Hospital Exemption; still a big risk for pharma

Written by: Angeliki Meletsi; Analyst

Contact: [email protected]

The European Commission proposals on the EU pharmaceutical legislation revision, announced in April 2023, have not included any significant changes to the hospital exemption (HE) provision.

The HE legislation was introduced by the European Commission (EC) in 2007, to enhance patients’ access to Advanced Therapy Medicinal Products (ATMPs). This legislation allows for the use of an ATMP without a marketing authorisation, in a hospital, under the exclusive responsibility of a medical practitioner. Products that are being granted HE are usually indicated for diseases that are characterised by a high unmet clinical need and can only be used in a hospital setting on a non-routine basis for an individual patient and when no centrally authorised treatment or clinical trial is available.

ATMP manufacturers have expressed their concerns around the lack of clear HE guidance which specifies that this legislation should not be used when an authorised ATMP is available for a given indication or in case the patient could have access to an investigational medicine in an ongoing clinical trial. Also, manufacturers have highlighted that the HE should not be granted in products that have established large-scale, industrial production methods.

In the proposals for the EU pharmaceutical legislation revision, which were published by the EC in April 2023, the biopharma industry were expecting to see:

  • Set standards of the implementation of the HE, specifically around the “non routine basis” definition 
  • Establishment of publicly accessible European registries that will record the implementation of the HE legislation and the collection of safety and clinical outcome data of patients treated with HE products, and encourage the publication or sharing of these data

Despite industry’s high expectations, HE is not expected to change conceptually. The HE legislation will retain its “ambiguous” terminology, given that no additional clarifications were made regarding its definition, while its implementation pre-requisites will remain the same. Some similarities of the updated version of the HE with the current legislation are that it will still be the national authorities’ responsibility to approve any hospital exemption requests and notify the EMA whenever a HE is granted. An important update is that member states shall ensure that data on the use, safety and efficacy of ATMPs prepared under HE is collected and reported by the HE approval holder to the national competent authority at least annually. This data should be transmitted to the EMA annually, and the Agency will be obliged to provide to the EC a report on the experience acquired with the hospital exemption approvals on the basis of contributions from member states and the annually reported data. The EC have also expressed their interest in understanding the implementation of the HE legislation and will engage with all the relevant stakeholders to conduct a study to collect data and get insights on its implementation at a national level. The main goal of this study will be to help the EC understand the therapeutic indications that these ATMPs are developed for, the number of patients that get access to these products, and whether their administration continues for the same indication.

The lack of clear definitions around the HE legislation, in combination with the increasing number of ATMPs that are being granted a HE, poses a significant threat to biopharma companies that develop ATMPs. The main threat that these companies are exposed to is significantly lower cost ATMPs obtaining access to EU markets, with ARI-0001 being a great example of a very low-cost CAR-T that received authorisation from the Spanish governmental drug agency, outside the centralised marketing authorisation pathway, and achieved decentralised uptake from hospitals in Barcelona at a price that is one-third of the commercial CAR-Ts already marketed in Spain. As a result, stakeholders should act immediately in order to develop a plan that will help them best position themselves in the ATMP HE landscape. However, the evolving HE environment could also act as an opportunity for the biopharma industry to establish new partnership models that will be sustainable for both companies and public organisations. ATMP manufacturers could also explore alternative strategies that might help them introduce more affordable treatments to the market.

P4A has been monitoring the EC pharmaceutical legislation revision and the HE changes, and will continue to do so to best advise our clients how to plan and adapt to the changing environment. Contact P4A to learn more about our services to support with determining ideal access pathways and to remain up to date on how to seize opportunities in an ever-changing space.



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