Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Background Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them. Objectives To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology—specifically economic evaluation methods—in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges. Methods An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs. Results Three key topics were identified and prioritised for discussion—uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making. Conclusions ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. Classification code I

The cost-effectiveness of long-term antiviral therapy in the management of HBeAg-positive and HBeAg-negative chronic hepatitis B in Singapore

The purpose of this study was the economic evaluation of short-duration treatments of chronic hepatitis B (CHB) and longer duration antiviral treatment for up to 5 years. Two 10-health state Markov models were developed for hepatitis B e antigen (HBeAg)-positive and HBeAg-negative CHB patients respectively. The perspective of this economic evaluation was the Singapore healthcare system and CHB patient. The models followed cohorts of HBeAg-positive and HBeAg-negative CHB patients, respectively, over a period of 40 years, by which time the majority of the cohorts would have died if left untreated. Costs and benefits were discounted at 5% per annum. Annual rates of disease progression and the magnitude of treatment effects were obtained from the literature, with a focus on data obtained in Asian patients and meeting the criteria for therapy as described in internationally recognized management guidelines. Short-course therapy with α-interferon, or 1-year treatment with pegylated interferon α-2a, lamivudine or adefovir had limited impact on disease progression. In contrast, treatment of CHB with antiviral therapy for 5 years substantially decreased the rate of disease progression. Treatment with lamivudine for 1-year is highly cost-effective compared with no treatment of CHB but has limited effect on reducing the rate of disease progression. Compared with 1-year treatment with lamivudine, sequential antiviral therapies for up to 5 years (i.e. lamivudine plus adefovir on emergence of lamivudine resistance or adefovir plus lamivudine on emergence of adefovir resistance) are highly cost-effective by international standards. These conclusions are robust to uncertainties in model inputs and are consistent with the findings of other recently published studies

Copyright and cultural work: an exploration

This article first discusses the contemporary debate on cultural “creativity” and the economy. Second, it considers the current state of UK copyright law and how it relates to cultural work. Third, based on empirical research on British dancers and musicians, an analysis of precarious cultural work is presented. A major focus is how those who follow their art by way of “portfolio” work handle their rights in ways that diverge significantly from the current simplistic assumptions of law and cultural policy. Our conclusions underline the distance between present top-down conceptions of what drives production in the cultural field and the actual practice of dancers and musicians

Algorithmic Complexity for Short Binary Strings Applied to Psychology: A Primer

Since human randomness production has been studied and widely used to assess executive functions (especially inhibition), many measures have been suggested to assess the degree to which a sequence is random-like. However, each of them focuses on one feature of randomness, leading authors to have to use multiple measures. Here we describe and advocate for the use of the accepted universal measure for randomness based on algorithmic complexity, by means of a novel previously presented technique using the the definition of algorithmic probability. A re-analysis of the classical Radio Zenith data in the light of the proposed measure and methodology is provided as a study case of an application.Comment: To appear in Behavior Research Method

The politics of the digital single market:Culture vs. competition vs. copyright

This paper examines the implications for European music culture of the European Union’s Digital Single Market strategy. It focuses on the regulatory framework being created for the management of copyright policy, and in particular the role played by Collective Management Organisations (or Collecting Societies). One of the many new opportunities created by digitalization has been the music streaming services. These depend on consumers being able to access music wherever they are, but such a system runs counter to the management of rights on a national basis and through collecting organisations who act as monopolies within their own territories. The result has been ‘geo-blocking’. The EU has attempted to resolve this problem in a variety of ways, most recently in a Directive designed to reform the CMOs. In this paper, we document these various efforts, showing them to be motivated by a deep-seated and persisting belief in the capacity of ‘competition’ to resolve problems that, we argue, actually lie elsewhere - in copyright policy itself. The result is that the EU’s intervention fails to address its core concern and threatens the diversity of European music culture by rewarding those who are already commercially successful

The logic-bias effect: The role of effortful processing in the resolution of belief-logic conflict.

According to the default interventionist dual-process account of reasoning, belief-based responses to reasoning tasks are based on Type 1 processes generated by default, which must be inhibited in order to produce an effortful, Type 2 output based on the validity of an argument. However, recent research has indicated that reasoning on the basis of beliefs may not be as fast and automatic as this account claims. In three experiments, we presented participants with a reasoning task that was to be completed while they were generating random numbers (RNG). We used the novel methodology introduced by Handley, Newstead & Trippas (Journal of Experimental Psychology: Learning, Memory, and Cognition, 37, 28-43, 2011), which required participants to make judgments based upon either the validity of a conditional argument or the believability of its conclusion. The results showed that belief-based judgments produced lower rates of accuracy overall and were influenced to a greater extent than validity judgments by the presence of a conflict between belief and logic for both simple and complex arguments. These findings were replicated in Experiment 3, in which we controlled for switching demands in a blocked design. Across all three experiments, we found a main effect of RNG, implying that both instructional sets require some effortful processing. However, in the blocked design RNG had its greatest impact on logic judgments, suggesting that distinct executive resources may be required for each type of judgment. We discuss the implications of our findings for the default interventionist account and offer a parallel competitive model as an alternative interpretation for our findings

Market access agreements for pharmaceuticals in Europe: diversity of approaches and underlying concepts

<p>Abstract</p> <p>Background</p> <p>Market Access Agreements (MAA) between pharmaceutical industry and health care payers have been proliferating in Europe in the last years. MAA can be simple discounts from the list price or very sophisticated schemes with inarguably high administrative burden.</p> <p>Discussion</p> <p>We distinguished and defined from the health care payer perspective three kinds of MAA: Commercial Agreements (CA), Payment for Performance Agreements (P4P) and Coverage with Evidence Development (CED). Apart from CA, the agreements assumed collection and analysis of real-life health outcomes data, either from a cohort of patients (CED) or on per patient basis (P4P). We argue that while P4P aim at reducing drug cost to payers without a systematic approach to addressing uncertainty about drugs' value, CED were implemented provisionally to reduce payer's uncertainty about value of a medicine within a defined time period.</p> <p>Summary</p> <p>We are of opinion that while CA and P4P have a potential to reduce payers' expenditure on costly drugs while maintaining a high list price, CED address initial uncertainty related to assessing the real-life value of new drugs and enable a final HTA recommendation or reimbursement and pricing decisions. Further, we suggest that real cost to health care payers of drugs in CA and P4P should be made publicly available in a systematic manner, to avoid a perverse impact of these MAA types on the international reference pricing system.</p

Health care utilisation under the 30-Baht Scheme among the urban poor in Mitrapap slum, Khon Kaen, Thailand: a cross-sectional study

Background: In 2001, the Government of Thailand introduced a universal coverage scheme with the aim of ensuring equitable health care access for even the poorest citizens. For a flat user fee of 30 Baht per consultation, or for free for those falling into exemption categories, every scheme participant may access registered health services. The exemption categories include children under 12 years of age, senior citizens aged 60 years and over, the very poor, and volunteer health workers. The functioning of these exemption mechanisms and the effect of the scheme on health service utilisation among the poor is controversial. Methods: This cross-sectional study investigated the prevalence of 30-Baht Scheme registration and subsequent self-reported health service utilisation among an urban poor population in the Teparuk community within the Mitrapap slum in Khon Kaen city, northeastern Thailand. Furthermore, the effectiveness of the exemption mechanisms in reaching the very poor and the elderly was examined. Factors for users' choice of health facilities were identified. Results: Overall, the proportion of the Teparuk community enrolled with the 30-Baht Scheme was high at 86%, with over one quarter of these exempted from paying the consultation fee. User fee exemption was significantly more frequent among households with an above-poverty-line income (64.7%) compared to those below the poverty line (35.3%), χ2 (df) = 5.251 (1); p-value = 0.018. In addition, one third of respondents over 60 years of age were found to be still paying user fees. Self-reported use of registered medical facilities in case of illness was stated to be predominantly due to the service being available through the scheme, with service quality not a chief consideration. Overall consumer satisfaction was high, especially among those not required to pay the 30 Baht user fee. Conclusion: Whilst the 30-Baht Scheme seems to cover most of the poor population of Mitrapap slum in Khon Kaen, the user fee exemption mechanism only works partially with regard to reaching the poorest and exempting senior citizens. Service utilisation and satisfaction are highest amongst those who are fee-exempt. Service quality was not an important factor influencing choice of health facility. Ways should be sought to improve the effectiveness of the current exemption mechanisms.Sophie Coronini-Cronberg, Wongsa Laohasiriwong and Christian A Gerick