What is the ‘hospital exemption’, and why does it matter?
The impact on Advanced Therapy Medicinal Products (ATMPs) is one of the key emerging flashpoints of European Commission proposals to revise the EU’s pharmaceutical legislation which were tabled in April last year.
ATMPs are medicines for human use based on genes, tissues or cells, and are mainly used to treat rare diseases, 80% of which are genetic and can be targeted by these innovative treatments. Although ATMPs have transformed treatment of rare diseases, high production and research costs make them inaccessible for many patients.
Of 18 gene therapies authorised for EU use in recent years, only 15 remain on the market with three companies withdrawing products from market due to lack of profitability.
To counter these challenges, in 2007 the EU introduced the principle of hospital exemption (HE) to regulation for ATMPs, allowing some treatments to be used without marketing authorisation under specific circumstances.
To qualify for the exemption, ATMPs must be prepared - on a non-routine basis - in a hospital setting, be intended for individual patients, and be used within the same member state, and there must be no centrally authorised treatment or clinical trial available for the drug.
The proposed new pharmaceutical regulation embellishes the 2007 text, introducing requirements for annual collation, reporting and reviewing of data by competent national authorities and publication by the European Medicines Agency (EMA) in a central repository.
The International Society for Cell and Gene Therapies warned in 2022 that enforcement of the HE rule at member state level has led to divergent implementation across the EU.
HE use varies significantly across member states depending on national legal implementation, local policy makers’ interpretations, clarity of guidance at the national level, reimbursement opportunities and the level of ATMP research and development activities carried out by domestic academic and commercial organizations.
The study also concluded that “with important variations in how quality, safety and efficacy standards are implemented and controlled across EU Member States for ATMPs provided via the HE rule and a lack of transparency around its use, the HE rule draws concern around its potential impact on public health”.
The European Commission also recognises in the new proposal that “experience has shown that there are great differences in the application of hospital exemption among member states” and calls on the EMA to provide a report on the implementation of hospital exemption with information from national systems.
The European Parliament added later a special focus on the evidence on quality, safety and expected efficacy of the ATMPs when presenting the application for hospital exemption.
