequently led to positive decisions compared to non-orphan submissions.ConclusionsThis review highlights the crucial role and widespread use of ITCs in global healthcare decision-making, particularly when direct evidence is lacking, and in the discernment of market-specific clinical benefits. This work contributes to bolstering the credibility and recognition of ITCs across regulatory and HTA agencies of diverse regions and assessment frameworks.
| S-EPMC11782458 | biostudies-literature
Project description:Introduction: How the launch delay of drugs and other factors of interest can influence the length of the review period by drug agencies is still unknown, and understanding this can help better strike the trade-off related to review speed. Methods: We included all new oncology drug applications submitted to China's National Medical Product Administration (NMPA) between 1 January 2018 and 31 December 2021, and ultimately succeeded in achieving marketing approval. For each drug, the length of the NMPA review process and other major characteristics were collected, including the registration class, approval class, priority review designation, and launch delay relative to the United States, as well as the number of patients enrolled, comparator, and primary endpoint of the pivotal trials supporting the approval. Linear regression model was employed to analyze the effects of factors of interest on the NMPA review time. Results: From 2018 to 2021, NMPA received 137 oncology applications that were ultimately approved. Half of the approvals [76 (55.5%)] were first licensed in the US, leaving a median launch delay of 2.71 years (IQR, 1.03-5.59) in China. In the pivotal studies, the median enrollment was 361 participants (IQR, 131-682), and the use of control groups [90 (65.7%)] and surrogate endpoints [101 (73.7%)] was prevalent. The median review time was 304 days (IQR, 253-376). Multivariate analysis for log-transformed review time showed that larger enrollment (> 92) was associated with a drop of 20.55% in review time (coefficient = -0.230; 95% CI, -0.404 to -0.055; p = 0.010); and a short delay (0 < delay ≤ 1.95 years) was associated with a drop of 17.63% in review time (coefficient = -0.194; 95% CI, -0.325 to -0.062; p = 0.004). Discussion: The short launch delay relative to the US was one important driver to the review speed of NMPA, which might suggest its latent regulatory reliance on the other global regulator during the post-marketing period when new information on the drug's clinical benefit was still lacking.
| S-EPMC10654631 | biostudies-literature
Project description:BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or 'opportunity cost'-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.
| S-EPMC4342524 | biostudies-literature
Project description:Healthcare expenditure on pharmaceuticals, especially innovative oncology drugs, is escalating. Current knowledge on this topic is largely limited to studies conducted upon reimbursement of new drugs. We investigated how endogenous factors (e.g., changed reimbursement criteria, such as an expanded indication) and exogenous factors (e.g., competing drugs) affect the level and trends of innovative oncology drug utilization in the Taiwan National Health Insurance (NHI) system, both upon reimbursement and afterward. This retrospective longitudinal study analyzed monthly data (January 2009 to December 2014) from the NHI Research Database on the consumption (prescribing volume) of 15 innovative oncology drugs reimbursed by the NHI between 2007 and 2013. Effects of endogenous and exogenous factors on drug utilization were evaluated using interrupted time series analyses. In segmented regression analyses, changed drug prescribing volume after the indication expanded (endogenous factor) was statistically significant; however, drug volume did not change significantly after prescription restrictions changed. First-competitors and non-first-competitors (exogenous factors) were significantly associated with drug prescription levels or utilization rates. Taking sorafenib as an example, the post-reimbursement drug prescribing volume did not change significantly after its therapy line changed (endogenous factor), whereas the reimbursement of first-competitors (exogenous factor) was significantly associated with a lower level or usage rate of sorafenib. Utilization of innovative oncology drugs in Taiwan changed dramatically after NHI reimbursement, driven largely by expanded indications and new competitors. Drug utilization evaluations should investigate both endogenous and exogenous factors.
| S-EPMC7719368 | biostudies-literature
Project description:Public reimbursement systems face the challenge of balancing provision of needed treatments and the reality of limited resources. Canada has a complex system for drug approval and public reimbursement, with jurisdiction divided between the federal government and the provinces/territories. A pivotal role is that of health technology assessment (HTA), which relies primarily on health economic principles to analyze the value of drugs on a population health basis and make recommendations about public reimbursement. The Canadian Agency for Drugs and Technologies in Health (CADTH) provides recommendations to all provinces but Quebec. This article provides an overview of Canada's approval and public reimbursement pathway, including the role of HTA and the economic principles on which it relies. Starting in late 2020, CADTH reduced the cost per quality-adjusted life year (QALY) threshold, the metric relied upon in making recommendations to public payers. An analysis of all 56 oncology drug final recommendations issued from January 2020 to January 2022 was conducted and confirms this reduction in the cost per QALY threshold. As a result of this threshold reduction, recommendations to the provinces include, in a number of cases, substantially greater price reductions. The potential implications for successful price negotiation with the pan-Canadian Pharmaceutical Alliance (pCPA), the public negotiating body for the provinces, are discussed.
| S-EPMC8947453 | biostudies-literature
Project description:PurposeTo assess the reporting quality of published economic evaluations of the negotiated oncology drugs listed for China's 2020 National Reimbursement Drug List (NRDL).MethodsA comprehensive search was conducted to identify economic evaluation studies of negotiated oncology drugs listed in China's 2020 NRDL using the PubMed/MEDLINE, Embase, Web of Science, CNKI, SinoMed, and WanFang Database up to March 31, 2021. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist scored the reporting quality between 0 and 100. A linear regression analysis was employed to examine the influence of various characteristics on the reporting quality scores.ResultsEighty papers were included in the study, with the majority published during the past decade. Furthermore, more than half of the articles (57.5%, or 46 out of 80) were written in English. The average CHEERS score was 74.63 ± 12.75 and ranged from 43.48 to 93.75. The most inadequately reported items included choice of model, characterization of heterogeneity, and discussion, as well as currency, price date and conversion. Higher scores were associated with articles published from 2019 to 2021 and English publications.ConclusionThe economic evaluation studies of negotiated oncology drugs listed in 2020 NRDL had moderate reporting quality. The Chinese economic evaluation publications could improve the reporting quality if the CHEERS checklist is consistently implemented. Also, the Chinese journals maybe explore introducing a reporting standard for economic evaluations.
| S-EPMC9719239 | biostudies-literature
Project description:Background and objectivesMultiple reforms aimed at improving the Chinese population's health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends.MethodsTargeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs.ResultsDrug reimbursement in China is becoming increasingly centralized due to the removal of provincial pathways, the establishment of the National Healthcare Security Administration and the implementation of the National Reimbursement Drug List (NRDL), which is now the main route for drug reimbursement in China. There is also an increasing number of other channels via which patients may access innovative treatments, including various types of commercial insurance and special access. Health technology assessment (HTA) and health economic evidence are becoming pivotal elements of the NRDL decision-making process. Alongside the optimization of HTA decision making, innovative risk-sharing agreements are anticipated to be increasingly leveraged in the future to optimize access to highly specialized technologies and encourage innovation while safeguarding limited healthcare funds.ConclusionsDrug public reimbursement in China continues to align more closely with approaches widely used in Europe in terms of HTA, health economics and pricing. Centralization of decision-making processes for public reimbursement of innovative drugs allows consistency in assessment and access, which optimizes the improvement of the Chinese population's health.
| S-EPMC10266112 | biostudies-literature
Project description:BackgroundAnnual Chinese National negotiations for including innovative drugs in the National Reimbursement Drug List (NRDL) reveal an increasing number of new drugs with overlapping action mechanisms of action and similar indications. Yet, it is unclear if competition affects reimbursement decisions. Thus, we explored the impact of competition on reimbursement decisions for cancer drugs in China.MethodsWe identified the cancer drugs involved in NRDL negotiations from 2017 to 2022 and focused on the initial reimbursement decision for eligible newly negotiated drugs. Drugs were classified as within-class competitors based on their equivalent biological mechanisms of action and approved indications, including identified and potential competitors. Other variables included drug type, clinical benefit and safety, monthly drug cost, and disease incidence rate. We employed traditional univariate and multivariate Firth's penalized logistic regression to assess the association between reimbursement decisions and variables at the indication and drug levels.FindingsBetween 2017 and 2022, 102 cancer drugs corresponding to 141 indications were studied, and 66 drugs (64.7%) covering 95 indications (67.4%) were added to the NRDL. The proportion of reimbursements for indications with identified competition was significantly higher than that for indications without identified competition (84.6% vs 52.6%, p < 0.0001). However, the difference in reimbursement proportions between groups with and without potential competition was not statistically significant (66.7% vs 68.3%, p = 0.84). Firth's penalized logistic regression showed that identified competition was positively correlated with successful NRDL inclusion, whereas potential competition had no significant effect on negotiation outcomes. Improved overall survival or progression-free survival were positively associated with NRDL inclusion, whereas disease incidence negatively impacted reimbursement decisions.InterpretationImproved clinical benefit and identified competition were positively correlated with NRDL inclusion. In China's value-based negotiation model, clinical benefits served as a crucial foundation of price negotiation for cancer drugs, and market competition helped these drugs enter the NRDL at more reasonable prices. This has important implications for reimbursement decisions and accessibility and affordability improvement for innovative drugs worldwide.FundingNational Natural Science Foundation of China (No. 72104151).
| S-EPMC11326914 | biostudies-literature
Project description:IntroductionClinical decision-making in oncology is a complex process, with the primary goal of identifying the most effective treatment tailored to individual cancer patients. Many factors influence the treatment decision: disease- and patient-specific criteria, the increasingly complex treatment landscape, market authorization and drug availability, financial aspects, and personal treatment expertise. In the domain of genitourinary cancers, particularly prostate cancer, decision-making is challenging. Despite the prevalence of this malignancy, there are few in-depth explorations of these factors within real-world scenarios. Understanding and refining this intricate decision-making process is essential for future successful clinical decisions and the integration of computerized decision support into clinicians' workflows.AimThe objective of this study is to improve the current knowledge base and evidence of the factors that influence treatment decision-making for patients with genitourinary cancers.MethodsAssessment of how routine treatment decisions are made for genitourinary cancers was performed by a mixed-methods study, encompassing field observations and focus group discussions.ResultsIn total, we identified 59 factors that influence clinical decision-making in oncology, specifically for genitourinary and prostate cancer. Of these, 23 criteria can be classified as decision-maker-related criteria encompassing personal, cognitive, and emotional attributes and factors of both, healthcare professionals and patients. Moreover, 20 decision-specific criteria have been identified that refer to clinical and disease-related factors, followed by 16 contextual decision factors that describe the relevant criteria introduced by the specific circumstances and environment in which the treatment decision is made.ConclusionBy presenting an exhaustive set of decision factors and providing specific examples for genitourinary cancers, this observational study establishes a possible framework for a better understanding of decision-making. Moreover, we specify and expand the set of decision factors, while emphasizing the importance of cognitive, emotional, and human factors, as well as the quality and accessibility of decision-relevant information.
| S-EPMC11494402 | biostudies-literature
Project description:BackgroundFunding of drugs for rare diseases (DRDs) requires decisions that balance fairness for all individuals within the healthcare system with compassion for affected individuals. Our study objective was to conduct a national online survey to determine the Canadian public's perspective, including regional variations, associated with DRD decision-making.MethodsThe survey collected responses from 1631 Canadians. Respondents were asked to rank at least three and up to five DRD decision-making priorities, out of a total of eight priorities presented. They were also asked to compare and rate their agreement level on a 5-point Likert scale with four funding scenarios described. The frequency of each priority, independent of where it was ranked in relation to the other priorities, was calculated. Regression analyses were conducted to measure the association between respondents' demographics and selected priorities with their agreement level for each funding scenario.ResultsAmong the survey respondents, Improved Quality of Life and Effective Health Care were most frequently selected as top priorities. Also, 79.2% of respondents agreed with equal access to DRDs across Canada, and 73.0% agreed with DRD funding if additional expenses are justified in the DRD's cost-effectiveness. Approximately half agreed to pay for DRDs independent of their effectiveness. There were no geographic differences in priorities. Selecting Effective Health Care in the top priorities was positively associated with both prioritizing other programs over programs for rare diseases and DRD funding only if deemed as cost-effective. Respondents, who selected National Access as one of the top priorities, were less likely to agree to fund DRDs only if deemed as cost-effective and were more likely to agree with the scenario to provide national access to DRDs.ConclusionsThe survey results suggest the level of public support for funding decisions and programs that incorporate assessment of the effectiveness of drugs for improving quality of life, and to promote similar access across Canada. The responses anticipate public responses to different policy scenarios and the priorities that underlie them. Decision-makers may find it useful to consider whether and how to incorporate these results into policy decisions and their justification to citizens and patients.
| S-EPMC5446683 | biostudies-literature