Marketing authorisation programme

Such is the emphasis on the scientific development of medicines for paediatric use that Section 4 of the application for marketing authorisation specifically requires the applicant to state whether or not there is a paediatric development programme. Following the Council Resolution of December 2000, in February 2002 the EC published a consultation paper on Better Medicines for Children - proposed regulatory actions in... 

Once the clinical and safely evaluation studies for a new medicinal product have shown it to be safe, effective and of acceptable quality, the pharmaceutical company will usually want to submit a Marketing Authorisation Application (MAA) or New Drug Application (NDA) to the regulatory authorities. The chemistry, manufacturing and controls (CMC) section will form a major part of the application. For an MAA in Europe, a development pharmaceutics section is required to describe how the product was developed, and to explain the rationale for the selection of the formulation, pack, manufacturing process and specifications. Also required for Europe are expert reports for each of the pharmaceutical, safety and clinical parts of the application. These have to be written by experienced scientists nominated by the pharmaceutical company who have to critically appraise the development programme for the product. The pharmaceutical expert must acknowledge the acceptability of the CMC part of the application. 

Medicines are available for patients as authorised medicines or as pharmacy preparations (unlicensed medicines). Market logic ensures that only medicines with sufficient return on investment will be marketed. However, health care logic requires pharmacists to provide their patients with necessary medicines. There are regulations that cover medicines for clinical research, marketing authorisation and import, as well as traffic between European countries (parallel imports). If medicines are not available as authorised medicines, various options such as compassionate use or parallel trial programme can be considered. The authorisation of medicines for orphan diseases is promoted by the orphan dmg regulations. The system of reimbursement will be discussed briefly due to its special situation at the interface of both public health and social insurances. 

Compassionate use is a treatment option that allows the use of an unlicensed medicine. Compassionate use programmes are for patients in the European Union (EU) who have a disease with no satisfactory authorised therapies or cannot enter a clinical trial. They are intended to facilitate the availability to patients of new treatment options under development. To qualify for a compassionate use programme, the manufacturer calls on the national authorities for permission. The manufacturer must submit a request for the granting of a marketing authorisation or he must perform research in the context of a research programme with a cohort. Compassionate use procedures are also applicable for unlicensed medicines withdrawn from the market or for off-label use of licensed medicines. 

The legislation makes specific provision for the time between authorisation and placing on the market. In some Member States, where it is necessary to obtain approval for reimbursement, this may entail a delay of some months. During this time, where a compassionate-use programme has previously been set up, article 84.8 states that the applicant shall ensure that patients taking part also have access to the new medicinal product. ... 

In the United Kingdom, manufacturers who provide special products must have an authorisation allowing special manufacture. A wholesale distributor will not be in breach of wholesale distribution rules and conditions if he handles a special product, provided that there is no departure from the terms of the rules. The exclusions facilitate compassionate use programmes where product is not authorised, either because it is still at the experimental stage or because it has been withdrawn from the market (for commercial or for safety reasons) or the product may simply never have been intended for full-scale... 

Promotion should not be disguised. Clinical assessments, post-marketing surveillance and experience programmes, and post-authorisation studies must not be disguised promotion. Such assessments, programmes and studies must be conducted with a primarily scientific or educational purpose. Material relating to pharmaceutical products and their uses, whether or not promotional in nature, that is sponsored by a company should clearly indicate by whom it has been sponsored. 

Clinical assessments, post-marketing surveillance, experience programmes and post-authorisation studies must not constitute a disguised promotion. Such assessments, programmes and studies must be performed mainly for a scientific or educational purpose. 

Several options to get authorisations for named patients have been applied in the past and still are in practice in order to maintain the supply chain with the most important medicines. As long as they are classified as IMPs and thus not allowed on the market, patients may be treated in some countries, e.g. Switzerland, in a parallel trial programme or within an extended access. Procedures follow those for compassionate use and requests have to be submitted to the ethical committee as well. A parallel trial programme will always require Ethical Approval and would therefore be a Clinical trial in the UK. Single cases different from compassionate use have to be authorised but do not need an ethical committee approval. 

Existing active substances that have been identified only (Annex III) will not be evaluated within the review programme and will not be included in Annex I, lA or IB of the Biocidal Products Directive. The same applies to any existing active substance/product-type combination of which a notification has not been accepted. The effective date from which Member States shall cancel existing authorisations and registrations for biocidal products containing the substances listed in Annex III and from which such biocidal products may not placed on the market is September 1, 2006. 

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