Dataset Information

HTA decision-making for drugs for rare diseases: comparison of processes across countries.

ABSTRACT:

Introduction

Drugs for rare diseases (DRDs) offer important health benefits, but challenge traditional health technology assessment, reimbursement, and pricing processes due to limited effectiveness evidence. Recently, modified processes to address these challenges while improving patient access have been proposed in Canada. This review examined processes in 12 jurisdictions to develop recommendations for consideration during formal government-led multi-sectoral discussions currently taking place in Canada.

Methods

(i) A scoping review of DRD reimbursement processes, (ii) key informant interviews, (iii) a case study of evaluations for and the reimbursement status of a set of 7 DRDs, and (iv) a virtual, multi-stakeholder consultation retreat were conducted.

Results

Only NHS England has a process specifically for DRDs, while Italy, Scotland, and Australia have modified processes for eligible DRDs. Almost all consider economic evaluations, budget impact analyses, and patient-reported outcomes; but less than half accept surrogate measures. Disease severity, lack of alternatives, therapeutic value, quality of evidence, and value for money are factors used in all decision-making process; only NICE England uses a cost-effectiveness threshold. Budget impact is considered in all jurisdictions except Sweden. In Italy, France, Germany, Spain, and the United Kingdom, specific factors are considered for DRDs. However, in all jurisdictions opportunities for clinician/patient input are the same as those for other drugs. Of the 7 DRDs included in the case study, the number that received a positive reimbursement recommendation was highest in Germany and France, followed by Spain and Italy. No relationship between recommendation type and specific elements of the pricing and reimbursement process was found.

Conclusions

Based on the collective findings from all components of the project, seven recommendations for possible action in Canada are proposed. These focus on defining "appropriate access", determining when a "full" HTA may not be needed, improving coordination among stakeholder groups, developing a Canadian framework for Managed Access Plans, creating a pan-Canadian DRD/rare disease data infrastructure, genuine and continued engagement of patient groups and clinicians, and further research on different decision and financing options, including MAPs.

SUBMITTER: Stafinski T

PROVIDER: S-EPMC9264608 | biostudies-literature |

REPOSITORIES: biostudies-literature

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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.

Project description:INTRODUCTION:Regardless of the type of health system or payer, coverage decisions on drugs for rare diseases (DRDs) are challenging. While these drugs typically represent the only active treatment option for a progressive and/or life-threatening condition, evidence of clinical benefit is often limited because of small patient populations and the costs are high. Thus, decisions come with considerable uncertainty and risk. In Canada, interest in developing a pan-Canadian decision-making approach informed by international experiences exists. OBJECTIVE:To develop an inventory of existing policies and processes for making coverage decisions on DRDs around the world. METHODS:A systematic review of published and unpublished documents describing current policies and processes in the top 20 gross domestic product countries was conducted. Bibliographic databases, the Internet and government/health technology assessment organization websites in each country were searched. Two researchers independently extracted information and tabulated it to facilitate qualitative comparative analyses. Policy experts from each country were contacted and asked to review the information collected for accuracy and completeness. RESULTS:Almost all countries have multiple mechanisms through which coverage for a DRD may be sought. However, they typically begin with a review that follows the same process as drugs for more common conditions (i.e., the centralized review process), although specific submission requirements could differ (e.g., no need to submit a cost-effectiveness analysis). When drugs fail to receive a positive recommendation/decision, they are reconsidered by "safety net"-type programs. Eligibility criteria vary across countries, as do the decision options, which may be applied to individual patients or patient groups. CONCLUSIONS:With few exceptions, countries have not created separate centralized review processes for DRDs. Instead, they have modified components of existing mechanisms and added safety nets.

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