scholarly journals Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations

2014 ◽  
pp. 1 ◽  
Author(s):  
Georgi Iskrov ◽  
Rumen Stefanov
2019 ◽  
Vol 4 (2) ◽  
pp. 238146831989223
Author(s):  
Alec Morton ◽  
Abigail Colson ◽  
Axel Leporowski ◽  
Anna Trett ◽  
Taimur Bhatti ◽  
...  

Antibiotics have revolutionized the treatment of bacterial infections. However, it is widely held that there is underinvestment in antibiotics research and development relative to the socially optimal level for a number of reasons. In this article, we discuss whether existing health technology assessment procedures recognize the full economic and societal value of new antibiotics to patients and society when making reimbursement decisions. We present three recommendations for modelling the unique attributes of value that are specific to novel antibiotics. We find, based on a review of the literature, that some of the value elements proposed by our framework have previously been discussed qualitatively by health technology assessment bodies when evaluating antibiotics, but are not yet formally captured via modelling. We present a worked example to show how it may be possible to capture these dimensions of value in a more quantitative manner. We conclude by answering the question of the title as follows: the unique attributes of novel antibiotics should be considered in reimbursement decision making, in a way that captures the full range of benefits these important technologies bring to patients, health care systems, and society.


2020 ◽  
Vol 36 (S1) ◽  
pp. 38-38
Author(s):  
Maíra Catharina Ramos ◽  
Margarete Martins de Oliveira ◽  
Erica Tatiane da Silva ◽  
Daniella Cristina Rodrigues Pereira ◽  
Flávia Tavares da Silva Elias

IntroductionThe interaction of health technology assessment (HTA) and health regulatory agencies has been widespread, especially for decision-making in health system coverage. The objective of this paper is to report the HTA-regulatory interaction in Brazil.MethodsThis is a case study on the interaction between HTA and regulation in Brazil. Technical documents and Brazilian legislation on health regulation and HTA were analyzed. The study was conducted in July 2019.ResultsHTA-Regulatory Interaction in Brazil is still incipient. There is no responsible agency for interaction between agencies, as there is in Europe and Canada, for example. In the last 4 years, cooperation has started between the Brazilian Health Surveillance Agency (Anvisa) and the Oswaldo Cruz Foundation (Fiocruz) for post-registration monitoring of medicines. During this partnership, 170 post-marketing drug opinions were prepared, assisting the regulatory agency in decision-making.ConclusionsBrazil legislation guarantees essential medicines at low cost or free. The interaction between HTA and regulation has the potential to reduce the time taken to incorporate technology to the patient, in addition to ensuring greater safety for users of the Unified Health System. In this sense, it was observed that the interaction between health regulation and science and technology institutions has innovative potential in this approach.


2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Samantha Hollingworth ◽  
Ama Pokuaa Fenny ◽  
Su-Yeon Yu ◽  
Francis Ruiz ◽  
Kalipso Chalkidou

Abstract Background Countries in Sub-Saharan Africa (SSA) are moving towards universal health coverage. The process of Health Technology Assessment (HTA) can support decisions relating to benefit package design and service coverage. HTA involves institutional cooperation with agreed methods and procedural standards. We systematically reviewed the literature on policies and capacity building to support HTA institutionalisation in SSA. Methods We systematically reviewed the literature by searching major databases (PubMed, Embase, etc.) until June 2019 using terms considering three aspects: HTA; health policy, decision making; and SSA. We quantitatively extracted and descriptively analysed content and conducted a narrative synthesis eliciting themes from the selected literature, which varied in study type and apporach. Results Half of the 49 papers identified were primary research studies and mostly qualitative. Five countries were represented in six of ten studies; South Africa, Ghana, Uganda, Cameroon, and Ethiopia. Half of first authors were from SSA. Most informants were policy makers. Five themes emerged: (1) use of HTA; (2) decision-making in HTA; (3) values and criteria for setting priority areas in HTA; (4) involving stakeholders in HTA; and (5) specific examples of progress in HTA in SSA. The first one was the main theme where there was little use of evidence and research in making policy. The awareness of HTA and economic evaluation was low, with inadequate expertise and a lack of local data and tools. Conclusions Despite growing interest in HTA in SSA countries, awareness remains low and HTA-related activities are uncoordinated and often disconnected from policy. Further training and skills development are needed, firmly linked to a strategy focusing on strengthening within-country partnerships, particularly among researchers and policy makers. The international community has an important role here by supporting policy- relevant technical assistance, highlighting that sustainable financing demands evidence-based processes for effective resource allocation, and catalysing knowledge-sharing opportunities among countries facing similar challenges.


Author(s):  
Olina Efthymiadou ◽  
Panos Kanavos

Abstract Background Managed Entry Agreements (MEAs) are increasingly used to address uncertainties arising in the Health Technology Assessment (HTA) process due to immature evidence of new, high-cost medicines on their real-world performance and cost-effectiveness. The literature remains inconclusive on the HTA decision-making factors that influence the utilization of MEAs. We aimed to assess if the uptake of MEAs differs between countries and if so, to understand which HTA decision-making criteria play a role in determining such differences. Methods All oncology medicines approved since 2009 in Australia, England, Scotland, and Sweden were studied. Four categories of variables were collected from publicly available HTA reports of the above drugs: (i) Social Value Judgments (SVJs), (ii) Clinical/Economic evidence submitted, (iii) Interpretation of this evidence, and (iv) Funding decision. Conditional/restricted decisions were coded as Listed With Conditions (LWC) other than an MEA or LWC including an MEA (LWCMEA). Cohen's κ-scores measured the inter-rater agreement of countries on their LWCMEA outcomes and Pearson's chi-squared tests explored the association between HTA variables and LWCMEA outcomes. Results A total of 74 drug-indication pairs were found resulting in n = 296 observations; 8 percent (n = 23) were LWC and 55 percent (n = 163) were LWCMEA. A poor-to-moderate agreement existed between countries (−.29 < κ < .33) on LWCMEA decisions. Cross-country differences within the LWCMEA sample were partly driven by economic uncertainties and largely driven by SVJs considered across agencies. Conclusions A set of HTA-related variables driving the uptake of MEAs across countries was identified. These findings can be useful in future research aimed at informing country-specific, “best-practice” guidelines for successful MEA implementation.


2019 ◽  
Vol 35 (S1) ◽  
pp. 48-48
Author(s):  
Leonor Varela-Lema ◽  
Janet Puñal-Riobóo ◽  
Paula Cantero-Muñoz ◽  
Maria José Faraldo-Vallés

IntroductionDecision making regarding national population-based prenatal and newborn screening policies is recognized to be highly challenging. This paper aims to describe the formalized collaboration that has been established between the Spanish National Public Health Screening Advisory Committee (PHSAC) and the Spanish Network of Health Technology Assessment (HTA) agencies to support the development of evidence- and consensus-based recommendations to support this process.MethodsIn-depth description and analysis of the strategic and methodological processes that have been implemented within the Spanish National Health System prenatal and newborn screening frameworks, with special emphasis on the role, actions, and responsibilities of HTA agencies.ResultsThe role of HTA agencies is threefold: (i) support the PHSAC by providing evidence on safety, effectiveness and cost/effectiveness of the screening tests/strategies, as well as contextualized information regarding costs, organizational, social, legal and ethical issues; (ii) collaborate with the PHSAC in the development of formal evidence- and consensus-based recommendations for defining population screening programs, when required; (iii) analyze real-world data that is generated by piloted programs. This paper will provide real-life examples of how these processes were implemented in practice, with a special focus on the development of the non-invasive prenatal testing (NIPT) policy. Recommendations for NIPT were developed by a multidisciplinary group based on the European network for Health Technology Assessment (EUnetHTA) rapid assessment report and the predictive models that were built using national statistics and other contextualized data.ConclusionsThe current work represents an innovative approach for prenatal and newborn screening policymaking, which are commonly difficult to evaluate due to the low quality of evidence and the confounding public health issues. The paper raises awareness regarding the importance of joint collaborations in areas where evidence is commonly insufficient for decision making.


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