Abstract: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 int… Show more
“…Similar to Canada, most 20 OECD countries with a socially funded health care system or universal health care do not have a national program for funding DRDs, except for the United Kingdom. Instead, many apply a “safety net” program or use modified decision criteria when their common drug review processes are insufficient for funding decisions for DRDs [ 5 ].…”
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.Electronic supplementary materialThe online version of this article (doi:10.1186/s12913-017-2310-4) contains supplementary material, which is available to authorized users.
“…Similar to Canada, most 20 OECD countries with a socially funded health care system or universal health care do not have a national program for funding DRDs, except for the United Kingdom. Instead, many apply a “safety net” program or use modified decision criteria when their common drug review processes are insufficient for funding decisions for DRDs [ 5 ].…”
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.Electronic supplementary materialThe online version of this article (doi:10.1186/s12913-017-2310-4) contains supplementary material, which is available to authorized users.
“…Similar to ICER's consideration of the type of health technology, ICER also considers modifications to its methods for assessing value for treatments of serious, ultra-rare disorders and high-impact single-administration or short-term therapies. Most often, HTA agencies make recommendations for rare disease drugs based on the same centralized national review process as other drugs 33 ; however, several HTA bodies have created separate mechanisms, adapted existing processes, 29,33 and/or added "safety nets" that allow for individual patient requests for access to medicines that do not receive a positive reimbursement requests. 33 These alternatives have arisen primarily because orphan drugs are not able to meet typical cost-effectiveness standards, including high costs and uncertainty in the clinical evidence.…”
Section: Type Of Medical Conditionmentioning
confidence: 99%
“…Most often, HTA agencies make recommendations for rare disease drugs based on the same centralized national review process as other drugs 33 ; however, several HTA bodies have created separate mechanisms, adapted existing processes, 29,33 and/or added "safety nets" that allow for individual patient requests for access to medicines that do not receive a positive reimbursement requests. 33 These alternatives have arisen primarily because orphan drugs are not able to meet typical cost-effectiveness standards, including high costs and uncertainty in the clinical evidence. 34,35 A study of 32 countries (mostly European) found that 41% of countries had a supplemental process for rare or ultra-rare disease treatments, and the countries were classified based on the extent to which these processes were integrated with standard HTA processes.…”
Deliberation — the bringing together of individuals with multiple perspectives, experiences, and values to critically examine evidence, share reasons and rationales, and reach a decision on a course of action — occurs at all stages of the health technology assessment (HTA) process.
The focus of this report is on deliberation at the committee appraisal stage of HTA.
The objectives are to review and conceptualize the evidence and key considerations for deliberative processes at the committee appraisal stage of HTA and the available guidance for assessing the quality of the deliberative process at this stage.
National and international practices for deliberative processes at the committee appraisal stage of HTA vary depending on contextual considerations (e.g., type of health technology and the medical condition being treated), the mandate of the HTA organization, health system and funding structures, and societal values.
Each aspect of the deliberative appraisal process should be rooted in the values of the health system and be principled, clear, transparent, purposeful, and reasoned.
Background
The patient voice is becoming increasingly prominent across all stages of therapeutic innovation. It pervades research domains from funding and recruitment, to translation, care, and support. Advances in genomic technologies have facilitated novel breakthrough therapies, whose global developments, regulatory approvals, and confined governmental subsidisations have stimulated renewed hope amongst rare disease patient organisations (RDPOs). With intensifying optimism characterising the therapeutic landscape, researcher-advocate partnerships have reached an inflexion point, at which stakeholders may evaluate their achievements and formulate frameworks for future refinement.
Main text
Through this narrative review, we surveyed relevant literature around the roles of RDPOs catering to the rare paediatric neurological disease community. Via available literature, we considered RDPO interactions within seven domains of therapeutic development: research grant funding, industry sponsorship, study recruitment, clinical care and support, patient-reported outcome measures, and research prioritisation. In doing so, we explored practical and ethical challenges, gaps in understanding, and future directions of inquiry. Current literature highlights the increasing significance of ethical and financial challenges to patient advocacy. Biomedical venture philanthropy is gaining momentum amongst RDPOs, whose small grants can incrementally assist laboratories in research, training, and pursuits of more substantial grants. However, RDPO seed funding may encounter long-term sustainability issues and difficulties in selecting appropriate research investments. Further challenges include advocate-industry collaborations, commercial biases, and unresolved controversies regarding orphan drug subsidisation. Beyond their financial interactions, RDPOs serve instrumental roles in project promotion, participant recruitment, biobank creation, and patient registry establishment. They are communication conduits between carers, patients, and other stakeholders, but their contributions may be susceptible to bias and unrealistic expectations.
Conclusion
Further insights into how RDPOs navigate practical and ethical challenges in therapeutic development may enhance cooperative efforts. They may also inform resources, whose distribution among advocates, parents, and clinicians, may assist decision-making processes around rare disease clinical trials and treatments.
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