The inclusion of the hospital exemption for advanced therapies in the new pharmaceutical regulation does not satisfy health organizations and industry amidst accessibility concerns.
ADVERTISEMENTA European Commission proposal to tighten conditions in which Advanced Therapy Medicinal Products (ATMPs) may benefit from a special exemption for use in hospitals has divided health stakeholders with concerns about the lack of EU-wide data and calling for guarantees on the specific use conditions.
The principle of hospital exemption (HE) was first introduced in the EU regulation for ATMPs – medicines for human use based on genes, tissues or cells, and are mainly used to treat rare diseases – in 2007 to allow some treatments to be used without marketing authorisation under certain specified 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 legislation 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.
In its subsequent position, the Parliament strengthened the focus on the evidence on quality, safety and expected efficacy of the ATMPs when presenting applications for hospital exemption.
François Houÿez, director of treatment information and access at the European organisation for rare diseases EURORDIS, told Euronews that hospital exemptions can play a crucial role in increasing access to therapies that are unlikely to succeed in the pharmaceutical market.
Houÿez explained that for the exemptions to be truly effective there is a need for more harmonized criteria in the authorization, the creation of registries to track all treated patients, with the possibility of merging registries or exchanging data between hospitals, and transparency regarding the costs of ATMPs.
Paolo Morgese, from the Alliance of Regenerative Medicine (ARM), also agreed on the need for a rigorous regulatory process.
From the Parliament’s text, he welcomed the increased oversight over clinical and safety data on hospital exemption products.
“We hope to see greater clarity concerning the specific circumstances in which the HE scheme should be used, in order to preserve the integrity and expertise of the EMA centralised approval system,” he added regarding the ongoing discussions in the EU Council.
ARM’s Morgese added that the way the system currently works, there is no quality data on the number of patients benefiting from these treatments at the EU level.
“The different interpretations and implementations of the scheme across the EU have led to a situation where the hospital exemption is used in a large series of patients in some Member States,» agreed the European Federation of Pharmaceutical Industries and Associations (EFPIA), in a statement sent to Euronews. As such the organization questions whether the non-routine basis principle is being respected.
The EFPIA statement noted that the exemption should be applied only when no authorised medicinal product is available and called for the necessary safeguards to guarantee this.
“This is crucial to ensure that patients are protected and that developers will continue to invest, conduct clinical trials, and seek marketing authorisation in the EU, bringing more transformative ATMPs to patients in Europe,” it added.
However, not everyone is on the same page regarding the conditions for using the hospital exemption.
Ten European organizations, including the European Blood Alliance, the Association of European Cancer Leagues and Health Action International, published a statement warning of the dangers of over-restricting use of the exemption.
“The duration requirements of a hospital exemption cannot lead to the interruption or unavailability of patient treatment, nor create an unnecessary administrative burden for healthcare professionals that takes away from patient care or slows decision-making,” read the text.
They also consider that hospital exemption should not be limited to cases where no medicine is approved at the EU level, as approval does not always translate to availability and access as ATMPs are often not available due to marketing strategies of rights holders, high prices, or negative reimbursement decisions.
