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Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe. This paper elicited the opinions of European stakeholders on how HTA can be improved to facilitate access. Methods: A scoping review identified HTA features that influence access to medicines within markets and areas for improvement, while three access dimensions were identified (availability, affordability, timeliness). Using the Delphi method, we elicited the opinions of European stakeholders to validate the literature findings. Results: Nineteen participants from 14 countries participated in the Delphi panel. Thirteen HTA features that could be improved to optimise access to medicines in Europe were identified. Of these, 11 recorded a positive impact on at least one of the three access dimensions. HTA features had mostly a positive impact on timeliness and a less clear impact on affordability. ‘Early scientific advice’ and ‘clarity in evidentiary requirements’ showed a positive impact on all access dimensions. 'Established ways to deal with uncertainty during HTA’ could improve medicines’ availability and timeliness, while more ‘reliance on real-world evidence’ could expedite time to market access. Conclusions: Our results reiterate that increased transparency during HTA and the decision-making processes is essential; the use of and reliance on new evidence generation such as real-world evidence can optimise the availability of medicines; and better collaborations between regulatory institutions within and between countries are paramount for better access to medicines.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed disease associated with high mortality rates and the patient journey is characterized by increased complexities. Accurate and timely diagnosis and prompt initiation of disease-modifying treatment constitute the contemporary unmet need in ATTR-CM. ATTR-CM diagnosis is characterized by considerable delays and high rates of misdiagnosis. The majority of patients present themselves to primary care physicians, internists, and cardiologists, and many have undergone repeated medical evaluations before an accurate diagnosis has been made. The disease is diagnosed mainly after the development of heart failure symptoms, reflecting a long course of missed opportunities before diagnosis and disease-modifying treatment initiation. Early referral to experienced centers ensures prompt diagnosis and therapy. Early diagnosis, better care coordination, acceleration of digital transformation and reference networks, encouragement of patient engagement, and implementation of rare disease registries are the key pillars to improve the ATTR-CM patient pathway and achieve important benefits in ATTR-CM outcomes.

Objectives: Unlike other high-income countries, Canada has no national policy for drugs treating rare diseases (orphan drugs). Nevertheless, in 2022, the Canadian government committed to creating a national strategy to make access to these drugs more consistent. Our aim was to study whether recommendations made by the Canadian Agency for Drugs and Technology in Health (CADTH) translated into coverage decisions for orphan drugs in Ontario, the largest Canadian province. This study is the first to look at this question for orphan drugs, which are at the center of policy attention. Methods: We included 155 orphan drug-indication pairs approved and marketed in Canada between October 2002 and April 2022. Cohen's kappa was used to test the agreement across health technology assessment (HTA) recommendations and coverage decisions in Ontario. Logistic regression was used to test which factors, relevant to decision-makers, might be associated with funding in Ontario. Results: We found only fair agreement between CADTH's recommendations and coverage decisions in Ontario. Although a positive and statistically significant association between favorable HTA recommendations and coverage was found, more than half of the drugs with a negative HTA recommendation were available in Ontario, predominately through specialized funds. Successful pan-Canadian pricing negotiations were a strong predictor of coverage in Ontario. Conclusions: Despite efforts to harmonize access to drugs across Canada, considerable room for improvement remains. Introducing a national strategy for orphan drugs could help increase transparency, consistency, promote collaborations, and make access to orphan drugs a national priority.

Access to medicines treating rare diseases (‘orphan medicines’) has proven challenging due to high prices and clinical uncertainty. To optimise market access to these medicines, some healthcare systems are implementing specialised pathways and/or processes during marketing authorisation (MA) and/or health technology assessment (HTA). Comparing one setting where these medicines are classed as “orphan” (Scotland) to another where they considered “non-orphan” (Canada), this study aims to explore whether the presence of specialised pathways and processes at MA and HTA levels is associated with more favourable funding recommendations and faster time to market access. A matched sample of 116 medicine-indication pairs with MA approval from 2001 to 2019 in Europe and Canada was identified, and publicly available sources were used for data extraction. Descriptive statistics were used for data analysis. All medicines were commercially marketed in both countries, except one instance in Scotland. In Scotland, more orphan medicines (68.1%) had a favourable HTA recommendation than in Canada (60.4%), while Canada issued more negative HTA recommendations (20.7%) than Scotland (15.5%). Low levels of agreement on HTA recommendations and the main reasons driving recommendations were found between settings. In both countries, medicines with specialised MA approval were less likely to receive negative HTA recommendations than medicines with standard MA. Time to market access was faster in Canada than Scotland, though medicines with specialised MA approval had slower timelines than medicines with standard MA approval in both countries. However, it is unclear whether the presence of orphan designation and HTA specialised processes alone could result in favourable funding recommendations without accounting for other healthcare system-related factors and differences in the decision-making processes across settings. Holistic approaches and better alignment of evidentiary requirements across

Health technology assessment (HTA) systems across countries vary in the way they are set up, according to their role and based on how funding decisions are reached. Our objective was to study the characteristics of these systems and their likely impact on the funding of technologies undergoing HTA. Based on a literature review, we created a conceptual framework that captures key operating features of HTA systems. We used this framework to map current HTA activities across 32 countries in the European Union, the UK, Canada and Australia. Evidence was collected through a systematic search of competent authority websites and grey literature sources. Primary data collection through expert consultation validated our findings and further complemented the analysis. Sixty-three HTA bodies were identified. Most have a national scope (76%), are independent (73%), have an advisory role (52%), evaluate pharmaceuticals predominantly or exclusively (76%), assess health technologies based on their clinical and cost-effectiveness (73%) and involve various stakeholders as members of the HTA committee (94%) and/or through external consultation (76%). The majority of HTA outcomes are not legally binding (81%). Although all study countries implement HTA, the way it fits into decision-making, negotiation processes, and coverage and funding decisions differs significantly across countries. HTA is a dynamic and transformative process and there is a need for transparency to investigate whether evidence-based information influences coverage decisions.

In this paper we outline and compare pharmaceutical pricing policies for in-patent prescription pharma- ceuticals with emphasis on external reference pricing (ERP) in eleven countries across the Middle East and North Africa (MENA) region and explore possible improvements in their pricing systems. Primary and secondary evidence was used to inform our analysis. Comparative analysis of ERP systems across countries followed an analytical framework distilling ERP into twelve salient features, while ERP system performance was benchmarked against a framework of best practice principles across (a) objectives and scope, (b) administration and operations, (c) methods used, and (d) implementation. Results suggest that ERP is the dominant pricing method for in-patent pharmaceuticals. Although several good practice cases were identified, none of the eleven countries satisfy all best practice principles. ERP basket sizes vary sig- nificantly and are commonly composed using geographical proximity and low-price countries as criteria. Nine countries do not use the mean or median prices, but resort to using the lowest. Exchange rate fluc- tuations are routinely used to arrive at price reductions in local currency. Significant opportunities exist for MENA countries to develop their ERP regimes to achieve greater compliance with best practice princi- ples. Over the short-term, incremental changes could be implemented to several ERP salient features and can be achieved relatively easily, thereby enhancing the functionality and performance of national ERP systems. Countries in the region can also focus on the development of explicit value assessment systems, and minimize their dependence on ERP over the longer-term.

Background. External reference pricing (ERP) seeks to rationalize prices and contain costs using foreign prices as a reference for the determination of domestic prices and is often used as the starting point for the facilitation of negotiations between health authorities and pharmaceutical manufacturers. Methods. A systematic literature review was used to identify characteristics of ERP implementation across 29 countries. Primary data collection, in the form of surveys directed at key stakeholders, was also used to supplement data in instances where information received from the systematic literature review was outdated or minimal. Findings from the systematic literature review and primary evidence from key stakeholders were bench-marked against 14 best practice principles inherent to an optimal ERP system.Results. Significant heterogeneity in ERP implementation across countries was identified. Country basket size, pricing calculation, and frequency of price revisions varied between countries. Belgium, France, and South Africa were more likely to adhere to the best practice principles, whilst Bulgaria, Hungary, and Romania had the most instances of non-adherence.Conclusion. The observed heterogeneity has policy implications for governments including globally declining pharmaceutical prices, launch delays in lower income countries, reduced incentive for continued R&D, and reduced access to medicines. Overcoming this issue to ensure that ERP is beneficial to all stakeholders will require a focus on developing sustainable, transparent, simple, and stable systems using a set of key guidelines that should maximize the benefits of the pricing policy.

Background: External reference pricing (ERP) is widely used to regulate drug prices. Although the literature has largely focused on the impact of ERP on a number of policy endpoints and its impact from a geographical perspective, a comparative study drawing on evidence from different settings does not exist to date. Methods: A systematic literature review was conducted on pre-defined endpoints on the impact of ERP across countries, such as price stability, price convergence and launch delays. Expert consultation was undertaken to analyse whether or not the international implications of ERP are considered during its design. Results: 46 studies were included in the analysis. Across countries, ERP may cause launch delays, price instability and lead to price convergence. However, these effects cannot be solely attributed to ERP, as there may be other factors at play, such as the size and the GDP of a country and other regulations in place, which can trigger these effects or reduce their effect. Nevertheless, the nature of ERP facilitates these unintended consequences and directly links them to it. Despite these cross-country implications being well known to decision-makers, they are not necessarily considered during the design of ERP. Conclusions: As the effects of ERP as a stand alone policy are very difficult to isolate in the presence of other regulatory measures implemented within countries and the presence of other extrinsic factors across countries, our findings are inconclusive. Still, there is an unquestionable unmet need related to the design of ERP systems to attain a positive impact internationally.

An assessment of the way that 29 countries implement external reference pricing (ERP), which aims to contain medicine costs, using a systematic literature review-based process.

An assessment of the impact of external reference pricing (ERP) systems on important health system goals such as availability, affordability and diffusion/utilisation of pharmaceuticals; and an analysis of the impact that ERP systems have at the domestic and international levels, particularly considering their likely spillover effects.