Pharmaceuticals Policy and Law - Volume 12, issue 1,2
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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 orphan status of a medicinal product in Europe is regulated by Reg (EC) No 141/2000 and Reg (EC) No 847/2000 that states that: "Persons suffering from rare conditions should be entitled to the same quality of treatment as other patients" but "the pharmaceutical industry would be unwilling to develop the medicinal product under normal market conditions" as "some conditions occur so infrequently that the cost of developing and bringing to the market a medicinal product ……would not be recovered by the expected sales".
Abstract: The orphan designation procedure relates to medicinal products for human use aimed at the treatment, prevention or diagnosis of diseases. It is a free of charge procedure and can be requested at any stage of the development by a sponsor who can be either a company or an individual, as long as is established in EU, Iceland, Liechtenstein, and Norway. Once the Committee for Orphan Medicinal Products (COMP) adopts opinion on designation, the European Commission grants…a decision that gives access to the incentives.
Abstract: Having achieved orphan designation, sponsors complete clinical investigations and submit the outcome of these investigations to the regulatory authorities to gain marketing authorisations. The (mandated) centralised review process for such applications is similar to that for non-orphan applications, relying on demonstration of adequate product quality, clinical safety and efficacy. However, due to the rarity of the condition under investigation it is likely that the data package provided with respect to clinical safety and…efficacy will be more limited and may lead to the CHMP having to work with the PDCO to assess whether benefit risk has been appropriately demonstrated to the full extent possible and whether conditions should be placed on any marketing authorisation issued due to the limited available evidence. This may lead to conditional or exceptional marketing authorisation approvals where the company is either able or unable (respectively) to eventually provide a complete evidence package. Such approval status for an orphan drug would have to be clearly highlighted in corresponding labelling and would require more regular reporting of safety experience due to a less well documented risk profile for the product. In addition companies may be required to commit to further clinical investigations to the extent permitted by the prevalence of the disease. These post marketing conditions (PMCs) or follow up measures would be reported on a regular basis allowing a more frequent re-assessment of the current benefit risk profile of the product in the disease. Amgen has experience of approval of a non-orphan designated product gaining a conditional approval and of regulatory assessment of a marketing authorisation application for an orphan designated product.
Keywords: Orphan designation, centralized procedure, post marketing condition
Abstract: In 1983, the United State's Congress passed the U.S. Orphan Drug Act, which established public policy that the Federal Government would assist in the development of products for the diagnosis, prevention, or treatment of rare diseases or conditions . The Orphan Drug Act defined an "orphan product" as one that is intended to treat a rare disease or condition that affects fewer than 200,000 people in the United States OR as a product which will not…be profitable within seven years of approval by the FDA. There are over 6,000 conditions that meet the definition of a rare disease. The Orphan Drug Act allows the government to provide a package of financial incentives to companies who decide to undertake the development of products for a rare disease or condition. The Office of Orphan Products Development (OOPD) conducts scientific and regulatory review of orphan product and humanitarian use device designation requests and administers a scientific grant program to defray the cost of clinical studies for orphan product development.
Keywords: FDA, office of orphan products development, orphan designation, orphan drug act
Abstract: The randomised, controlled trial (RTC) is considered the gold standard for the assessment of the effect of any therapeutic intervention. However, rare diseases carry several issues in terms of population size, life-threatening nature of the condition and lack of other available treatments, which basically render this approach not always feasible. Alternative methodological approaches are being developed to better suit the need of an orphan drug development.
Keywords: Randomised, controlled trial, population size, rare disorder
Abstract: Orphan legislation provides incentives to industry to investigate rare conditions that otherwise would be unlikely to be investigated. These incentives include, free access to scientific advice during the development of their clinical programs ('protocol assistance'); reduction in fees payable to EMA for review of the marketing authorisation and subsequent licence maintenance fees; sustained market protection in the form of market exclusivity in addition to a range of national incentives. To qualify for these incentives…companies must apply for orphan designation confirming the seriousness of the condition which has inadequate alternative therapeutic options (for the diagnosis, prevention or treatment) and that the condition qualifies in terms of low prevalence in the European community. The company must also provide evidence of medical plausibility including likely significant medical benefit for patients to be treated with the product for the proposed orphan indication. If an application for orphan designation is not accepted then a company is able to appeal providing detailed grounds for reassessment and will receive a second opinion from the PDCO. The period of market exclusivity awarded by orphan designation will require that future applications received by the EMA for the same condition have to undergo an assessment of similarity to ensure that no variations or new applications for 'similar' medicinal products are granted licences during this ten year period. Products gaining orphan designation provide regular/annual reports regarding their development status, status of global regulatory submissions and any change likely financial returns predicted for the indication.
Abstract: Scientific advice constitutes an important part of the development strategy as it can increase the likelihood of regulatory success. For this reason, its timing has to be carefully planned. There are essentially two routes which can be followed to get scientific advice in the EU: a "national advice", which is a decentralised procedure and involves meeting with one national authority; a "scientific advice" at the CHMP level, which is centralised, called protocol assistance for designated…orphan drugs. This is a pan-European advice, which is adopted by the CHMP based on the recommedations of the SAWP.
Keywords: Scientific advice, centralised procedure, national scientific advice, CHMP, SAWP
Abstract: Evidence from a limited population may be inappropriate to characterise exposure-response relationships and subsequently define dosing recommendations for a wider target group. This limitation is particularly important if standard trial designs and data analysis methods are used to assess pharmacokinetics and pharmacodynamics. Models are a theoretical representation of a phenomenon. According to this definition, PKPD models can be applied to the development of new compounds, enabling inferences about efficacy and safety, which…cannot be obtained otherwise.
Abstract: The European Parliament, and the Council Regulation on Medicinal Products for Paediatric Use, intend to increase children's health in Europe by promoting research, development and authorisation of medicines for paediatric use. The main objectives are: to increase the development of medicine for children to ensure that paediatric medicines are subject to high quality research to ensure that medicines for children are appropriately authorised for paediatric use to improve information available on the…use fo medicines for children to avoid unnecessary studies in children.