Claudio Jommi

The new pharmaceutical policy in Italy

Pressed by an impressive series of corruption scandals and by a change of attitude towards cost-containment, the Italian pharmaceutical sectors regulatory environment was radically changed in 1994. Regulatory power was concentrated on a national technical body (CUF) and a new set of measures was taken, including a nationwide drug expenditure budget, a redefinition of both the positive list and the cost-sharing rules, and new price-setting models. As a result, in the period 1993-1996, nominal expenditures decreased by about L 1600 billion (ECU 83.6 billion at 1997 exchange rate), that is from 13.3% to 11.0% of current National Health Service (NHS) expenditure. While in the 1980s Italy was one of the most generous countries in funding pharmaceuticals, it is now one of the most parsimonious. Although the overall pharmaceutical market shrank in 1994 and 1995, a substantial part of NHS drug-bill savings resulted from cost-shifting from the public sector to patients, mainly because physicians have not aligned their prescribing behaviour to the new positive list. The new Italian approach to containing pharmaceutical costs has been certainly effective, at least in the short run. However, new relevant issues are emerging regarding the fall of NHS pharmaceutical coverage, the centralised nature of the Italian pharmaceutical policy and the gap between scientific based policies and actual prescribing behaviours.

The last decade of Italian pharmaceutical policy: instability or consolidation?

Pharmaceutical policy in Italy has been reshaped as a result of the 1993–4 crisis in which it was revealed that pharmaceutical companies, policy makers and top Department of Health officers had constructed an illegal system to set prices. Following this crisis, the rise of technical competency and leadership in the Italian Department of Health and, since 2000, in the Drug Regulatory Agency (Agenzia Italiana del Farmaco; AIFA) has achieved major improvements in many aspects of Italian pharmaceutical policy. These improvements have included increased transparency of decision making, the use of evidence-based medicine principles for reimbursement and pricing, and the use of generic drugs to lower prices.As a result of these changes, pharmaceutical expenditure has been controlled and equity has improved, mainly because co-payments have been reduced, thus reducing private expenditure on reimbursable drugs. However, a short-term approach to cost containment has prevailed, and Italian pharmaceutical policy has neglected industrial parameters. Hence, the trend in pharmaceutical expenditure has been erratic, and Italy has not favoured localization of research and development and production in its territory.The dominant issue of Italian health policy in recent years has been devolution of powers to regions, the intermediate tier of the Italian State. Overall, devolution has increased regional accountability on healthcare spending. However, regions react to enhanced freedom in different ways, reflecting their institutional capacity and competencies. This process has also affected pharmaceutical policy, more than in other decentralized healthcare systems (such as Germany and Spain). Such a situation is causing increasing regional variations and geographical equity concerns. In addition, the regional level appears rather inadequate to promote an industrial perspective unless it is supported by national initiatives.

Diagnostic accuracy of HLA-B*57:01 screening for the prediction of abacavir hypersensitivity and clinical utility of the test: a meta-analytic review

AIM To determine diagnostic accuracy of HLA-B*57:01 testing for prediction of abacavir-induced hypersensitivity and to quantify the clinical benefit of pretreatment screening through a meta-analytic review of published studies. METHODS A comprehensive search was performed up to June 2013. The methodological quality of relevant studies was assessed by the QUADAS-2 tool. The pooled diagnostic estimates were calculated using a random effect model. RESULTS Despite the presence of heterogeneity in sensitivity or specificity estimates, the pooled diagnostic odds ratio to detect abacavir-induced hypersensitivity on the basis of clinical criteria was 33.07 (95% CI: 22.33-48.97, I(2): 13.9%), while diagnostic odds ratio for detection of immunologically confirmed abacavir hypersensitivity was 1141 (95% CI: 409-3181, I(2): 0%). Pooled analysis of risk ratio showed that prospective HLA-B*57:01 testing significantly reduced the incidence of abacavir-induced hypersensitivity. CONCLUSION This meta-analysis demonstrates an excellent diagnostic accuracy of HLA-B*57:01 testing to detect immunologically confirmed abacavir hypersensitivity and corroborates existing recommendations.

Stakeholders involvement by HTA Organisations: Why is so different?

OBJECTIVE To investigate stakeholder involvement by Health Technology Assessment Organisations (HTAOs) in France, Spain, England and Wales, Germany, Sweden, and The Netherlands and to examine whether this involvement depends on (i) the administrative tradition and the relevant conception of the relationship between state and society (contractarian and corporative vs. organic), (ii) the general structure of the healthcare system (HCS) (Bismarckian vs. Beveridgian system), and (iii) the role of Health Technology Assessment (HTA) and HTAOs in the HCS. METHODS Given the exploratory nature of the study, we considered interviews based on semi-structured questionnaires the most appropriate data-gathering technique. The interviews were administered to 16 key personnel in the HTAOs concerned. We have also carried out a literature review on HTAOs and stakeholders (1999-2011) using PubMed, Ebsco, JSTOR and Wiley Science. RESULTS In contractarian and (to a lesser extent) Bismarckian models, stakeholders are more involved. The administrative tradition and the HCS appear less important when the HTA is binding and used for regulatory purposes. In such situations, stakeholders are more intensively involved because their participation provides an opportunity for HTAOs to achieve consensus and legitimacy in advance. CONCLUSIONS Despite the limitations of the research (we did not conduct multiple interviews for each HTAO, and key informants were not always available) and its exploratory nature, we can conclude that models of stakeholders involvement cannot easily be transferred from one country to another due to the importance of national administrative traditions and the characteristics of HCSs.

The role of health technology assessment bodies in shaping drug development.

The use of health technology assessment (HTA) to inform policy-making is established in most developed countries. Compared to licensing agencies, HTA agencies have different interests and, therefore, different evidence requirements. Criteria for coverage or reimbursement decisions on pharmaceutical compounds vary; however, it is common to include, as part of the HTA, a comparative effectiveness evaluation. This type of clinical data might go beyond that required for market authorization, thus creating an additional evidence gap between the regulatory and the reimbursement submission. The relevance of submissions to HTA agencies is consistently increasing in a pharmaceutical company’s perspective, as market prospects are strongly influenced by third-party payers’ coverage. In this study, we aim to describe current HTA activities with a potential impact throughout the drug development process of pharmaceuticals, with a comparative emphasis on the systems in place in Italy and in the UK. Based on an extensive literature and website review, we identified three major classes of HTA activities, beyond mainstream HTA, with the potential to influence the drug development program: 1) horizon scanning and early HTA; 2) bipartite and tripartite early dialogue between manufacturers, regulators, and HTA assessors; and 3) managed market entry agreements. From early stages of clinical research up to postauthorization studies, there is a trend toward increased collaboration between parties, anticipation of market access evidence collection, and postmarketing risk-sharing. Heterogeneity of HTA practices increases the complexity of the market access environment. Overall, there are signals that market access departments are gaining importance in the pharmaceutical companies, but there is still a lack of evidence and reporting on how the increasing relevance of HTA has reshaped the way clinical development is designed and managed.

Multi-tier drugs assessment in a decentralised health care system. The Italian case-study

OBJECTIVE To investigate the organisation and decision-making processes of regional and local therapeutic committees in Italy, as a case-study of decentralised health care systems. METHODS A structured questionnaire was designed, validated, and self-administered to respondents. Committee members, prioritisation, assessment process and criteria, and transparency of committees were investigated. RESULTS The respondents represent 100% of the 17 regional committees out of 21 regions (in 4 regions there is not any regional formulary), 88% of the 16 hospital networks and 42% of the 183 public hospitals. The assessment process appears fragmented and may take a long time: drugs inclusion into hospital formularies requires two steps in most regions (regional and local assessment). Most of the therapeutic committees are closed to industry and patients associations involvement. Prioritisation in the assessment is mostly driven by disease severity, clinical evidence, and the absence of therapeutic alternatives. Only 13 out of the 17 regional committees have a public application form for drugs inclusion into regional formulary. Regional and local committees (i) often re-assess the clinical evidence already evaluated at central level and (ii) mostly rely on comparative drug unit prices per DDD and drug budget impact. The level of transparency is quite low. CONCLUSIONS The Italian case-study provides useful insights into an appropriate management of multi-tier drugs assessment, which is particularly complex in decentralised health care systems, but exists also in centralised systems where drugs are assessed by local therapeutic committees. A clear definition of regulatory competences at different levels, a higher collaboration between central, regional and local actors, and increased transparency are necessary to pursue consistency between central policies on price and reimbursement and budget accountability at the regional and local levels.

The impact of HTA and procurement practices on the selection and prices of medical devices

Technological innovation in healthcare yields better health outcomes but also drives healthcare expenditure, and governments are struggling to maintain an appropriate balance between patient access to modern care and the economic sustainability of healthcare systems. Health Technology Assessment (HTA) and centralized procurement are increasingly used to govern the introduction and diffusion of new technologies in an effort to make access to innovation financially sustainable. However, little empirical evidence is available to determine how they affect the selection of new technologies and unit prices. This paper focuses on medical devices (MDs) and investigates the combined effect of various HTA governance models and procurement practices on the two steps of the MD purchasing process (i.e., selecting the product and setting the unit price). Our analyses are based on primary data collected through a national survey of Italian public hospitals. The Italian National Health Service is an ideal case study because it is highly decentralized and because regions have adopted different HTA governance models (i.e., regional, hospital-based, double-level or no HTA), often in combination with centralized regional procurement programs. Hence, the Italian case allows us to test the impact of different combinations of HTA models and procurement programs in the various regions. The results show that regional HTA increases the probability of purchasing the costliest devices, whereas hospital-based HTA functions more like a cost-containment unit. Centralized regional procurement does not significantly affect MD selection and is associated with a reduction in the MD unit price: on average, hospitals located in regions with centralized procurement pay 10.1% less for the same product. Hospitals located in regions with active regional HTA programs pay higher prices for the same device (+23.2% for inexpensive products), whereas hospitals that have developed internal HTA programs pay 8.3% on average more for the same product.

Pharmaceutical Pricing Policies in Italy

This chapter provides an overview of drug price and reimbursement regulations in Italy within the general context of Italian pharmaceutical policy.

Generic Preference-based Measures for Low Back Pain: Which of Them Should Be Used?

Study Design. Systematic review. Objective. This systematic review examines validity and responsiveness of three generic preference-based measures in patients with low back pain (LBP). Summary of Background Data. LBP is a very common incapacitating disease with a significant impact on health-related quality of life (HRQoL). Health state utility values can be derived from various preference-based HRQoL instruments, and among them the most widely ones are EuroQol 5 dimensions (EQ-5D), Short Form 6 Dimensions (SF-6D), and Health Utilities Index 3 (HUI III). The ability of these instruments to reflect HRQoL has been tested in various contexts, but never for LBP populations. Methods. A systematic search on electronic literature databases was undertaken to identify studies of patients with LBP where health state utility values were reported. Records were screened using a set of predefined eligibility criteria. Data on validity (correlations and known group methods) and responsiveness (effect sizes, standardized response means, tests of statistical significance) of instruments were extracted using a customized extraction template, and assessed using predefined criteria. Results. There were substantial variations in the 37 included papers identified in relation to study design and outcome measures used. EQ-5D demonstrated good convergent validity, as it was able to distinguish between known groups. EQ-5D was also able to capture changes of health states as results of different interventions. Evidence for SF-6D and HUI III was limited to allow an appropriate evaluation. Conclusion. EQ-5D performs well in LBP population and its scores seem to be suitable for economic evaluation of LBP interventions. However, the paucity of information on the other instruments makes it impossible to determine its relative validity and responsiveness compared with them. Level of Evidence: 2

Prospettive di utilizzo della Valutazione Economica e della Budget Impact Analysis nelle politiche del farmaco in Italia: i risultati di uno studio Delphi

In Italy decisions concerning drug reimbursement and prices seem that are not based on cost-effectiveness analysis (CEA) results or on the expected impact on health care budget i.e., budget impact analysis (BIA). This study aimed at scrutinizing the reasons of this attitude, anticipating possible future changes, by using a Delphi panel with thirteen experts in economic evaluation and pharmaceutical policy. Politicians’ distaste for explicit rationing, short-term cost-containment pressure and silos budgeting have been identified as the main obstacles to a systematic use of CEA and BIA. Although these obstacles are likely to persist in the future, consensus was reached among the experts for a broader use of CEA results at the national level, should a new drug provide considerable benefits versus a very high price, and of BIA at the regional level to support local sustainability. It is also imperative that both central and regional governments invest in post-marketing studies to evaluate whether the initial decision made when the new product was launched are consistent with a rational use of the scarce resources. Experts also advocated for (i) stronger interactions between the industry and third party payers, both at national and regional levels, especially at the drug market launch, when the industry is required to provide economic evidence and third party payers to assess this evidence, and (ii) an accredited network of research centers and professionals, supporting third party payers in this type of assessment, provided that the conflict of interest is rigorously verified.