ATMP Issue #1

Welcome to the 1st edition of the CREATIC ATMP Newsletter!
The CREATIC ATMP newsletter gives you a window to the rapidly evolvingregulatory and ethical landscape for therapies utilizing genes-, cells and tissue. Youwill find a collection of news regulatory developments and discussions spiced upwith perspectives and analysis – all with a focus on legal and ethical issues.
The newsletter will be published quarterly, with the next issue being scheduled forJanuary. If you're interested in staying updated on the latest legal and ethical newsrelated to ATMPs, please sign up here.

News
- About CREATIC
- Preparing for HTAR to enter into force in January 2025
- Commission publishes new guidance on validity of clinical studies
- Preparing for stricter standards for substances of human origin
- EMA COMP has elected at new chair
- It is not about the price…
- 10-year anniversary of CAR-T cell treatment in Europe
- CREATIC news
- The EU-Commissions proposed Pharmaceutical Legislation

Monthly opinion
From exceptions to norms: The need for transparent and predictablereimbursement of ATMPs in Czechia

Upcoming events

CREATIC is Horizon Europe Teaming for Excellence project, which focuses onundiagnosed untreatable rare diseases, paediatric patients with high-risk tumours,and adult cancer patients suitable for ATMP. The overall aim is to translate researchand innovation in cell and gene therapies into clinical practice for the benefit ofpatients.

On 17 July 2024, the new Regulation on standards of quality and safety forsubstances of human origin (SoHO) intended for human application (EC) No2024/1938 entered into force. The regulation aims to harmonize safety and qualitystandards across Europe, improve human health protection for both donors andrecipients and prevent the human body or its parts from being a source of financialgain. The regulation is expected to have several impacts on companies developingATMP's.

The SoHO Regulation will become applicable on 7 August 2027, 3 years after itspublication and entry into force, with an extra year for certain provisions.

The diagnosis of a rare disease can sometimes take several years. One of the stepsto shorten the long diagnostic process is the establishment of specialized centerswhere patients are treated by an interdisciplinary team with access to advanceddiagnostic methods.
👩‍⚕️ One such center is the Outpatient Clinic for Pediatric Patients without Diagnosisat the University Hospital of Brno, which focuses on patients suspected of having agenetic disease but who have not yet been able to receive a diagnosis.
🤝 MUNI | CREATIC Center has established a mutual partnership with theoutpatient clinic at the beginning of the year and is also involved in the registrationof undiagnosed patients.
For more updates follow CREATIC on Linkedin!

In 2023, the EU-Commission adopted a new proposal that revises and replaces the existing generalpharmaceutical legislation, as well as the legislation on medicines for children and for rare diseases.The proposed regulation is expected to have several impacts on the development of ATMP's.

From exceptions to norms: The need fortransparent and predictable reimbursement ofATMPs in Czechia

ATMPs and orphan drugs are critical treatment options for rare diseases, particularly for pediatric patients. However, the current system in the Czech Republic for financing and reimbursing these medicines is often unsustainable and lacks adequate transparency. Legislative reforms are needed to ensure fair access and equitable treatment for rare disease patients in out patient care, in contrast to the well-established system for standard medicinal products approved for larger patient populations. Although a budget impact analysis (BIA) is required to secure reimbursement, certain orphan drugs are exempt from a cost-effectiveness analysis (CEA); however, this exemption is not applied uniformly (§39 da Coll. 48). As a result, ATMPs are often excluded from standard reimbursement pathways and instead rely on alternative funding, such as exceptional reimbursement pathways or support from external sources, including foundations and public donations.
While Czech legislation allows for the reimbursement of ATMPs and orphan drugs from public funds, there are still significant gaps and ambiguities in how these treatments are financed for healthcare providers. Exceptional reimbursement has become a temporary means of accessing many treatments, especially for pediatric patients. However, the frequent use of these exceptions suggests that they are increasingly serving as a standard solution, which differs from the original intent of health insurance legislation. This reliance has led to rising,unmonitored spending outside the standard reimbursement framework, reducing the incentive for manufacturers to advocate for much-needed legislative reforms that would bring consistency and transparency to reimbursement decisions

Our newsletter shares the latest developments, changes, and discussions in the world of ATMP's and keep youupdated on our upcoming events and initiatives with a special focus on Social Science and Humanities (SSH)including legal and regulatory issues.
For further information about CREATIC, contact Jakob Wested: jakob.wested@jur.ku.dk
For further information about the newsletter, publication of events, opinion and commentaries, contactmanaging editor Emma Schou Nielsen: vzk859@jur.ku.dk.
Link to CREATIC homepage. Created in cooperation with Masaryk University.

This is an archived issue, therefore we cannot guarantee that the links will work.