Regional incentives and patient cross-border mobility: evidence from the Italian experience

Background: In recent years, accreditation of private hospitals followed by decentralisation of the Italian National Health Service (NHS) into 21 regional health systems has provided a good empirical ground for investigating the Tiebout principle of “voting with their feet”. We examine the infra-regional trade-off between greater patient choice (due to an increase in hospital services supply) and financial equilibrium, and we relate it to the significant phenomenon of Cross-Border Mobility (CBM) between Italian regions. Focusing on the rules supervising the financial agreements between regional authorities and providers of hospital care, we find incentives for private accredited providers in attracting patient inflows. Methods: The analysis is undertaken from an institutional, regulatory and empirical perspective. We select a sample of five regions with higher positive CBM balance and we examine regional regulations governing the contractual agreements between purchasers and providers of hospital care. According to this sample, we provide a statistical analysis of CBM and apply a Regional Attraction Ability Index (RAAI), aimed at testing patient preferences for private/public accredited providers. Results: We find that this index is systematically higher for private providers, both in the case of distance/boundary patients and of excellence/general hospitals. Conclusion: Conclusions address both financial issues regarding the coverage of regional healthcare systems and equity issues on patient healthcare access. They also raise concerns on the new European Union (EU) directive inherent to patient mobility across Europe

La media impresa farmaceutica in Italia: strategie, performance e prospettive

In the pharmaceutical industry, large-scale research and innovation have been broadly recognized as the major drivers of performance. While this paradigm is still valid, owing to the need to increase firms dimension in order to cope with the high risks related with innovation processes, it would seem that is no longer the only feasible strategy. Empirical research point to a poor corporate performance after mergers and acquisitions. Even in Italy small and medium- size firms prove to be more successful than bigger ones; the evidence resulting from an econometric exercise, we have carried out on a set of Italian firms, is consistent with the fact that the firms' dimension has no significant impact on performance. Many factors are contributing to a change in the industry structure. For example, the increasing difficulties in discovering new chemical entities and, in particular, new "blockbuster" drugs (while the existing ones have recently lost, or are about to lose, their patent protection). The advent of pharmacogenomics is rapidly changing the features of the research process, reducing its time, costs and probability of failure and, at the same time, is segmenting demand because new drugs are more specifically targeted. Even regulation can give medium-size firms a competitive advantage, due to their greater flexibility, which allows them to tackle a highly perturbed environment. Interviews with the top management of medium-size Italian pharmaceutical firms have confirmed that innovation, specialization and active internationalization are recognized as the key drivers of good performance. In order to operate in this new context firms should integrate themselves in large (international) networks, to benefit of economies of specialization. Besides they should rapidly enter more dynamics markets, to secure return on investment and profitability. As a result, support to network creation, regulatory stability and tax policies would seems to be the major issues to be cope with by public policies in the pharmaceutical industry

Valutazione di efficienza nella somministrazione dell’ormone della crescita (GH)

Treatment with growth hormone (somatropin) is effective in six different medical conditions: growth hormone deficiency (GHD), Turner syndrome (TS), growth retardation in children born small for gestational age (SGA), Prader-Willi syndrome (PWS), growth retardation due to chronic renal insufficiency (CRI), growth retardation associated with a deficiency of the gene SHOX (Short Stature HOmeoboXContaining gene). The treatment proved to be also effective in adults who have an impaired growth hormone (acquired in adulthood or childhood).The growth hormone (GH) is generally cost effective and, therefore, is usually reimbursed by public health services. In financial terms, GH is a major cost item for health systems. According to the Report OSMED 2010, GH ranks first in Italy between systemic hormonal preparations, excluding sex hormones, distributed by public system, with an annual value of approximately € 88 million (+ 12.7% compared to 2009).Considering the increasing need to control pharmaceutical expenditure, there is a strong interest for the efficient supply of the hormone by the regional health service. From this point of view, the comparison of the specialties on the market is normally carried out on the basis of the cost per mg; this approach, also used in the main studies of cost-effectiveness, is derived from a logic of cost minimization, but it may still be distorting, not taking into account the efficiency of devices used for the administration of the hormone.The proposed analysis verifies the efficiency of different available devices, evaluating the potential waste of product, depending both on the device used and on the characteristics of the population exposed to the treatment.Only in the case of single-dose and disposable formulations is theoretically possible to have zero waste and thus an equivalence between the actual cost of the treatment and the price charged. In other cases, the inefficiency causes a deviation between the actual cost and price. In the latter cases, since the theoretical amount of the population exposed to the treatment, it is estimated that the total share of potentially unused product can vary between 208,000 mg / year to 750,000 mg / year, depending on the device used. In particular, there is an actual average cost of treatment ranging between +9.9% and +11.4% of the ex-factory price; depending on the different doses and even between the different devices, the difference between the actual price and the theoretical price varies from a minimum of +6.9% and a maximum of +18.7%

The general results of the RN4CAST survey in Italy

The issue of health workforce shortage and in particular of nurses, has been debated globally for almost three decades (Aiken & Mullinix 1987, Aiken et al. 1996, 2001, 2010), and has been exacerbated by the recent global financial crisis. The European RN4CAST project has shifted focus from considering only nursing workforce planning and workforce volumes to considering the impact of adequate nurse-patient ratios and work environment on patient safety and the quality of care (Sermeus et al. 2011)

The use of Real World Evidence in the European context: An analysis of key expert opinion

Randomised controlled trials (RCT), traditionally seen as the gold standard in drug approval requirement terms, are becoming more difficult due to, among other reasons, budget constraints, increasing complexities and the shrinking of patient populations. Real world evidence (RWE), data used for decision making that is not derived from traditional RCT, may in future play an increasing role in market access and reimbursement decisions. This paper analyses key pricing and reimbursement stakeholders’ opinions of RWE across five European countries via a focus group-style discussion. Areas probed included regulatory implications and the role of RWE in the study countries, RWE processes and implementation on decision making, meaningful outcomes from RWE and priorities for future focus and industry support. Results showed that RWE was used to some extent in all countries, generally in accelerated access and re-review situations, with accepted endpoints including overall survival, morbidity, avoidable mortality and quality of life among others, but that there were a number of areas where improvement was necessary if RWE use was to become more common place

Orphan drugs in Italy: availability and time-to-access at regional level

Introduction: There are an estimated 26 million rare disease patients in Europe, about 2 million in Italy. Access to orphan drugs has been evaluated nationally and internationally, and delays have been observed due to evaluation of price and therapeutic value. The objectives of this study are: to assess the availability of EMA-authorized orphan drugs at national and regional level; to study time trends and regional variability in consumption and spending, and to estimate the time to access in Italian regions. Methods: We evaluate the availability of EMA authorized orphan drugs in Italy. Based on data from the Traceability of medicines dataflow for period 2016-2021 we evaluate the expenditure, consumption and availability of orphan drugs in each region. To estimate the time to access we consider the days between the end of negotiation procedure and the first purchase by regions. Results: In 2021 in Italy are available 94% of EMA authorized orphan drugs. The expenditure and consumption have grown during the last decade. The availability is higher in bigger regions, that also take care of patients from smaller regions. The pro capite expenditure and consumption in DDD/1,000 ab die is similar in all the geographic area. Time to regional access is on average 123 days, that grows to 224 excluding the orphan drugs dispensed before the end of reimbursement procedure

Long-term spasticity management in post-stroke patients: issues and possible actions—A Systematic Review with an Italian expert opinion

Spasticity is a well-known motor dysfunction occurring after a stroke. A group of Italian physicians' experts in treating post-stroke spasticity (PSS) reviewed the current scientific evidence concerning the state-of-the-art clinical management of PSS management and the appropriate use of botulinum toxin, aiming to identify issues, possible actions, and effective management of the patient affected by spasticity. The participants were clinicians specifically selected to cover the range of multidisciplinary clinical and research expertise needed to diagnose and manage PSS. When evidence was not available, the panel discussed and agreed on the best way to manage and treat PSS. To address the barriers identified, the panel provides a series of consensus recommendations. This systematic review provides a focused guide in the evaluation and management of patients with PSS and its complications. The recommendations reached by this panel of experts should be used by less-experienced doctors in real life and should be used as a guide on how to best use botulinum toxin injection in treating spasticity after a stroke

RWE in Europe Paper II: The use of Real World Evidence in the disease context

Real World Evidence (RWE), the use of data not collected via traditional randomised controlled trials (RCT) for decision-making, is becoming more interesting to market-access and reimbursement decision-makers, despite potential methodological issues around its use. This paper, the second in a series looking at the use of Real World Evidence (RWE) in Europe, analyses the opinions of a number of key experts in pricing and reimbursement from a selection of countries across Europe. Discussion centred on the use of RWE in licensing, commissioning, clinical decision-making and patient and outcome related decision-making in the context of three different treatment areas – chronic disease, oncology and rare diseases. Results of discussion sessions with ‘RWE experts’ indicated that the associated benefits of RWE are becoming more relevant but there is a need for a well-organised, high quality system for data generation, interpretation and use. It is likely that different treatment areas will have differing RWE requirements and differing levels of utility. In the rare disease arena, RWE may have a role in licensing based decisions, but this is unlikely for chronic disease or oncology. In order to enhance the role of RWE, and to ensure it meets its full potential in all treatment areas, a multi-stakeholder approach at the EU level is required, with collaboration between national and supranational organisations and all stakeholders including patient organisations, manufacturers and reimbursement agencies

RWE in Europe Paper III: A Roadmap for RWE

Real world evidence (RWE) has been touted as a remedy for current market access issues, facilitating quicker approvals and increased odds of reimbursement at a good price. It is therefore an attractive avenue for pursuit for manufacturers today. This paper, the third in a series looking at the use of RWE in Europe, outlines the discussions held between key opinion leaders in pricing and reimbursement across a number of European countries at a roundtable-style meeting. The aim of the meeting was to develop a 3-year roadmap, and resulting action plan, of initiatives for the enhanced use of RWE in decision-making in the pharmaceutical industry. Following a series of brainstorming sessions across the areas of commissioning and access, clinical evidence and patients and outcomes, contributors were asked to prioritise the importance of a refined set of initiatives identified in these brainstorming sessions to develop the three-year road map. Finally, four key points from the roadmap were identified for initial action: actively engage in early dialogue with payers on RWE needs; consensus exercise on RWD/E in clinical decisions, develop a definition of patient reported/relevant outcomes and develop a model approach for the collection of patient reported/relevant outcomes data. These action points are seen as the most imperative steps for enhancing the role of RWE. If its use is to become more common addressing these steps, as quickly and efficiently as possible, will be vital for all stakeholders in the pharmaceutical arena