The role of health technology assessment on pharmaceutical reimbursement in selected middle-income countries

ObjectiveMiddle-income countries are often referred to as developing or emerging economies and face multiple challenges of severe financial stresses in their health care sectors, and high disease burden. The objective of this study is to provide an overview of how health technology assessment (HTA) is used and organized in selected middle-income countries and its role in the process of pharmaceutical coverage.MethodsWe selected middle-income countries where HTA activities are evident: Argentina, Brazil, China, Colombia, Israel, Mexico, Philippines, Korea, Taiwan, Thailand, and Turkey. We collected and reviewed relevant information to describe the health care and reimbursement systems and how HTA relates to coverage decision-making of pharmaceuticals. This was supplemented by information from a structured survey among professionals working in public and private health insurance, industry, regulatory authorities, ministries of health, academic units or HTA.ResultsAll countries require market authorization for pharmaceuticals to be sold and most countries have a national plan defining which pharmaceuticals can be reimbursed. However, the use of HTA in reimbursement decisions is still in its early stages with varying levels of HTA guidance implementation.ConclusionsThe study provides evidence of the development of HTA in coverage decision-making in middle-income countries. Increased health care spending and the resulting access to modern technology give a strong impetus to HTA. However, HTA is developing with uneven speed in middle-income countries and many countries are building on the organisational and methodological experience from established HTA agencies.     

Shedding Light on Reimbursement Policies of Companion Diagnostics in European Countries

ObjectivesEnsuring access to precision medicine has been an issue because in some European countries, desynchronized reimbursement decision-making occurs between the medicine and the companion diagnostic (CDx). This has resulted in cases in which precision medicine is reimbursed but not the CDx. In overcoming this issue, an alignment of the decision-making process for reimbursement between the 2 entities should be considered. As pharmaceutical reimbursement procedures are meticulously covered in the literature, we set out to systematically map in vitro diagnostic (IVD) reimbursement procedures and identify policies for aligning these procedures with the pharmaceutical reimbursement procedures.MethodsWe selected 8 European countries for this analysis. For each country, we characterized the national benefit basket entailing the IVD medical acts in outpatient care, evaluated the procedure for inclusion, and identified alternative reimbursement practices for CDx. Targeted searches, using publicly accessible sources, were conducted to identify relevant reimbursement policies and laws.ResultsWe systematically describe the reimbursement process in 8 European countries. Alternative procedures for CDx reimbursement were identified in Belgium and Germany. Alternative policies attributed to the practice of precision medicine were identified in England and Italy. In France, some CDx are included in the “coverage with evidence” development program. Specifically, the health technology assessment agencies of France and England commented on the assessment of companion diagnostics and their clinical utility.ConclusionCDx reimbursement procedures have recently been implemented in some countries. This was seemingly done primarily to ensure access to the precision medicine and only secondary to the value they would provide.     

Establishing a national HTA program for medical devices in Italy: Overhauling a fragmented system to ensure value and equal access to new medical technologies

Differing contexts have greatly influenced HTA development in various countries, with considerable effort recently made by international HTA networks (e.g., EUnetHTA) and the European Union (EU) to make HTA a more coherent, equal, and efficient process. Medical devices (MDs) present particular challenges for HTA because of frequent, rapid innovation, outcomes influenced by end-user competence, dynamic pricing and often low-quality scientific evidence. Our objective is to describe the development, structure and governance of a National HTA Program for MDs (PNHTADM) in Italy, a highly participatory, stakeholder-engaged, evidence-based process to reform a fragmented system of appraisal and approval. Based largely on EUnetHTA methods, the resulting process delineates a standardized system for proposing MDs by any stakeholders, accrediting HTA producers, setting criteria for prioritization and appraisals, and innovatively linking recommendations with coverage, reimbursement and procurement of MDs. Expected benefits include reduced disparities in pricing and reimbursement policies and improved access to new technologies across 21 regional healthcare systems in Italy's decentralized, universal system, complete with provisions to require additional evidence collection and centrally monitor diffusion. Though devised for Italy, the design, resources and underlying analysis provide a framework for other nations seeking to consolidate HTA initiatives, particularly in light of new EU regulation.     

Impact of health technology assessment implementation with a special focus on middle-income countries

ObjectivesBuilding an efficient health technology assessment (HTA) system requires significant effort and political commitment, in addition to human and financial resources. Expectations of what HTA can offer to middle-income countries (MICs) are continuously rising, which drives health policymakers to raise the question of whether HTA could help secure the financial sustainability needed to implement universal health coverage. In this study, we explored the impact HTA adoption may have on the countries and its impact on health system objectives, as well as transferability of benefits and drawbacks observed in higher-income to middle-income countries.MethodsWe utilized secondary data from a systematic literature review and primary data by disseminating a survey among local stakeholders in three MICs across three continents to capture their perspective on the impact of HTA implementation from a local context.ResultsIt was evident from the results of both the literature review and survey that the positive impacts of HTA implementation outweigh the negative impacts. Most of the reviewed literature discussed the impact of HTA on the intermediate objectives of the health finance policy in relation to the broad health system goals. According to the survey respondents, the most evident benefit of HTA implementation is improving the transparency and accountability of healthcare decisions.ConclusionsOverall, HTA implementation can introduce a myriad of benefits to healthcare systems in MICs as well. Our findings show that while HTA implementation may have the potential to generate cost savings in specific areas, there is no guarantee that HTA can generate savings at the macro level.Public Interest SummaryHealth technologies (medicines, devices, and interventions) are rapidly increasing in complexity and cost. Health Technology Assessment (HTA) guides healthcare decision-makers in choosing the most suitable, effective, affordable, and acceptable health technology to invest limited healthcare resources. However, healthcare decision-makers in middle-income countries (MICS) are still uncertain about whether adopting HTA would help them achieve the financial sustainability needed to achieve universal health coverage.Thereforewe sought to gather evidence on how HTA has affected the health systems of countries that have already adopted it by reviewing published research reports. In addition, healthcare decision-makers from three MICs were questioned about their perception of how HTA implementation will affect their country's health system.Wefound that the positive effects of HTA implementation outweigh the negative ones; specifically, the transparency and accountability of decisions are improved. However, although HTA implementation may generate cost savings in specific areas, it may not significantly contribute to overall financial sustainability.     

Therapies for advanced cancers pose a special challenge for health technology assessment organizations in many countries

Health technology assessment organizations evaluate medical therapies and technologies to help inform coverage and reimbursement decisions for payers around the globe. Even as they establish strict review processes, these organizations--and the reimbursement authorities that use their assessments--have sometimes handled cancer interventions with special care. We found that some countries have created separate health technology assessment pathways for cancer treatment, while others have eased access to cancer treatments through end-of-life or disease-severity exceptions within health technology assessment policies. In the United States, although no separate evaluation pathways exist for cancer, cancer drugs receive special status by virtue of unique Medicare rules covering off-label indications. Worldwide, we demonstrate that health technology assessment organizations are struggling with cancer's "exceptionalism."     

Evaluation of new medicines in Spain and comparison with other European countries

ObjectiveTo compare the use of health technology assessment (HTA) as a tool to support pricing and reimbursement (P&R) of new medicines in Spain with England, Sweden, France and Germany.MethodFor each country, the literature is used to identify the purpose and timing of the P&R decision, the HTA and decision-making procedures used to generate evidence, and the criteria used to make decisions.ResultsResults are presented as a summary of the HTA landscape for P&R of new medicines in each country. Comparisons are made between Spain and other countries regarding the procedure and implementation of HTA.ConclusionsBased on these assessments, we made recommendations for how HTA might develop in Spain with the aim of improving governance and efficiency. Spain has made considerable progress in recent years, but still falls short of international standards in terms of independence of the HTA agencies and decision-making committees from political influence and industrial policy, the setting of prices of medicines in relation to health gain, improve the transparency of the process and results of the evaluation, and promote the participation of stakeholders. In common with other countries, Spain needs to clarify the role of cost-effectiveness criteria. Further progress needs to be made to coordinate effort across the various agencies, strengthen technical staff, and ensure equitable access to medicines between regions.     

Patient access to pharmaceuticals: an international comparison

We have identified eight sub-dimensions of patient access to pharmaceuticals: marketing approvals, time of marketing approval, coverage, cost sharing, conditions of reimbursement, speed from marketing approval to reimbursement, extent to which beneficiaries control choice of their drug benefit, and evenness of the availability of drugs to the population. For a sample of commonly used best-selling drugs in the United States (US), we measured these eight access sub-dimensions across four health systems: France, the Netherlands, the United Kingdom (UK), and the US. Although the US approved between 15 and 18% more drugs than the other three countries, the US was slower than France and the UK to approve drugs licensed in all four countries. The percentage of drugs covered is approximately the same for all four countries. For covered drugs, we observe the least cost sharing by patients in the Netherlands. The Netherlands imposes conditions of reimbursement on a much larger percentage of drugs. France seems to be the slowest in respect of speed from marketing approval to reimbursement. The US is the most flexible in terms of the extent to which beneficiaries control their choice of drug benefit but it is the least universal in terms of evenness of the availability of drugs to the population. Our study confirms the frequently cited problems of access in European countries: lag between marketing approval and reimbursement, and inflexibility in respect of the extent to which beneficiaries control their choice of drug benefit. At the same time, our study confirms, qualitatively, different kinds of access problems in the US: relatively high patient cost sharing for pharmaceuticals, and wide variation in coverage.

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An overview of critical decision-points in the medical product lifecycle: Where to include patient preference information in the decision-making process?

BackgroundPatient preference (PP) information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients’ healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making.ObjectivesThe aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decision-points can potentially include PP information.MethodsA scoping literature review was conducted using five scientific databases. Semi-structured interviews were conducted with representatives from seven European countries and the US, including industry (n = 24), regulatory authorities (n = 23), reimbursement/HTA (n = 23). Finally, validation meetings with key stakeholders (n = 11) were conducted.ResultsSix critical decision-points were identified for industry decision-making, three for regulatory decision-making, and six for reimbursement/HTA decision-making. Stakeholder groups agreed that PP information is not systematically integrated, either as obligatory information or pre-set criteria, but would benefit all the listed decision-points in the future.ConclusionCurrently, PP information is not considered as obligatory information to submit for any of the MPLC decision-points. However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders.     

Budgeting for a billion: applying health technology assessment (HTA) for universal health coverage in India

Background

India recently launched the largest universal health coverage scheme in the world to address the gaps in providing healthcare to its population. Health technology assessment (HTA) has been recognised as a tool for setting priorities as the government seeks to increase public health expenditure. This study aims to understand the current situation for healthcare decision-making in India and deliberate on the opportunities for introducing HTA in the country.

Methods

A paper-based questionnaire, adapted from a survey developed by the International Decision Support Initiative (iDSI), was administered on the second day of the Topic Selection Workshop that was conducted as part of the HTA Awareness Raising Workshop held in New Delhi on 25–27 July, 2016. Participants were invited to respond to questions covering the need, demand and supply for HTA in their context as well as the role of their organisation vis-à-vis HTA. The response rate for the survey was about 68% with 41 participants having completed the survey.

Results

Three quarters of the respondents (71%) stated that the government allocated healthcare resources on the basis of expert opinion. Most respondents indicated reimbursement of individual health technologies and designing a basic health benefit package (93% each) were important health policy areas while medical devices and screening programmes were cited as important technologies (98% and 92%, respectively). More than half of the respondents noted that relevant local data was either not available or was limited. Finally, technical capacity was seen as a strength and a constraint facing organisations.

Conclusion

The findings from this study shed light on the current situation, the opportunities, including potential topics, and challenges in conducting HTA in India. There are limitations to the study and further studies may need to be conducted to inform the role that HTA will play in the design or implementation of universal health coverage in India.

     

Policies for Use of Real-World Data in Health Technology Assessment (HTA): A Comparative Study of Six HTA Agencies

Background

Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies’ policies for RWD use in relative effectiveness assessments (REA) is lacking.

International migration patterns of physicians to the United States: A cross-national panel analysis

ObjectivesTo analyze the dynamics of physician international migration patterns and identify the countries deviating most from expected migration rates.MethodsA negative binomial log-linear model of physician migration to the United States from every other country was constructed using a panel of country-level data for years 1994–2000. The model was used to identify factors associated with physician migration and to identify countries with higher or lower rates of physician migration than expected.ResultsPhysician migration varied with a country's GDP per capita in an inverse-U pattern, with highest migration rates from middle-income countries. The absence of medical schools, immigrant networks in the United States, medical instruction in English, proximity to the United States, and the lack of political and civil liberties were also associated with higher migration rates. Countries with higher-than-predicted migration rates included Iceland, Albania, Armenia, Dominica, Lebanon, Syria, the United Arab Emirates, and Bulgaria. Countries with lower-than-predicted migration rates included Mexico, Japan, Brazil, Zimbabwe, Mauritania, Portugal, Senegal, and France.ConclusionsThis analysis shows that many of the most powerful factors associated with physician migration are difficult or impossible for countries to change through public policy. GDP per capita and proximity to the U.S. are two of the most powerful predictors of physician migration. Networks of immigrants in the U.S. and fewer political and civil liberties also put countries at higher risk for physician emigration. Several other factors that were associated with physician migration might be more easily amenable to policy intervention. These factors include the absence of a medical school and medical instruction in English. Policies addressing these factors would involve making several difficult tradeoffs, however. Other examples of policies that are effective in minimizing physician migration might be found by examining countries with lower-than-expected migration rates.     

Impact of reimbursement policies on the adoption of medical devices in an outpatient setting

ObjectivesTo consider how reimbursement systems in the UK, Germany, Italy, France and Spain affect adoption of medical devices that facilitate care in an ambulatory setting.MethodsExamples of technologies that could be used in an outpatient setting but are predominantly used on inpatients were identified. Hospital payment systems were explored and the implications of funding policies for the adoption of medical devices in an outpatient setting considered.ResultsAlthough many countries attempt to develop ambulatory care payments, their DRG/HRG systems introduce a time lag for the uptake of new treatments and do not routinely identify and adopt enabling technologies. Arrangements to fund new technologies are often localised and inconsistent which can result in missed opportunities for savings. There are fewer reimbursement codes for outpatient procedures and this appears to present a barrier to the take-up of new technologies that reduce inpatient bed days. Current levels of outpatient fees are suited to fast, high volume, low cost procedures.ConclusionsThis review identifies attempts to improve coding tariffs, increase the frequency of updates and introduce more out-patient DRG codes. Healthcare payers need to be satisfied that new technologies are cost effective before they agree funding outside DRG based fee systems and the negotiation process would be more efficient if payers pooled expertise for reviewing cost-effectiveness evidence and fed conclusions directly to tariff setting authorities. New DRG codes and higher outpatient tariffs for cost effective technologies that enable a switch to ambulatory care could incentivise hospitals to revise care pathways.     

Do Reimbursement Recommendations by the Canadian Agency for Drugs and Technology in Health Translate Into Coverage Decisions for Orphan Drugs in the Canadian Province of Ontario?

ObjectivesUnlike 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.MethodsWe 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.ResultsWe 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.ConclusionsDespite 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.     

Reimbursement of pharmaceuticals: reference pricing versus health technology assessment

Reference pricing and health technology assessment are policies commonly applied in order to obtain more value for money from pharmaceuticals. This study focussed on decisions about the initial price and reimbursement status of innovative drugs and discussed the consequences for market access and cost. Four countries were studied: Germany, The Netherlands, Sweden and the United Kingdom. These countries have operated one, or both, of the two policies at certain points in time, sometimes in parallel. Drugs in four groups were considered: cholesterol-lowering agents, insulin analogues, biologic drugs for rheumatoid arthritis and “atypical” drugs for schizophrenia. Compared with HTA, reference pricing is a relatively blunt instrument for obtaining value for money from pharmaceuticals. Thus, its role in making reimbursement decisions should be limited to drugs which are therapeutically equivalent. HTA is a superior strategy for obtaining value for money because it addresses not only price but also the appropriate indications for the use of the drug and the relation between additional value and additional costs. However, given the relatively higher costs of conducting HTAs, the most efficient approach might be a combination of both policies.     

Towards Integrated Health Technology Assessment for Improving Decision Making in Selected Countries

Objectives

To assess the level of comprehensiveness of health technology assessment (HTA) practices around the globe and to formulate recommendations for enhancing legitimacy and fairness of related decision-making processes.

Divergent evidence requirements for authorization and reimbursement of high-risk medical devices – The European situation

BackgroundUnsafe and dangerous medical devices have entered the European market during the last decade, raising public awareness. Consequently, regulatory processes and their requirements for evidence are under discussion.ObjectiveThis research aims to explore the authorization and reimbursement processes and associated evidence requirements for high-risk medical devices in four regions: Europe, the United States, Australia and Canada.MethodsA literature search in PubMed about the authorization and reimbursement processes in the four regions was performed. Seven high-risk medical devices were selected as examples, and their authorization and reimbursement status were analyzed. Information was extracted from publicly available summaries of the authorization agencies of the regions, from the Controlled Clinical Trial Database, supplemented by information from HTA and reimbursement organizations.ResultsThe evidence required for the authorization and reimbursement processes differs strongly in the four regions regarding the levels of methodology and scrutiny. All seven devices have been authorized in Europe, three in Australia, one in the USA, and one in Canada. Currently none of the devices is recommended for reimbursement in the regions except one, in the USA. Devices that have been authorized in more than one region show that authorization has been two to three years earlier in Europe.ConclusionHuge differences and gaps in the evidence required for market authorization and for reimbursement were observed, especially between the two processes (authorization and reimbursement) in Europe. To ensure the high quality and safe provision of medical devices, harmonization of requirements and transparency in processes are needed.     

Variation in Health Technology Assessment and Reimbursement Processes in Europe

Background

It has been suggested that differences in health technology assessment (HTA) processes among countries, particularly within Europe, have led to inequity in patient access to new medicines.

Funding health technologies in decentralized systems: A comparison between Italy and Spain

Although cost-containment policies in Europe are focusing increasingly on medical devices, the impact of these policies has yet to be fully investigated, particularly in cross-country settings. This paper analyses coverage, procurement, and reimbursement of three inpatient medical devices (coronary stent, knee endoprosthesis and implantable cardioverter defibrillator) in the Italian and Spanish healthcare systems. The research was carried out by reviewing published and grey literature, as well as national and regional legislation; in addition, 19 experts from hospitals and the industry were interviewed.In both countries, there has been a shift in political power from the national to the regional level. At the same time, the content of public coverage has become more explicit. A major issue in both systems is reimbursement, i.e. the rules about funding the delivery of services included in the benefit baskets. The differences in procurement and funding mechanisms create different incentives that may have an impact on the uptake and diffusion of technologies. These mechanisms, however, can only partially explain organizational and professional behaviour, as the use of technologies in both countries is mainly left to professionals who are exposed to a variety of incentives. There is limited direct and indirect guidance of national and regional authorities over the use of technologies in both countries. It is likely that the difficult search for a balance between introducing innovations, containing costs and assuring equity will require stronger regulatory action in the next future.