Masterstudiengang "Drug Regulatory Affairs"
Master-Thesis
"Earlier access to medicines" EAMS in the UK: A comprehensive overview and comparison to existing accelerated licensing procedures in the EU and Germany ***
Jan Horn (Abschlußjahr: 2015)
Summary
Language: English
The thesis provides a review of the new UK Early Access to Medicines Scheme (EAMS) essentials in context of existing early access schemes and licensing flexibilities for medicine products in Europe, in particular, a comparison of the corresponding scheme in Germany in regard to legal basis, regulatory requirements, applicability, and time lines, advantages for patients and industry, and reimbursement issues. Consequently implementation options for EAMS parts to regular licensing procedures are focused.
On 7th April 2014 on current EU and national legislation based the two-step EAMS has been launched, intended to give patients suffering from life threatening or seriously debilitating conditions early access to medicines, which are used off-label or do not yet have a marketing authorisation, in case of clear unmet medical need identified. Abreast, it is considered enhancing the UK landscape for developing, licensing, and procuring innovative medicines by reflection of the profound changes driven by genomics, data, and the rise of stratified and personalised medicines transforming the drug landscape from traditional blockbuster models to the world of translational or experimental medicines where drugs are designed with and around patient data in clinical research and hospitals.
Positioned between phase III, eventually phase II and final marketing approval, once introduced the 3rd step health technology appraisal and reimbursement, it can be regarded extended to licensing and rapid commissioning, reducing local variations in medicines access by provision of a clear regulatory opinion to a nation-wide approach. The scheme complements the European licensing landscape and can support adaptive licencing pathways via generation of real-life data on safety and efficacy out of clinical trials. Bridging the licensing period, it potentially provides life-saving treatment options one to two years (some expect 5 years) earlier than regular approval procedures. More insight and understanding of the disease, the efficacy, safety and quality of the medicines given at the earliest stage the evidence collection for health technology appraisal purposes can start earlier. Early assessment start at phase II, the short assessment period, and several scientific advices implemented, as well as the early CTD compilation fortunately for later marketing application use clearly provides advantages. In respect to prescribers and pharmacists liabilities for prescribing, dispensing and preparing the medicine still unlicensed, the EAMS Opinion inherent treatment protocol issued held as a reference for justification can accelerate treatment uptake. Premises for EU exceptional/conditional marketing authorisation applications narrow the subset of medicine products eligible dramatically to about 10% of centralised applications. The flexibilities provided with the EAMS potentially can overcome these limitations for patient access to medicines making available the medicines before granting (full) license. The possibility of intellectual property rights or data protection periods erosion discussed as regards restricted central licensing schemes in contrast remains unaffected by EAMS or compassionate use schemes.
The scheme only allows UK based patient earlier access to unmet medical need medicines but not EU citizens. The impact on the latter will have to be monitored. From stakeholder perspective the scheme is complex, also expert advice is recommended at various stages. It is expected that experience will help to optimise the scheme and certainly will have an impact on decisions in other European countries or Europe as to discuss and adopt similar ideas.
Pages: 67, Annexes: 2, Pages: 80