 | |  | | | | Welcome to the 1st edition of the CREATIC ATMP Newsletter!
The CREATIC
ATMP newsletter gives you a window to the rapidly evolving regulatory and
ethical landscape for therapies utilizing genes-, cells and tissue. You will
find a collection of news regulatory developments and discussions spiced up
with perspectives and analysis – all with a focus on legal and ethical issues.
The newsletter will be published quarterly, with the next issue
being scheduled for January. If you're interested in staying updated on the latest legal and ethical news related to ATMPs, please sign up here.
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| | | |  | | | | 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 predictable reimbursement of ATMPs in Czechia
Upcoming events
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|  | CREATIC is
Horizon Europe Teaming for Excellence project, which focuses on undiagnosed
untreatable rare diseases, paediatric patients with high-risk tumours, and
adult cancer patients suitable for ATMP. The overall aim is to translate
research and innovation in cell and gene therapies into clinical practice for
the benefit of patients.
You can read
more about CREATIC here.
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Preparing for HTAR to enter
into force in January 2025 | | | | |
| In January 2022, the Health Technology Assessment Regulation (HTAR) entered into force and will be fully applicable as of January 2025. Its goal is to improve Health Technology Assessment quality and harmonize evaluations of new medicines across EU. This regulation replaces the prior EU project-based system with a permanent framework for joint work, encompassing joint scientific consultations, identification of emerging technologies, and clinical assessments.
Read more here
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Commission publishes new
guidance on validity of clinical studies | | | | |
| On 19 September 2024, the Commission adopted a new guidance under the HTA Regulation on assessing the validity of clinical studies, detailing strengths, weaknesses, and recommendations for different study designs. You can find the guideline here.
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| | | | | | | What is HTA?
HTA is a procedure for assessing the added value, effectiveness,
costs and broader impact of health care interventions including medicines,
medical devices and procedures.
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Preparing for stricter standards for substances of human origin
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| | | | On 17 July 2024, the new Regulation on standards of quality and
safety for substances of human origin (SoHO) intended for human application (EC) No 2024/1938 entered
into force. The regulation aims to harmonize safety and quality standards across
Europe, improve human health protection for both donors and recipients and
prevent the human body or its parts from being a source of financial
gain. The regulation is expected to have several impacts on companies
developing ATMP's. Read more here.
The SoHO Regulation will become applicable on 7 August 2027, 3 years after its publication and entry into force, with an extra year for certain provisions. The official final text can be found here.
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| | | |  | | | | EMA COMP has elected at new chair | |
| | | | Tim Leest has been elected as the new chairman of the EMA's Committee for Orphan
Medicinal Products (COMP). He started his three-year mandate on 16 September
2024.
Read more here.
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| | | |  | | | | It is not about the price… | |
| | | | The collaboration between the pharmaceutical industry, public authorities, and research needs to be rethought if new advanced therapies are to benefit patients.
You can
read the full article here (in Danish).
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| | | |  | | | | 10-year anniversary of CAR-T cell treatment in Europe
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| | | | On May 13th, 2014, the first chimeric antigen receptor (CAR) T cell treatment in Europe was administered to a lymphoma patient as part of a clinical trial at Uppsala University Hospital.
Read more here.
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CREATIC News | |
| | | | The diagnosis of a rare disease can sometimes take several years. One of the steps to shorten the long diagnostic process is the establishment of specialized centers where patients are treated by an interdisciplinary team with access to advanced diagnostic methods.
👩⚕️ One such center is the Outpatient Clinic for Pediatric Patients without Diagnosis at the University Hospital of Brno, which focuses on patients suspected of having a genetic disease but who have not yet been able to receive a diagnosis.
🤝 MUNI | CREATIC Center has established a mutual partnership with the outpatient clinic at the beginning of the year and is also involved in the registration of undiagnosed patients.
For more updates follow CREATIC on Linkedin! | | | | |
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| | | The EU-Commissions proposed Pharmaceutical Legislation | |
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| In 2023, the EU-Commission adopted a new proposal that revises and replaces the existing general pharmaceutical 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.
You can read more about the proposed regulation here. | |
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| | | |  | The identification of public contributions
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| Article 57 of the proposed Pharmaceutical
Legislation will require both applicants and marketing authorisation
holders to publicly disclose
any direct financial support for research and development (R&D) from public
authorities. The aim of article 57 is to significantly improve the transparency
of public funding for the development of medical innovations through mandatory
information on cash flows.
You can read more about how the framework facilitates the identification of public contributions to the development of innovations in healthcare here.
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| | | | | | | What is the hospital exemption?
Article 28(2) of the ATMP Regulation (EC) No 1394/2007 introduces the Hospital Exemption where Member States can permit the provision
of an advanced therapy, without marketing authorisation if the ATMP fulfills a list of requirements.
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| | | |  | Hospital Exemption for ATMPs – Expanded Scope | | | | |
| Parliament has introduced a few additional requirements for the hospital exemption, to the Commission proposal to reform the Pharmaceutical Legislation. Notably, Parliament has significantly expanded the scope of the hospital exemption for ATMPs. One example is the inclusion of "cross-border exchange" of ATMPs in cases of "absence of other solutions for the individual patient" - something that has not been possible until now.
The European Parliament has on 10 April
2024 adopted its position on the Commission proposal to reform the Pharmaceutical
Legislation. A key takeaway from the amendments is both the expansion
of the Hospital Exemptions scope and the additional requirements.
You can read more
about the key takeaways from Parliaments amendments here.
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From exceptions to norms: The need for transparent and
predictable reimbursement of ATMPs in Czechia
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| | | | PharmDr. Adéla Bártová, Health
Economics Specialist at Masaryk
University, Faculty of Medicine
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 outpatient 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 (§39da 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
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| | | Conference | 25-06-2025 to 27-06-2025 | |
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| | | Conference | 18-03-2025 to 19-03-2025 | |
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