Pharmaceuticals Policy and Law - Volume 7, issue 0
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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: Haemophilia is an x-chromosomal linked, hereditary lifelong disease which causes damages of bones and joints as well as early death. Per definition there are three different forms, the severe form (less than 1% of coagulation factor activity) demands regular substitution therapy with the missing coagulation factor. Haemophilia therapy developed since the 1960ies rapidly and today safe clotting factor concentrates are available, plasma – derived as well as recombinant. Nevertheless some 80% of the patients…affected do not receive treatment at all and only some 5 to 10% receive the so called "state of the art" treatment of their bleeding disorder. Non- viral- inactivated- clotting factor concentrates caused an epidemic of HIV/Aids within this population in the 1970ies and 1980ies, with deaths of some 50% (in some Haemophilia Treatment Centers even higher) of continuous treated patients. This fatal experience promoted research not only in gene- and biotechnology, but, despite of the early promises of the involved scientists and industries, a satisfying solution for Persons with Haemophilia is still missing. Today plasma derived clotting factor concentrates have to undergo various viral- inactivation- procedures, donor screening and pool- as well as final product testing is essential, strict legislation on blood safety issues became standard in most countries.
Abstract: Medical knowledge on the clinical investigation of human normal immunoglobulin for intravenous administration and treatment regimens are in a state of continuous development. Due to recent achievements, the question arises whether the currently established indications and their respective posologies might require modification to more adequately reflect current clinical practice with intravenous or subcutaneous immunoglobulins as substitutive therapy in patients affected by primary and secondary immunodeficiencies and as immunomodulators in patients affected by…autoimmune disorders. In addition, immunoglobulin therapies used "off label" have proved beneficial and other possible inclusion will be expected in the next future. In this review we will identify the clinical and immunological benefits and limits of imunoglobulin treatments and outline the physician's persepective on this topic.
Abstract: Plasma products are used to treat a number of serious inherited disorders, of which the best known is haemophilia. However, a number of other conditions exist at a similar frequency in the population but are much less well known: Alpha 1-Antitrypsin (AAT) deficiency or 'Alpha-1' is one of these. Alpha-1 is a life-threatening genetic disorder in some individuals who demonstrate clinical evidence of emphysema. Left untreated, this form of emphysema can be fatal. Historically, people with…Alpha-1 have been concerned with the critical short supply of the therapy. Recent product approvals will provide the desperately needed product, so people with Alpha-1 can focus more on their lives and less on when their therapy will be available.
Abstract: Human plasma is used to prepare essential therapeutic products with unique features compared to traditional pharmaceutical products and other biopharmaceuticals. The raw material is inherently unique due to its human origin. The need to fulfill strict collection practices and testing, and the shrinking population of eligible donors, limit plasma availability and influence greatly the economics of the whole plasma industry. Consolidation in the industry has amplified and is making access to plasma, a highly valuable…natural commodity, more difficult. In this industry, plasma has become the single most important factor of the cost of end-products. In addition, the fractionation of human plasma is a "cracking" process where manufacturing technologies must allow extracting an economically balanced portfolio of products with appropriate quality, safety, and recovery profiles. Over the years, although the size of production batches has generally increased (within limits accepted by regulatory authorities), the manufacturing costs of plasma products have increased as production methods have become more complex (in particular with the introduction of viral reduction treatments) and product recovery has been affected negatively (especially for coagulation factors). Therefore (a) ensuring the production of at least 3 to 4 products from each batch of plasma and (b) economies of scale have been leading to major consolidation in this industry to ensure appropriate allocation of plasma and manufacturing costs. Efforts need to be pursued to improve the efficiency of the fractionation process, improve proteins recovery, and expand the universe of ailments treatable by niche plasma products to enhance plasma utilization and cost-sharing potentials. Historically, the supply of plasma products has never met the global needs and has averaged a 20–80% deficiency. To date, the inherent economical requirement to ensure a production balance of at least 3 or 4 products from plasma implies that with current fractionation technologies and current reimbursement practices, shortage of market-leading products, like immunoglobulins or alpha 1-antitrypsin, will continue if not worsen.
Abstract: The French transfusion organization is based on principles and structures; the principles are: voluntary, anonymous, non-remunerated donations, focussing on self-sufficiency. There are three structures: the EFS (Etablissement Français du Sang – French Blood Establishment), for labile blood products; the LFB (Laboratoire Français du Fractionnement et des Biotechnologies – French Laboratory for Fractionation and Biotechnologies), for blood derivatives; the INTS (Institut National de la Transfusion Sanguine – Blood Transfusion National Institute) for…activities such as Reference, Research, Education & Training. The French health policy aims at defending ethics and self-sufficiency for all human blood derivatives. The French self-sufficiency is limited to cellular products as France is considered a rare user compared with other European countries � its annual growth rate is of about 1% only. Situation looks quite different with blood derivatives: between 2001 and 2004, consumptions of albumin, clotting factors, and immunoglobulins have shown an average annual increasement of 6.1% to 9.7%. All these elements have to be appreciated bearing in mind that beyond the 10 years ahead no substitute at all will be able to replace the human blood, making it a full necessity.
Abstract: Plasma protein therapies are either manufactured using human plasma as starting material or through the use of alternative technologies to produce recombinant plasma protein therapies. Most of the chapter is written with a focus on the plasma that is used for the manufacture of stable therapies.