Pharmaceuticals Policy and Law - Volume 13, issue 3-4
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The new international review,
Pharmaceuticals Policy and Law, appears with the aim of studying and evaluating the
legal status of medicinal products in the European Union, and its implications in other markets such as the USA and Japan, without neglecting the specific problems of developing countries.
Pharmaceuticals Policy and Law intends to participate in the process of world convergence of pharmaceutical legislation helped by a network of academic centers specializing in pharmaceutical law, without omitting a scientific, economic and social approach to medicinal products.
The specificity of medicinal products conditions their legal status. Legislation regulating other goods cannot be applied to them. To begin with, they are the result of scientific and technical innovation. Research policies determine their progress. The pharmaceutical industry is, by nature, multinational. But, next to these global trends, different traditions still remain at a national level. Within the EU, barriers to free trade in medicinal products still remain despite more than thirty years of harmonisation. The social dimension of medicinal products is complex and very significant in the preoccupations of our societies. Patenting is essential but not sufficient. The life-cycle of medicinal products is protected by professional responsibility, required in the general concept of health safety. It is important to remember their ethical dimension, including research and innovation in new fields such as genetic manipulation and biotechnology, which requires social consent to preserve human dignity and fundamental rights.
Abstract: There is an increasing focus on designing health services that are `patient focussed'. This is also reflected in health technology assessment (HTA), with a growing emphasize on patient perspectives and involvement. The purpose of this article is to discuss why and how patient aspects may be addressed in future HTAs'. While involving patients in the HTA process we argue that the assessments of the included aspects must be based in research to provide both relevant and…valid results that can inform health policy decision makers. Patient aspects need to be explored when a technology, the organisation and/or economic aspects of a technology affect and influence patients in one way or another. "Patients" are in this sense understood as; any current or potential health service user or beneficiary of a health technology, and since illness and the use of technology often involves the family, their perspectives may also be included. When exploring patient aspects in relation to the use of technology it is necessary to reflect on the modes of knowledge. It is important that researchers bear in mind that their understanding of knowledge always have implications in relation to the validity and scope of the research results, they produce.
Abstract: Patients' organisations call for an assessment of the clinical added value of orphan drugs at the time of the marketing authorisation evaluation. The European Commission has commissioned Ernst & Young to explore the feasibility of a new Committee at the European Medicines Agency. A new drug candidate faces a number of hurdles on its journey to reach the patient. Regulators are the first, and right after are the Health Technology Agencies and payers. Things can turn sour, from…a political point of view, when that second scenario happens, when the regulators say "yes this drug is safe and effective" and the payers say "Oh well but we won't reimburse it". Regulators and health technology assessment experts have recently started to harmonise their respective methodologies to avoid some degree of redundancy and contradictions in their respective opinions. Patients are participating in the decision making at the European Medicines Agency, and work closely with some national regulatory agencies. More systematic involvement with health technology assessment agencies is only beginning. This paper illustrates some of the issues patients are discussing with health technology assessment bodies.
Keywords: Health technology assessment, patients' representatives, Quality-Adjusted Life Years, Health Services Accessibility
Abstract: The United States enacted legislation in 2010 that will promote the use of comparative effectiveness research in the making of healthcare decisions. Of concern with this relatively new mandate is the possibility of using comparative effectiveness research as a means only to mitigate costs rather than to focus on quality of care. This is of particular concern for patients with rare and chronic conditions, such as primary immunodeficiency diseases. The Immune Deficiency Foundation (IDF) uses their…survey research to advocate for the needs of patients with primary immunodeficiency diseases to ensure that their unique medical concerns are not overlooked but instead, integrated within an overall healthcare emphasis on personalized medicine and differences in patient treatment response. IDF research shows the efficacy of treatment with immunoglobulin replacement therapy (IG therapy) for many patients with primary immunodeficiency diseases, that IG therapy is underused in the primary immunodeficient community, and that IG therapies are unique and not interchangeable.
Abstract: Epposi is an independent, not-for-profit, partnership-led and multi-stakeholder think tank based in Brussels, Belgium, committed to supporting the realisation of the EU-2020 strategic vision through the health and wellbeing of its citizens. Our Goal is to work at the "cutting edge" of European health policy-making providing members and the wider public with high-quality independent research, capacity-building, knowledge exchange and dissemination with the aim of bridging the gap between innovation and…improved public health outcomes. Membership is equally weighted between academic sciences, industry and patient organisations.
Abstract: HTA is an interdisciplinary assessment of evidence and knowledge about the intended and unintended effects of using a health technology. Patients with rare diseases have valuable knowledge about the illness in the real-life setting, but too often their views are seen as anecdotal or biased. So, more needs to be done to elicit patients' perspectives to add value to HTA through effective participation of patients throughout the HTA process and collection of evidence about patients' perspectives…through robust qualitative research. Traditionally HTA has been a broad assessment to move evidence into practice, but in recent years a more limited view of clinical and cost effectiveness has been the focus. For HTAs in rare diseases, this is not enough. Consideration of ethical, organizational and social issues are vital and here patients' perspectives could be particularly valuable to bring a real-life understanding of the potential impact of the health technology. As countries around the world put more emphasis on creating clear plans to manage rare diseases, we need to ensure that all stakeholders work together to ensure that HTAs are being used flexibly to ensure that there is equity of access to therapies for rare diseases that provide real added value.