Evaluating health policy and legal responses : how to reduce barriers and improve access to orphan drugs for rare diseases in Canada

Abstract : Rare diseases are debilitating conditions often leading to severe clinical manifestations for affected patients. Orphan drugs have been developed to treat these rare diseases affecting a small number of individuals. Incentives in the legal framework aimed to recoup the research and development cost of orphan drugs for pharmaceutical companies have been implemented in the United States and the European Union. At the present time, Canada is still lacking a legal and policy framework for orphan drugs. Several problems at the federal and provincial levels remain: lack of research funds for rare diseases, discrepancies on orphan drug policies between provinces, difficulties to access and reimburse these high price drugs. Recommendations and measures are proposed, such as a pan-Canadian (national) scientific committee to establish evidence-based guidelines for patients to access orphan drugs uniformly in all provinces with a disease specific registry, a formal agreement for a centralized Canadian public funding reimbursement procedure, and increasing the role of ���guardian��� for prices by the Patented Medicines Review Board in Canada. These recommendations and measures will be beneficial for the implementation of a policy framework for orphan drugs in Canada.

Standardised tobacco packaging:a health policy case study of corporate conflict expansion and adaptation

Objectives: To investigate opposition to standardised tobacco packaging in the UK. To increase understanding of how transnational corporations are adapting to changes in their access to policymakers precipitated by Article 5.3 of the Framework Convention on Tobacco Control (FCTC). Design: Case study web-based documentary analysis, using NVivo V.10. Examination of relationships between opponents of standardised packaging and transnational tobacco companies (TTCs) and of the volume, nature, transparency and timing of their activities. Setting: UK standardised packaging policy debate 2011-2013. Participants: Organisations selected on basis of opposition to, or facilitation thereof, standardised tobacco packaging in the UK; 422 associated documents. Results: Excluding tobacco manufacturing and packaging companies (n=12), 109 organisations were involved in opposing standardised packaging, 82 (75%) of which had a financial relationship with 1 or more TTC. These 82 organisations (43 actively opposing the measure, 39 facilitating opposition) were responsible for 60% of the 404 activities identified, including the majority of public communications and research production. TTCs were directly responsible for 28% of total activities, predominantly direct lobbying, but also financially underwrote third party research, communication, mass recruitment and lobbying. Active organisations rarely reported any financial relationship with TTCs when undertaking opposition activities. Conclusions: The multifaceted opposition to standardised packaging was primarily undertaken by third parties with financial relationships with major tobacco manufacturers. Low levels of transparency regarding these links created a misleading impression of diverse and widespread opposition. Countries should strengthen implementation of Article 5.3 of the FCTC by systematically requiring conflict of interest declarations from all organisations participating in political or media debates on tobacco control.

Alcohol consumption among university students: a typology of consumption to aid the tailoring of effective public health policy

Objective: Elevated levels of alcohol consumption among university students are well documented. Policymakers have attempted to combat this issue at a university, national and international level. Tailoring public health policy to effectively tackle alcohol use is crucial. Using Q-methodology, the current study aims to develop a typology of alcohol consumption in the Irish university student population. Setting: A large Irish university. Participants Hundreds of possible statements on types of consumption were generated from a systematic review and a set of one-on-one interviews. These were reduced to 36 statements, 6 statements which define each of the 6 previously defined consumption types. Participants were advised to scan through the 36 statements and fill the statements into a ���forced choice, standardised distribution���. Following this, a 45���90 min interview was conducted with students to illuminate subjectivity surrounding alcohol consumption. Analysis was conducted using PQ Method and NVivo software. Principal component analysis, followed by varimax rotation, was conducted to uncover the final factor information. Results: In total, 43 students completed the Q-study: 19 men and 24 women. A typology describing 4 distinct groupings of alcohol consumer was uncovered: the guarded drinker, the calculated hedonist, the peer-influenced drinker and the inevitable binger. Factor loadings of each of the consumer groupings were noted for type description. Conclusions: This is the first study to propose ideal types of alcohol consumption among a university student population. Further research is required to investigate the degree to which each of these ideal types is subscribed. However, this typology, in addition to informing public policy and strategies, will be a valuable analytic tool in future research.

Language policy spread: Learning from health and social policy models

Except for a few large scale projects, language planners have tended to talk and argue among themselves rather than to see language policy development as an inherently political process. A comparison with a social policy example, taken from the United States, suggests that it is important to understand the problem and to develop solutions in the context of the political process, as this is where decisions will ultimately be made.

The role of public policy in health care promotion: a comparative perspective

Comunica����o apresentada na 17th International conference on Health Promotio Hospitals and Health Services em Hersonissis, Crete, Gr��cia de 6-8 de maio de 2009

Health system responses to financial pressures in Ireland: policy options in an international context

Given the scale of the challenge facing the health system for 2013 and subsequent years, the Department of Health invited the European Observatory on Health Systems and Policies to prepare a report on the implications for the Irish health system of our current financial pressures. The Observatory is an international partnership hosted by the World Health Organisation (WHO). The partnership includes three other international agencies (European Commission, the European Investment Bank, World Bank), several national and decentralized governments, including Ireland, and academic institutions. As an independent and neutral knowledge broker the Observatory's core mission is to inform policy-making and decision-making processes by providing tailored, timely and reliable evidence on health policy and health systems. Click here to download PDF 2.1mb

Co-evolution of Policy Sectors: Health Care and Public Health in five Countries

Cette th��se analyse la co-��volution de deux secteurs dans la politique de la sant��: sant�� publique (public health) et soins aux malades (health care). En d'autres termes, la relation entre les dimensions curative et pr��ventive de la politique de la sant�� et leur d��veloppement dans la dur��e. Une telle recherche est n��cessaire car les probl��mes de la sant�� sont complexes et ont besoin de solutions coordonn��es. De plus, les d��penses de la sant�� ont augment�� sans arrt durant les derni��res d��cennies. Un moyen de r��duire une future augmentation des d��penses pourrait consister en davantage d'investissement dans des mesures pr��ventives. En relation avec cette id��e, ma recherche analyse les politiques de la sant�� publique et les soins aux malades de cinq pays: Allemagne, Angleterre, Australie, Etats-Unis et Suisse. En m'appuyant sur la litt��rature secondaire, des statistiques descriptives et des entretiens avec des experts et des politiciens, j'analyse la relation entre les deux secteurs depuis la fin du dix-neuvi��me si��cle. En particulier, je me focalise sur la relation des deux champs sur trois niveaux: institutions, acteurs et politiques. Mes r��sultats montrent les similitudes et les diff��rences d'��volution entre les cinq pays. D'un c^ot��, lorsque la profession m��dicale est politiquement active et que le pays consiste en une f��d��ration centralis��e ou en un gouvernement unitaire, les deux secteurs sont int��gr��s au niveau institutionnel, ralliant les professions et groupes d'int��r��t des deux secteurs la cause commune dans une activit�� politique. Par contre, dans tous les pays, les deux secteurs ont co-��volu�� vers une compl��mentarit�� malgr�� de la politisation des professions et la centralisation du gouvernement. Ces r��sultats sont int��ressants pour la science politique en g��n��ral car ils soulignent l'importance des professions pour le d��veloppement institutionnel et proposent un cadre pour l'analyse de la co-��volution des politiques publiques en g��n��ral. -- This Ph.D. thesis analyzes the co-evolution of the health care and the public health sectors. In other words, the relation between preventive and curative health policy and its evolution over time. Such research is necessary, because current health problems are complex and might need coordinated solutions. What is more, health expenditures have increased continuously in the last decades. One way to slow down further increase in health spending could be to invest more in preventative health policies. Therefore, I am connecting individual health care and public health into a common analysis, taking Australia, Germany, Switzerland, the UK and the U.S. as examples. Based on secondary literature, descriptive statistics and interviews with experts and policymakers, I am analyzing how the two sectors' relations co-evolved between the late nineteenth and the early twenty-first century. Specifically, I am researching how health care and public health were related on the levels of institutions, actors and policies. My results show that there are differences and similarities in the co-evolution of policy sectors between these countries. On the one hand, when the medical profession was politically active and the country a centralized federation or a unitary state, there was institutional integration and common political advocacy of the sectors' interest groups and professions. On the other hand, in all countries, both sectors co-evolved towards complementarity, irrespectively of the politicization of professions and centralization of government. These findings are interesting for the political science literature at large, because they underline the importance of professions for institutional development and propose an analytical framework for analyzing the co-evolution of policy sectors in general.

A model of factors determining levels of public support for health care policy in the United States

Public preferences for policy are formed in a little-understood process that is not adequately described by traditional economic theory of choice. In this paper I suggest that U.S. aggregate support for health reform can be modeled as tradeoffs among a small number of behavioral values and the stage of policy development. The theory underlying the model is based on Samuelson, et al.'s (1986) work and Wilke's (1991) elaboration of it as the Greed/Efficiency/Fairness (GEF) hypothesis of motivation in the management of resource dilemmas, and behavioral economics informed by Kahneman and Thaler's prospect theory. ^ The model developed in this paper employs ordered probit econometric techniques applied to data derived from U.S. polls taken from 1990 to mid-2003 that measured support for health reform proposals. Outcome data are four-tiered Likert counts; independent variables are dummies representing the presence or absence of operationalizations of each behavioral variable, along with an integer representing policy process stage. Marginal effects of each independent variable predict how support levels change on triggering that variable. Model estimation results indicate a vanishingly small likelihood that all coefficients are zero and all variables have signs expected from model theory. ^ Three hypotheses were tested: support will drain from health reform policy as it becomes increasingly well-articulated and approaches enactment; reforms appealing to fairness through universal health coverage will enjoy a higher degree of support than those targeted more narrowly; health reforms calling for government operation of the health finance system will achieve lower support than those that do not. Model results support the first and last hypotheses. Contrary to expectations, universal health care proposals did not provide incremental support beyond those targeted to ���deserving��� populations���children, elderly, working families. In addition, loss of autonomy (e.g. restrictions on choice of care giver) is found to be the ���third rail��� of health reform with significantly-reduced support. When applied to a hypothetical health reform in which an employer-mandated Medical Savings Account policy is the centerpiece, the model predicts support that may be insufficient to enactment. These results indicate that the method developed in the paper may prove valuable to health policy designers. ^

HEALTH STATUS - HEALTH SERVICE RESOURCES - ECONOSOCIOCULTURAL DEVELOPMENT: IMPLICATIONS FOR HEALTH CARE POLICY IN AFRICA

Rational health services planning requires an examination of the effects of various factors on the health status of a population within a given set of socioeconomic circumstances. The commonly accepted explanations for improved health in the less developed countries (LDCs) are: Health Service Resources available to a population, Environmental and Life conditions, and the Econosociocultural Characteristics of the population.^ In the context of the low economic base from which many LDCs initiate development activities, a strong imperative exists for identifying in which of these major areas public health policy would be most effective in terms of improving health. A new conceptual model is proposed that would be used for future policy analyses to assess what changes in health status of populations in LDCs can be expected as direct functions of increased health service resources, and of improved environmental and econosociocultural conditions.^ While direct policy analysis is ill-advised at this time due to data inadequacy, the model is illustrated using data presently available for twenty-five relatively homogeneous Sub-Sahara African countries. Within the limitations of available data, study findings indicate that while econosociocultural conditions were the most important explanatory factors of the three major independent variables in 1970, health service resources became the most important in 1975. Study findings are inconclusive at this time with regards to the relative contributions of physicians and medical assistants in explaining variances in mortality in these countries.^ Because of the deficient nature of available data, study findings should be interpreted very cautiously. Tests of statistical significance of study findings were by-passed because of their situational technical inappropriateness. This study is significant in being the first of its kind and scope to focus on the Sub-Sahara African region of the World Health Organization, using the Wroclaw Taxonomic Method in conjunction with a stepwise regression technique. It is desirable, therefore, to examine the observed magnitude and directional consistency of all hypothesized relationships, even if evidence is inconclusive. ^

Regional health governance: a comparative perspective on EU and ASEAN. EU Centre in Singapore Policy Brief No. 4, June 2012

Globalisation has led to new health challenges for the 21st Century. These challenges have transnational implications and involve a large range of actors and stakeholders. National governments no longer hold the sole responsibility for the health of their people. These changes in health trends have led to the rise of Global Health Governance as a theoretical notion for health policy-making. The Southeast Asian region is particularly prone to public health threats and it is for this reason that this brief looks at the potential of the Association of Southeast Asian Nations (ASEAN) as a regional organisation to take a lead in health cooperation. Through a comparative study between the regional mechanisms for health cooperation of the European Union (EU) and ASEAN, we look at how ASEAN could maximise its potential as a global health actor. Regional institutions and a network of civil society organisations are crucial in relaying global initiatives for health, and ensuring their effective implementation at the national level. While the EU benefits from higher degrees of integration and involvement in the sector of health policy making, ASEAN���s role as a regional body for health governance will depend both on greater horizontal and vertical regional integration through enhanced regional mechanisms and a wider matrix of cooperation.

In sickness and in health: protecting and supporting public investment in Europe. Bruegel Policy Contribution 2014/02, February 2014

The long-term decline in gross public investment in European Union countries mirrors the trend in other advanced economies, but recent developments have been different: public investment has increased elsewhere, but in the EU it has declined and even collapsed in the most vulnerable countries, exaggerating the output fall. The provisions in the EU fiscal framework to support public investment are very weak.The recently inserted ���investment clause��� is almost no help. In the short term, exclusion of national co-funding of EU-supported investments from the fiscal indicators considered in the Stability and Growth Pact would be sensible. In the medium term, the EU fiscal framework should be extended with an asymmetric ���golden rule��� to further protect public investment in bad times, while limiting adverse incentives in good times. During a downturn, a European investment programme is needed and the European Semester should encourage greater investment by member states with healthy public finances and low public investment rates. Reform and harmonisation of budgeting, accounting, transparency and project assessment is also needed to improve the quality of public investment.

Value of health - too often ignored and forgotten by EU policy-makers? EPC Commentary, 29 September 2014

Whether on national or European level, policy-makers tend to under-value health, healthy society, and healthy citizens in policy-making. As the European Commission continue to ponder how questions related to health should be reflected in EU policy-making and what role it should take, there are three issues to keep in mind: 1) there is a need to recognise health as a value, 2) health should be considered across policies, 3) the EU has the tools to promote a healthier European society.

Keeping health high on the EU agenda: role for economic governance? EPC Policy Brief, 7 May 2015

In an attempt to get Europe out of the economic crisis and establish right conditions for growth, the EU coordinates and monitors member states��� economic and budgetary policies via a system called the European Semester. As member states��� spending on the health sector accounts for 10% of GDP and is expected to grow, it is no wonder that an increasing emphasis has been paid to sustainability of health systems ��� an area that is traditionally considered as a national competence. In this Policy Brief, Annika Hedberg and Martina Morosi reflect on the strengths and weaknesses of the European Semester and country-specific recommendations in promoting more sustainable and efficient health systems in Europe, and why the EU must continue to play a role in encouraging member states to value health and improve their spending on health.