Pharmaceuticals Policy and Law - Volume 11, issue 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: The application of evidence-based medicine to the indications addressed by plasma protein therapies is problematic. So-called Level 1 evidence in the form of randomized clinical trials using classical designs to demonstrate efficacy is seldom possible, because the small numbers of patients denies the appropriate level of power from being built in the trial design. In addition, patient accrual is difficult because of understandable patient resistance and "fatigue" at the number of trials. Efforts to address this…by regulating agencies over the past years have yielded promising policies in some areas, which recognize the inherent problems and outline practical solutions. The use of patient registries to accrue safety and efficacy data, while rapidly becoming accepting an accepted component as a basis for pre-market review and approval, is also promising as a basis for the incorporation of post-market data, through Phase 4 surveys, of approved therapies. As such, innovative thinking to address the problem of rare disorders may also lead to desirable reform in all aspects of regulation, with a shift from pre- to post-market review.
Abstract: Background: Blood and plasma (b&p) are crucial but scarce resources for medical therapies. Collecting b&p poses technical and ethical challenges, as can be observed when it comes to regulation. Objective: To provide ethical orientation for organizing and regulating the donation and collection of b&p. Method: Analysis of ethical theories in regard to their potentials to reach the objective. Results: The ongoing ethical debate between paid vs. unpaid b&p donation is…shaped by a 'simple b&p ethics' approach that focuses on the individual donor and the existence or non-existence of one motivation: altruism. Utilitarian and deontological ethics come to different conclusions but basically apply the same simple approach. In contrast, forms of 'complex b&p ethics' acknowledge the ambiguity of altruism, try to find a common framework for the plurality of motives to give b&p (contractualism), or point out that giving b&p is part of a good life (virtue ethics). Complex b&p ethics furthermore widens the perspective from the individual donor to the collecting organization and the donation/collection regime and critically assesses them. Conclusion: Neither 'gift fetishism' nor total commodification seem to be ethically sound ways of enabling people to live good lives. Those engaged in b&p collection will be well advised to acknowledge the complex plurality of motives while simultaneously upholding the caring nature of b&p donation, collection, and distribution.
Keywords: Blood donors, plasma, ethics, altruism, gift giving
Abstract: FDA encourages the development of products that demonstrate promise for the treatment of rare plasma protein disorders. This is accomplished through a program of regulatory provisions, financial incentives, clinical trials designed to address small patient populations, and measures to increase the number of patients under study.
Keywords: Orphan drug act, rare plasma protein disorders