BASG and EU authorities warn against illegal gene, tissue or cell therapies New
Non-compliant advanced therapy medicinal products can pose serious risks to patients.
Advanced therapy medicinal products (i.e. somatic cell therapeutics, products made from bioengineered tissue preparations or gene therapeutics, so-called ATMPs (Advanced Therapy Medicinal Products)) can be a promising therapy for many patients and are valuable in cases where other medicines have limited or no effect. However, like any other medicinal product, they must be tested for safety and efficacy and are subject to strict regulatory control.
However, patients are sometimes treated at individual clinics or centres that offer medicines that also fall under the classification of advanced therapies, but which have neither been systematically tested nor approved by the authorities and can therefore pose serious risks to patients. These therapies therefore have no evidence of efficacy or safety and the conformity of their manufacturing and quality control procedures with applicable EU regulations has not been verified. In addition, other violations that may occur in connection with these therapies, such as violations of patients' rights or health regulations, are not uncommon.
In particular, these non-compliant, non-authorised medicinal products could be offered to exploit the need for protection, vulnerability or good faith of desperate patients with serious and/or chronic diseases, e.g. certain cancers or diseases with limited treatment options.
The competent national authorities therefore take action when unauthorised advanced therapy medicinal products are offered outside the strict criteria of the applicable pharmaceutical regulations or related activities are deemed to be breaches of regulatory requirements or even criminal offences. These measures may include direct regulatory sanctions against the products or facilities/companies and may also include action by enforcement authorities and prosecutors where necessary.
The EU-wide Working Group of Enforcement Officers (WGEO), as part of the association of European national marketing authorisation authorities for human and veterinary medicinal products (Heads of Medicines Agencies (HMA)), brings together experts from regulatory authorities, law enforcement agencies and customs to combat the problem of illegal and falsified medicinal products and the illegal trade in medicinal products.
To this end, the group maintains an operational network of official contact points in each EU member state. This enables the rapid coordination and dissemination of relevant information and warnings in cases that potentially affect more than one Member State, as part of the investigation and implementation of measures.
In addition, it is particularly important that patients are also aware of the dangers of unauthorised therapies and can recognise the risks associated with non-compliant, untested therapies themselves.
Indications of such questionable therapies can be, for example
- Therapies that are advertised as alternative or experimental but have not been tested in a clinical trial (and therefore are not listed in the public registry of clinical trials. https://euclinicaltrials.eu/).
- Therapies that are advertised as the supposed last hope for diseases by appealing conspicuously intensively to the urgent needs of patients while contradicting current medical knowledge about the respective diseases.
- Therapies that claim unprecedented and superior efficacy that has not been proven in the medical literature or is simply too good to be true.
- Products that claim to complement or even improve on conventional, authorised therapies.
The HMA, the BASG (Federal Office for Safety in Health Care) and all national European competent authorities ask the public to co-operate regarding the potential dangers and risks of non-compliant ATMP therapies and to report all suspicious cases in Austria to the local contact point of the BASG (Enforcement/Illegalities, enforcement@basg.gv.at) in order to protect themselves and public health.
ATMPs/advanced therapy medicinal products can only be used legally in the following three cases to ensure that their quality, safety and efficacy are guaranteed:
- As authorised medicinal products. They are subject to a centralised authorisation procedure at the EMA and are valid and authorised throughout the EU. All information about the authorisation is available on the EMA website and from the relevant national competent authorities.
- As part of an authorised clinical trial. In officially authorised clinical trials, patients' rights are protected and patients participating in clinical trials never have to pay for their treatment. Products participating in clinical trials can also be found in EU databases. https://euclinicaltrials.eu/
- Under a hospital exemption granted by the competent national authorities, as laid down in the European ATMP Regulation (Regulation (EC) No 1394/2007). This procedure allows the manufacture of advanced therapy medicinal products in strictly regulated exceptional cases outside the standardised centralised authorisation procedure and their use in a hospital under the professional responsibility of a physician.
Conclusion:
If there are cases of use of advanced therapy medicinal products that do not meet the three criteria mentioned above and which therefore appear suspicious, the HMA and the BASG recommend reporting all such cases suspected in Austria to the local contact point of the BASG (Enforcement/Illegalities, enforcement@basg.gv.at) in order to avoid risks to themselves and to protect public health.
Links: https://www.basg.gv.at/marktbeobachtung/amtliche-nachrichten/detail/illegale-stammzelltherapien
Reporting suspected cases:
E-mail: enforcement@basg.gv.at
Queries (technical):
Dr Christoph Baumgärtel, Phone: +43 505 55-36004
E-Mail: christoph.baumgaertel@ages.at
Queries (for media):
Communications Management, Tel.: +43 505 55-25000
E-mail: presse-basg@basg.gv.at
