Development of Medicines
The development of new medicines is a long, expensive and complex process. It can take 13 years from discovering a molecule to having a medicine approved for human use. In recent years, the pharmaceutical industry has made steps towards re-aligning its pipeline to society’s needs. In 2011, 37 non-orphan medicines were made available in Europe, including two new targeted treatments for melanoma which can help patients who had few treatment options in the past. 12 new orphan drugs were also approved. Orphan medicinal products are used for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons in the European Union.
Before being able to reach the patient, a medicine needs to demonstrate its safety, quality and efficacy with medicines regulators. The European Medicines Agency (EMA) is a key institution for regulatory approval of medicines in the EU, and has exclusive prerogative for this function in the area of biologics, whilst national authorities in member states are still active in other areas of medicines approvals. The exciting new research developments in biologics, personalized medicines and the genome mean that approvals processes need updating to keep pace with this new science, for example when it comes to inclusion of biomarkers and diagnostics as part of the approval process. Increasingly also, national decision-makers ask for information about the added value of new medicines for individual patients and society as a whole. Industry is committed to demonstrating the added value of its medicines with high quality, transparent data and considers that Health Technology Assessments (HTAs) when based on sound methodologies and input from all relevant stakeholders can be used as a tool to support efficient healthcare decision-making. Industry is and will continue to be involved in collaboration on HTA among EU Member States, to promote good practices and efficiencies.
The EU has a key role in ensuring that the regulatory framework for approving new medicines, and for the necessary clinical trials, are as stream-lined as possible, in order to get innovative medicines to the patient as quickly as possible. The EU’s coordination at international level with the USA is also key, in avoiding needless duplication of procedures due to differing requirements for clinical trials. Moreover, as new technologies such as personalised medicines advance, it will be important to have a flexible system of approvals and clinical trials, which can be updated according to new research.
A sensitive, but important, element in the development of medicines, is the use of animals in research. Modern day medicines would not exist without research in disease development. Nor without the necessary and complex testing processes involved in bringing them to market. Similarly, new medicines for conditions for which no effective treatment exists, cannot emerge without research and development. Some of this research involves testing on animals, which isalso required and regulated by European law. The industry has adopted the 3Rs principles – Replace-Reduce-Refine – in order to minimise the use or impact on animals wherever possible.
Incentivising innovation is a fundamental factor in ensuring sustainable innovation in the pharmaceutical sector. The increasing demands of an ageing population, coupled with falling public budgets have put pressure on the pharmaceutical industry. Whilst the industry understands the need to share the pain, the risk is that unilateral price cuts and unpaid bills, undermine the industry’s ability to continue producing innovative medicines in the long term. It is therefore important that for those medicines that society needs and wants, there should be a fair return on the substantial investment made in R&D.