Amelia Compagni

Orphan drug legislation: lessons for neglected tropical diseases

In the last 20 years, orphan drug legislation (ODL) has been adopted in several countries around the world (USA, Japan, Australia, and the European Union) and has successfully promoted R&D investments to develop new pharmaceutical products for the treatment of rare diseases. Without these incentives, many life-saving new drugs would have not been developed and produced. For economic reasons, the development of medicines for the treatment of diseases prevalent in the developing world (or tropical diseases) is lagging behind. Among several factors, the low average per-capita income makes pharmaceutical markets in developing countries appear relatively unprofitable and therefore unattractive for R&D-oriented companies. The case of ODL may offer some useful insights and perspectives for the fight against neglected tropical diseases. First, the measures used in ODL may also be effective in boosting R&D for neglected tropical diseases, if appropriately adapted to this market. Second, small-sized companies, which have played a successful role in the development of orphan drugs for rare diseases, may also represent a good business strategy for the case of tropical diseases.

Avoiding adverse drug reactions by pharmacogenetic testing: A systematic review of the economic evidence in the case of TPMT and AZA-induced side effects

OBJECTIVES The study aims at evaluating the economic evidence related to testing for genetic variants of the drug-metabolizing enzyme, TPMT. Detecting TPMT genetic variants before the administration of azathioprine (AZA) has the potential to prevent serious and costly adverse drug reactions (ADRs), such as neutropenia. In particular, our analysis concentrated on assessing the reliability of data on costs of neutropenia and performing the tests, the two main cost categories that could inform an economic evaluation of TPMT pharmacogenetic testing. METHODS A systematic literature review was performed to gather evidence on the costs of testing and neutropenia. Articles were critically appraised for their comprehensiveness and quality. To better estimate costs of TPMT tests, a small-scale survey of European diagnostic laboratories was conducted. RESULTS Only seven articles were retrieved specifying the costs associated with the management and treatment of AZA-induced neutropenia. Most of these studies are based on theoretical modeling reconstructed with key-informants or on very few cases of ADRs, and either the methodology for cost calculation is not specified or costs are based on national cost databases and tariffs. After critical appraisal of these studies, we considered 2,116 euros as the most reliable estimate for the cost of a case of neutropenia. Literature review accompanied by the survey of several diagnostic laboratories also provided an estimate (68 euros) for TPMT testing. Based on these values, the net cost per prevented case of neutropenia equals to 5,300 euros. CONCLUSIONS Solid economic considerations related to TPMT pharmacogenetic testing are still limited by underreporting of ADRs and high level of approximation related to cost data. Ad hoc observational studies and the ADR recording process embedded in pharmacovigilance systems, established across Europe, should represent more reliable sources of cost data in the future.

Sustaining Universal Health Coverage: The Interaction of Social, Political, and Economic Sustainability

The sustainability of health care systems, particularly those supporting universal health care, is a matter of current discussion among policymakers and scholars. In this article, we summarize the controversies around the economic sustainability of health care. We attempt to extend the debate by including a more comprehensive conceptualization of sustainability in relation to health care systems and by examining the dimensions of social and political sustainability. In conclusion, we argue that policymakers when taking decisions around universal health care should carefully consider issues of social, political, and economic sustainability, their interaction, and often their inherent trade-offs.

Genetic Screening for the Predisposition to Venous Thromboembolism: A Cost-Utility Analysis of Clinical Practice in the Italian Health Care System

OBJECTIVES In the Italian health care system, genetic tests for factor V Leiden and factor II are routinely prescribed to assess the predisposition to venous thromboembolism (VTE) of women who request oral contraception. With specific reference to two subpopulations of women already at risk (i.e., familial history or previous event of VTE), the study aimed to assess whether current screening practices in Italy are cost-effective. METHODS Two decisional models accrued costs and quality-adjusted life-years (QALY) annually from the perspective of the National Health Service. The two models were derived from a decision analysis exercise concerning testing practices and consequent prescribing behavior for oral contraception conducted with 250 Italian gynecologists. Health care costs were compiled on the basis of 10-year hospital discharge records and the activities of a thrombosis center. Whenever possible, input data were based on the Italian context; otherwise, the data were taken from the international literature. RESULTS Current testing practices on women with a familial history of VTE generate an incremental cost-effectiveness ratio of €72,412/QALY, which is well above the acceptable threshold of cost-effectiveness of €40,000 to €50,000/QALY. In the case of women with a previous event of VTE, the most frequently used testing strategy is cost-ineffective and leads to an overall loss of QALY. CONCLUSIONS This study represents the first attempt to conduct a cost-utility analysis of genetic screening practices for the predisposition to VTE in the Italian setting. The results indicate that there is an urgent need to better monitor the indications for which tests for factor V Leiden and factor II are prescribed.

Multiprofessional Primary Care Units: What Affects the Clinical Performance of Italian General Practitioners?

Multiprofessional primary care models promise to deliver better care and reduce waste. This study evaluates the impact of such a model, the primary care unit (PCU), on three outcomes. A multilevel analysis within a “pre- and post-PCU” study design and a cross-sectional analysis were conducted on 215 PCUs located in the Emilia-Romagna region in Italy. Seven dimensions captured a set of processes and services characterizing a well-functioning PCU, or its degree of vitality. The impact of each dimension on outcomes was evaluated. The analyses show that certain dimensions of PCU vitality (i.e., the possibility for general practitioners to meet and share patients) can lead to better outcomes. However, dimensions related to the interaction and the joint works of general practitioners with other professionals tend not to have a significant or positive impact. This suggests that more effort needs to be invested to realize all the potential benefits of the PCU’s multiprofessional approach to care.

The interplay between policy guidelines and local dynamics in shaping the scope of networks: the experience of the Italian Departments of Mental Health

In the mental health field, the creation of networks that can guarantee the smooth coordination of services and organizations across sectors is a priority in the policy agenda of several countries. In Italy, Departments of Mental Health (DMHs) have been designated responsible for the system of specialist mental health services, and also mandated as the conveners and leaders of interorganizational and cross-sectoral networks, by a system-wide reform. This study aims to understand how mental health networks have been assembled in this context and the factors and motivations that have shaped their scope. By combining an analysis of policies with a survey of DMH directors, we have determined that DMHs have preferentially formed collaborative relationships with social service providers (local governments) and the voluntary sector. In contrast, relationships with substance abuse and addiction services and primary care providers were weak and stifled by a lack of trust and by conflict about respective contributions to mental care. We explore the reasons for this selectivity in interorganizational relationships and propose that a lack of targeted incentives in policy guidelines, on the one hand, and the existence of a mandated network leadership, on the other, have led to a rather narrow range of collaborations.

PAR29 AZATHIOPURINE, TPMT POLYMORPHISMS AND ADRS: THE COST OF NEUTROPENIA

we have chosen the Deviance Information Criteria (DIC). The improvement in the deviance of the model for the number of principles per patient is of 1790 and in DIC of 1760. The maps reveal geographical inequalities and zones with greater average values. CONCLUSION: The Bayesian hierarchical models have been a very useful tool for incorporating geographical information in the analysis of pharmacological prescription data. They allow mapping spatial components expressing the trend of that geographical variation.

A Neuroscientist–Consumer Alliance to Transform Mental Health Care

The field of mental health has long suffered from a lack of convergence of disciplines that deal with the mind, the brain, and behavior. This mind–brain dualism has been particularly detrimental for consumers and their families who daily face stigma and discrimination. The understanding of the brain and its dysfunctions has benefited from the study of the human genome and, in particular, of the mutations and variations in its code. This analysis permits a better understanding of the biological basis of mental disease and will soon inform a generation of new diagnostic tools and individualized pharmacological therapies. A biological perspective on mental illness will be complemented by the analysis of the social factors influencing peoples behavior and their impact on brain biology and gene function. Neurobiology has progressed to a level for which the knowledge that is generated, even if still colored with uncertainty, could represent a catalyst for the creation of an alliance between neuroscientists and consumers. This partnership has the potential to benefit both parties but will require some concrete steps that might be outside of the usual courses of action for both consumers and scientists. It is by building collaborations based on personal contact and information sharing that a transformation of the mental health care system can occur.