Bone-anchored prostheses for transfemoral amputation: a systematic review of outcomes, complications, patient experiences, and cost-effectiveness.

Introduction: Bone-anchored prostheses (BAP) are an advanced reconstructive surgical approach for individuals who had transfemoral amputation and are unable to use the conventional socket-suspension systems for their prostheses. Access to this technology has been limited in part due to the lag between the start of a new procedure and the availability of evidence that is required before making decisions about widespread provision. This systematic review presents as a single resource up-to-date information on aspects most relevant to decision makers, i.e., clinical efficacy, safety parameters, patient experiences, and health economic outcomes of this technology. Methods: A systematic search of the literature was conducted by an information specialist in PubMed, MEDLINE, Embase, CINAHL, Cochrane Library, the Core Collection of Web of Science, CADTH's Grey Matters, and Google Scholar up until May 31, 2023. Peer-reviewed original research articles on the outcomes of clinical effectiveness (health-related quality of life, mobility, and prosthesis usage), complications and adverse events, patient experiences, and health economic outcomes were included. The quality of the studies was assessed using the Oxford Centre for Evidence-Based Medicine Levels of Evidence and ROBINS-I, as appropriate. Results: Fifty studies met the inclusion criteria, of which 12 were excluded. Thirty-eight studies were finally included in this review, of which 21 reported on clinical outcomes and complications, 9 case series and 1 cohort study focused specifically on complications and adverse events, and 2 and 5 qualitative studies reported on patient experience and health economic assessments, respectively. The most common study design is a single-arm trial (pre-/post-intervention design) with varying lengths of follow-up. Discussion: The clinical efficacy of this technology is evident in selected populations. Overall, patients reported increased health-related quality of life, mobility, and prosthesis usage post-intervention. The most common complication is a superficial or soft-tissue infection, and more serious complications are rare. Patient-reported experiences have generally been positive. Evidence indicates that bone-anchored implants for prosthesis fixation are cost-effective for those individuals who face significant challenges in using socket-suspension systems, although they may offer no additional advantage to those who are functioning well with their socket-suspended prostheses.

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

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.

Health Insurance Exemptions for Older Ghanaians: Stakeholder Perspectives on Challenges Confronting an Ambitious Policy.

The health and wellbeing of older adults have taken center-stage in global policy agendas in recent times. In 2003, Ghana introduced an insurance exemption policy to eliminate financial barriers to healthcare for older adults and other vulnerable population groups. Embedded within the National Health Insurance Scheme (NHIS), this policy ostensibly guarantees free healthcare for older adults at publicly-funded facilities across the country. In this paper, we applied the implementation problem framework to identify gaps in the implementation of the exemptions policy and their impact on the healthcare experiences of older adults. Our data collection involved qualitative interviews with a purposive sample of community-residing older adults, health workers, community leaders, and policymakers. Our thematic data analysis identified resource, substantive, bureaucratic, political, and administrative constraints in the policy implementation process which, in turn, affected quality healthcare delivery. While most of these constraints are general challenges confronting the NHIS, they serve to undermine the intent of the scheme's exemptions policy. In particular, despite the exemptions, older adults continued to pay out-of-pocket for certain categories of treatments and medications, creating as yet financial barriers to healthcare. We present policy recommendations for addressing these implementation challenges, including suggestions to decentralize, depoliticize, and financially liberate the operations of the NHIS.

Ghana's health insurance scheme has not eliminated financial barriers to healthcare for older adults.Older Ghanaians reported unmet healthcare needs, despite implementation of health insurance.Resource, substantive, bureaucratic, and political constraints impede the policy implementation process.Measures are needed to decentralize and depoliticize the NHIS implementation process.

Quality of life of older adults in two contrasting neighbourhoods in Accra, Ghana.

As is the case elsewhere in sub-Saharan Africa, Ghana is experiencing a rapid increase in the population of older adults. Despite their rising numbers, the living conditions and wellbeing of older Ghanaians remain woefully understudied. This paper presents the results of a study exploring the quality of life (QoL) of older adults in two contrasting neighbourhoods in Accra, Ghana. The objectives of the study were to: (1) explore and compare the QoL of older slum and non-slum dwellers in Ghana; and (2) determine the extent of QoL disparities between slum and non-slum older adults. To accomplish these objectives, we undertook a cross-sectional survey of older adults (N = 603) residing in a slum and non-slum neighbourhood. QoL was self-assessed in four domains - physical, psychological, social, and environment - using the World Health Organization (WHO) QoL assessment tool (WHOQoL-BREF). Multivariable linear regression analyses of the data revealed no statistically significant difference between the slum and non-slum respondents in physical (coeff: 0.5; 95% CI: -1.7, 2.8; p = 0.642) and psychological (coeff: -0.2; 95% CI: -3.0, 2.6; p = 0.893) QoL. However, the slum respondents reported significantly higher social QoL than the non-slum respondents (coeff: -3.2; 95% CI: -5.6, -0.8; p = 0.010), while the reverse was true in environmental QoL (coeff: 4.2; 95% CI: 2.3, 6.2; p < 0.001). The existence of strong social support systems in the slum and better housing and neighbourhood environmental conditions in the non-slum may have accounted for the observed variation in social and environmental QoL. Thus, contrary to popular discourses that vilify slums as health-damaging milieus, these findings offer a more nuanced picture, and suggest that some features of slums may constitute important health resources for older adults.

Health, quality of life, and wellbeing of older slum dwellers in sub-Saharan Africa: A scoping review.

A growing population of older adults resides in sub-Saharan Africa's urban slums. Although environmental conditions in slums are adversarial, there is limited knowledge on the wellbeing of older adults residing in these settings. This review sought to understand the scope, extent, and nature of current research on the health and wellbeing of older slum dwellers in sub-Saharan Africa. We searched 8 bibliographic databases for studies examining the health and wellbeing of older slum dwellers in the region. We also completed a grey literature search. The literature search together yielded 3,388 records, of which we selected 25 for review following a rigorous screening process. The included studies covered a variety of health issues of concern to older slum dwellers: (1) disease and injury prevalence; (2) self-assessed health and quality of life status; (3) physical/mental health impairment and disability; (4) healthcare access and utilisation; and (5) sociodemographic disparities in health and wellbeing. The gaps in this literature include a regional bias in research efforts, near absence of non-slum control samples, and limited research on the health impacts of the built environment of slums. Suggestions for future research are presented.

Capturing the Impact of Patient Portals Based on the Quadruple Aim and Benefits Evaluation Frameworks: Scoping Review.

BACKGROUND: Despite extensive and continuing research in the area of patient portals, measuring the impact of patient portals remains a convoluted process. OBJECTIVE: This study aims to explore what is known about patient portal evaluations and to provide recommendations for future endeavors. The focus is on mapping the measures used to assess the impact of patient portals on the dimensions of the Quadruple Aim (QA) framework and the Canada Health Infoway's Benefits Evaluation (BE) framework. METHODS: A scoping review was conducted using the methodological framework of Arksey and O'Malley. Reporting was guided by the PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) extension for scoping reviews. A systematic and comprehensive search was conducted using the Ovid platform, and the following databases were searched: Ovid MEDLINE (R) ALL (including epub ahead of print, in-process, and other nonindexed citations), EMBASE, and PsycINFO. CINAHL on the EBSCO platform and Web of Science were searched for studies published between March 2015 and June 2020. A systematic gray literature search was conducted using the Google search engine. Extracted data were tabulated based on a coding template developed to categorize the literature into themes and areas of interest. RESULTS: A total of 96 studies were included for data extraction. The studies were categorized based on the QA dimensions, with strict adherence to the definitions for each dimension. From the patients' perspective, it was determined that most evaluations focused on benefits and barriers to access, access to test results, medication adherence, condition management, medical notes, and secure messaging. From the population perspective, the evaluations focused on the increase in population outreach, decrease in disparities related to access to care services, and improvement in quality of care. From the health care workforce perspective, the evaluations focused on the impact of patients accessing medical records, impact on workflow, impact of bidirectional secure messaging, and virtual care. From the health system perspective, the evaluations focused on decreases in no-show appointments, impact on office visits and telephone calls, impact on admission and readmission rates and emergency department visits, and impact on health care use. Overall, 77 peer-reviewed studies were mapped on the expanded version of the BE framework. The mapping was performed using subdimensions to create a more precise representation of the areas that are currently explored when studying patient portals. Most of the studies evaluated more than one subdimension. CONCLUSIONS: The QA and BE frameworks provide guidance in identifying gaps in the current literature by providing a way to show how an impact was assessed. This study highlights the need to appropriately plan how the impact will be assessed and how the findings will be translated into effective adaptations.

The rationale and design of public involvement in health-funding decision making: focus groups with the Canadian public.

BACKGROUND: Worldwide, governments employ health technology assessment (HTA) in healthcare funding decision making. Requests to include public perspectives in this are increasing, with the idea being that the public can identify social values to guide policy development, increasing the transparency and accountability of government decision making. OBJECTIVE: To understand the perspectives of the Canadian public on the rationale and design of public involvement in HTA. DESIGN: A demographically representative sample of residents of a Canadian province was selected to take part in two sets of two focus groups (sixteen people for the first set and twenty for the second set). RESULTS: Participants were suspicious of the interests driving various stakeholders involved in HTA. They saw the public as uniquely impartial though also lacking knowledge about health technologies. Participants were also suspicious of personal biases and commended mechanisms to reduce their impact. Participants suggested various involvement methods, such as focus groups, citizens' juries and surveys, noting advantages and disadvantages belonging to each and commending a combination. DISCUSSION AND CONCLUSIONS: We identified a lack of public understanding of how decisions are made and distrust concerning whose interests and values are being considered. Public involvement was seen as a way of providing information to the public and ascertaining their views and values. Participants suggested that public involvement should employ a mixed-methods strategy to support informed debate and participation of a large number of people.

Understanding Canadian Health Technology Assessment through a systems lens.

OBJECTIVE: Governments around the world face challenges in maintaining sustainable, high-quality healthcare systems. Health Technology Assessment (HTA) is widely used as a method to assist in funding decisions. However, the scope and influence of HTA is still limited. We examined how policymakers can improve the usefulness of HTA. METHODS: We used Systems Thinking as a theoretical framework to examine HTA as a system. We purposely sampled stakeholders involved in Canadian HTA at a national or provincial level. We conducted 22 semi-structured interviews in September-December 2016. Data were analysed using NVivo10 and findings are presented as a concept map with explanatory text. FINDINGS: The HTA system is influenced by stakeholder interactions. Such interactions are, in turn, affected by stakeholders' worldviews and environmental factors. Stakeholders' worldviews includes individual's or groups' values and affect the exchange of information, and interpretation of events. External factors, such as changes to government structures, also affect the system. CONCLUSION: Most stakeholder groups are supportive of the system. However, participants identified a need for change, though the exact changes being recommended differed. Some interactions were praised (formal, inclusive collaborations to provide government with policy guidance on both broad and technology-specific matters), while other interactions were criticised (two-party alliances formed around purposes other than the common good, and lacklustre patient and industry engagement on the part of provincial government).

Defining the role of the public in Health Technology Assessment (HTA) and HTA-informed decision-making processes.

OBJECTIVES: The terminology used to describe community participation in Health Technology Assessment (HTA) is contested and frequently confusing. The terms patients, consumers, public, lay members, customers, users, citizens, and others have been variously used, sometimes interchangeably. Clarity in the use of terms and goals for including the different groups is needed to mitigate existing inconsistencies in the application of patient and public involvement (PPI) across HTA processes around the world. METHODS: We drew from a range of literature sources in order to conceptualize (i) an operational definition for the "public" and other stakeholders in the context of HTA and (ii) possible goals for their involvement. Draft definitions were tested and refined in an iterative consensus-building process with stakeholders from around the world. RESULTS: The goals, terminology, interests, and roles for PPI in HTA processes were clarified. The research provides rationales for why the role of the public should be distinguished from that of patients, their families, and caregivers. A definition for the public in the context of HTA was developed: A community member who holds the public interest and has no commercial, personal, or professional interest in the HTA process. CONCLUSIONS: There are two distinct aspects to the interests held by the public which should be explicitly included in the HTA process: the first lies in ensuring democratic accountability and the second in recognising the importance of including public values in decision making.

The Introduction of New Non-Drug Health Technologies into Canadian Healthcare Institutions: Opportunities and Challenges.

INTRODUCTION: A recent pan-Canadian survey of 48 health organizations concluded that structures, processes, factors and information used to support funding decisions on new non-drug health technologies (NDTs) vary within and across jurisdictions in Canada. METHODS: A self-administered survey was used to determine demographic and financial characteristics of organizations, followed by in-depth interviews with senior leadership of consenting organizations to understand the processes for making funding decisions on NDTs. RESULTS: Seventy-three and 48 organizations completed self-administered surveys and telephone interviews, respectively (with 45 participating in both ways). Fifty-five different processes were identified, the majority of which addressed capital equipment. Most involved multidisciplinary committees (with medical and non-medical representation), but the types of information used to inform deliberations varied. Across all processes, decision-making criteria included local considerations such as alignment with organizational priorities. CONCLUSIONS: NDT decision-making processes vary in complexity, depending on characteristics of the healthcare organization and context.

Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada.

BACKGROUND: In Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). While the agency specifies information requirements for the review of drug submissions, how that information is used by each process to formulate final reimbursement recommendations, particularly on drugs for rare diseases (DRDs) in which per patient treatment costs are often high, is unclear. The purpose of this study was to determine which factors contribute to recommendation type for DRDs. METHODS: Information was extracted from CDR and pCODR recommendations on drugs for diseases with a prevalence < 1 in 2000 from January 2012 to April 2018. Data were tabulated and multiple logistic regression was applied to explore the association between recommendation type and the following factors: condition/review process (cancer vs non-cancer), year, prevalence, clinical effectiveness (improvements in surrogate, clinical and patient reported outcomes), safety, quality of evidence (availability of comparative data, consistency between population in trial and indication, and bias), clinical need, treatment cost, and incremental cost-effective ratio (ICER). Two-way interactions were also explored. RESULTS: A total of 103 recommendations were included. Eleven were resubmissions, all of which received a positive recommendation. Among new submissions (n = 92), DRDs that were safe or offered improvements in clinical or patient reported outcomes were more likely to receive positive reimbursement recommendations. No associations between recommendation type and daily treatment cost, cost-effectiveness, or condition (cancer or non-cancer) were found. CONCLUSIONS: Clinical effectiveness, as opposed to economic considerations or whether the drug is indicated for cancer or non-cancer, determine the type of reimbursement recommendation.

A checklist for managed access programmes for reimbursement co-designed by Canadian patients and caregivers.

INTRODUCTION: Reimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does the risk of making a wrong decision, where harms outweigh benefits. Consequently, patients often face limited access to orphan drugs. Managed access programmes (MAPs) are a mechanism for managing risk while enabling access to potentially beneficial drugs. Patients and their caregivers have expressed support for these programmes and see patient input as critical to successful implementation. However, they have yet to be systematically involved in their design. OBJECTIVE: The aim of this study was to co-design with patients and caregivers a tool for the development of managed access programmes. METHODS: Building upon established relationships with the Canadian Organization for Rare Disorders, the project team collaborated with patients and caregivers using the principles of participatory action research. Data were collected at two workshops and analysed using a thematic network approach. RESULTS: Patients and caregivers co-designed a checklist comprised of six aspects of an ideal MAP relating to accountability (programme goals); governance (MAP-specific committee oversight, patient input, international collaboration); and evidence collection (outcome measures and continuation criteria, on-going monitoring and registries). They recognized that health-care resources are finite and considered disease or drug eligibility criteria for deciding when to use a MAP (eg drugs treating diseases for which there are no other legitimate alternatives). CONCLUSIONS: A patient and caregiver-designed checklist was created, which emphasized patient involvement and transparency. Further research is needed to examine the feasibility of this checklist and roles for other stakeholders.

Palliative care in the home: a scoping review of study quality, primary outcomes, and thematic component analysis.

BACKGROUND: The aim of palliative care is to improve the quality of life of patients and families through the prevention and relief of suffering. Frequently, patients may choose to receive palliative care in the home. The objective of this paper is to summarize the quality and primary outcomes measured within the palliative care in the home literature. This will synthesize the current state of the literature and inform future work. METHODS: A scoping review was completed using PRISMA guidelines. PubMed, Embase, CINAHL, Web of Science, Cochrane Library, EconLit, PsycINFO, Centre for Reviews and Dissemination, Database of Abstracts of Reviews of Effects, and National Health Service Economic Evaluation Database were searched from inception to August 2016. Inclusion criteria included: 1) care was provided in the "home of the patient" as defined by the study, 2) outcomes were reported, and 3) reported original data. Thematic component analysis was completed to categorize interventions. RESULTS: Fifty-three studies formed the final data set. The literature varied extensively. Five themes were identified: accessibility of healthcare, caregiver support, individualized patient centered care, multidisciplinary care provision, and quality improvement. Primary outcomes were resource use, symptom burden, quality of life, satisfaction, caregiver distress, place of death, cost analysis, or described experiences. The majority of studies were of moderate or unclear quality. CONCLUSIONS: There is robust literature of varying quality, assessing different components of palliative care in the home interventions, and measuring different outcomes. To be meaningful to patients, these interventions need to be consistently evaluated with outcomes that matter to patients. Future research could focus on reaching a consensus for outcomes to evaluate palliative care in the home interventions.

Assisted Reproductive Technologies in Alberta: An Economic Analysis to Inform Policy Decision-Making.

OBJECTIVE: Regulation and public funding of assisted reproductive technologies (ARTs) vary across the Canadian provinces. In Alberta, neither of these exists. We conducted this study to evaluate the cost effectiveness and budget impact of providing ARTs in Alberta under three different policy scenarios (a "restrictive" policy, a policy based on Quebec's model, and a "permissive" policy) in comparison with the status quo. METHODS: To predict the cost effectiveness and budget impact of three policy options for publicly funded ARTs in Alberta, we developed an economic model by combining a state transition Markov model and a decision tree. The primary outcome was cost per healthy singleton. Probabilistic and one-way sensitivity analyses were conducted. RESULTS: The restrictive policy was the most cost effective option for two subgroups of age (< 35 years and 35 to 39 years), while the Quebec policy option was most cost-effective for the ≥ 40 years subgroup. Budget impact analysis extending up to the age of 18 years for the children in the model showed the cost savings of $8.33 million for the restrictive policy for the < 35 years subgroup. For the ≥ 40 years subgroup, the Quebec policy option resulted in total cost savings of $3.75 million. Sensitivity analyses showed that the model results were robust. CONCLUSION: This economic modelling study shows that publicly funded and scientifically regulated ARTs could provide treatment access and save health care expenditures for the province.

Objectif : La réglementation et le financement public des techniques de procréation assistée (TPA) varient d'une province canadienne à l'autre. En Alberta, les TPA ne sont ni réglementées ni financées par les deniers publics. Nous avons mené cette étude dans le but d'évaluer la rentabilité de l'offre de TPA en Alberta et les effets d'une telle mesure sur le budget albertain en fonction de trois scénarios de politique différents (une politique « restrictive ¼, une politique fondée sur le modèle québécois et une politique « permissive ¼), par comparaison avec le statu quo. Méthodes : Pour prédire la rentabilité de ces trois options de politique (prévoyant l'offre de TPA financées par les deniers publics en Alberta) et leurs effets sur le budget provincial, nous avons élaboré un modèle économique en combinant un modèle Markov (transitions d'état) et un arbre décisionnel. Le coût par nouveau-né en santé issu d'une grossesse monofœtale constituait le critère d'évaluation principal. Des analyses simples de la variance en matière de sensibilité et des analyses probabilistes ont été menées. Résultats : La politique « restrictive ¼ a constitué l'option la plus rentable dans deux sous-groupes d'âge (< 35 ans et 35-39 ans), tandis que la politique fondée sur l'approche québécoise a constitué l'option la plus rentable dans le sous-groupe des ≥ 40 ans. L'analyse des effets sur le budget (jusqu'à ce que les enfants générés par le modèle ait atteint l'âge de 18 ans) a indiqué l'obtention d'économies de 8,33 millions de dollars pour ce qui est de la politique « restrictive ¼ dans le sous-groupe des < 35 ans. Dans le sous-groupe des ≥ 40 ans, l'option de la politique fondée sur l'approche québécoise a mené à l'obtention d'économies totales de 3,75 millions de dollars. Les analyses de la sensibilité ont indiqué que les résultats modélisés étaient robustes. Conclusion : Cette étude de modélisation économique indique que l'offre de TPA financées par les deniers publics et faisant l'objet d'une réglementation scientifique pourrait assurer l'accès au traitement et permettre l'obtention d'économies pour la province.

DEFINING ELIGIBILITY CRITERIA FOR FUNDING POLICIES AROUND IN VITRO FERTILIZATION.

OBJECTIVES: This review aims to assess the state of the science around the potential impact of certain patient characteristics on the safety and effectiveness of in vitro fertilization (IVF). METHODS: Following Cochrane Collaboration guidelines and the PRISMA statement, a comprehensive systematic review of reviews and recent primary studies examining the impact of paternal age and maternal age, smoking, and body mass index (BMI) on the safety and effectiveness of IVF was performed. Papers, published between January 2007 and June 2014, were independently reviewed and critically appraised by two researchers using published quality assessment tools for reviews and primary studies. Due to heterogeneity across papers (different study designs and patient selection criteria), a qualitative analysis of extracted information was performed. RESULTS: Seventeen papers (ten systematic reviews and seven primary studies) were included. They comprised evidence from retrospective observational studies in which maternal age, BMI, and smoking status were explored as part of secondary analyses of larger studies. The majority of papers found that the likelihood of achieving a pregnancy was lower among women who were >40 years, had a BMI ≥ 25 and smoked. Advanced maternal age and BMI were also associated with higher rates of preterm birth and low birth weight. CONCLUSIONS: Based on available evidence, it may be appropriate to consider "maternal age" and "morbid obesity" in public funding policies that aim to maximize the effectiveness of IVF. However, given inconsistencies in the effect of smoking across different pregnancy-related outcomes, support for incorporating it into funding conditions appears weak.

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

INTRODUCTION: Over the past 20 years, the number of therapies developed for rare diseases has rapidly increased. Often, these therapies represent the only active treatment for debilitating and/or life-threatening conditions. However, they create significant challenges for public and private payers. Because they target small patient populations, clinical evidence of efficacy/effectiveness is typically limited, while the cost per patient is high. In Canada, each province/territory establishes its own mechanisms for determining which drugs for rare diseases (DRDs) to provide. OBJECTIVES: To compare current mechanisms across provinces and territories, and explore their impact on access. METHODS: A systematic review of relevant published and unpublished documents was performed. Electronic bibliographic databases, the internet, and government websites were scanned using structured search strategies. Information was extracted independently by two researchers, and included aspects such as program type, condition/patient/therapy eligibility criteria, role of health technology assessment (HTA), decision options, ethical assumptions, and stakeholder input. It was validated through member-checking with provincial/territorial policy experts and tabulated to facilitate qualitative analyses. Impact on access was assessed through a cross-province/territory comparison of the coverage status of all non-cancer therapies reviewed by the Common Drug Review for indications affecting <1/2,000 Canadians using the Kappa statistic. Reasons for variations were explored using qualitative techniques. RESULTS: Each province/territory has formal and informal mechanisms through which such therapies may be accessed. In most cases, formal mechanisms constitute the centralized HTA processes that also apply to common therapies. While several provinces have established dedicated processes/programs, whether they have affected access is not clear. Despite broadly comparable approaches, there is less than perfect agreement on publicly funded DRDs across jurisdictions. CONCLUSIONS: Individual jurisdictions have developed different approaches to providing access to these therapies. However, as the number increases, a more systematic approach to decision-making may be needed.

A National Approach to Reimbursement Decision-Making on Drugs for Rare Diseases in Canada? Insights from Across the Ponds.

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.

Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.

BACKGROUND: The 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. OBJECTIVES: The 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. METHODS: A 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. RESULTS: The 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. CONCLUSION: Our 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.

An international survey of assisted reproductive technologies (ARTs) policies and the effects of these policies on costs, utilization, and health outcomes.

OBJECTIVES: The purpose of this study was to compare existing policies regarding access to 7 assisted reproductive technologies (ARTs) and to examine the effects of these policies on costs, utilization, and health outcomes in 14 countries and 2 Canadian provinces based on publicly available information. METHODS: A systematic review of publicly available information from peer-reviewed literature (using biomedical and social science databases) and grey literature (e.g., health ministry websites, health technology assessment agency websites, etc.) was performed. RESULTS: ARTs services permitted or funded vary across jurisdictions. The goals and eligibility restrictions (e.g., on maternal age, number of embryos transferred, and number of cycles) defined in ARTs policies also vary across jurisdictions. Studies on the impact of such policies have primarily been retrospective and focused on short-term service utilization. Nevertheless, they suggest that the policies have achieved specific outcomes, such as reductions in multiple births and in costs to payers. CONCLUSIONS: Based on the evidence reviewed, policies aimed at reducing public coverage of ARTs or restricting the number of embryos transferred have achieved these goals. However, their effects on maternal and infant health outcomes have not been well explored.