Day 1: Deep dive into the EMA qualification procedure with a focus on registries – closed workshop in person and online, targeting regulators, registry holders and industry; 

Day 2: Establish the value of patient registries for regulatory decision-making – broad representation of stakeholders in person and online. To enable wide participation, some parts of the workshop will be broadcast.
More information on how to register for online participation on Day 2 will be published shortly.

Should you have any questions, feel free to contact them at emaregistries@ema.europa.eu.

More information here

The pilot will focus on the needs of non-profit academic developers. They are a major contributor to the development of ATMPs and diagnostic and delivery devices, but experience has shown that navigating regulatory requirements can be challenging.

During the pilot, EMA will provide enhanced regulatory support for up to five selected ATMPs that address unmet clinical needs and are solely developed by academic and non-profit developers in Europe. EMA will guide the participants through the regulatory process with the aim to optimise the development of the ATMPs, starting from best practice principles for manufacturing to planning clinical development that meets regulatory standards.

The pilot’s first participant has already been selected. This ATMP is ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells, that is developed by the Hospital Clínic Barcelona. In December 2021, the product was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.

Importantly, no new regulatory tool will be introduced as part of this pilot. However, the aim is to assess what further support or regulatory tool may be provided to enhance the number of ATMPs reaching patients in the EEA. In the process, EMA is keen to learn how to better interact with and support academic developers.

The pilot participants will benefit from all the available regulatory flexibilities and development support measures, such as fee reductions and waivers. The progress will be closely monitored, and initial results of the pilot are expected to be available in 3-4 years. Upon completion, a report will be published and a workshop with relevant stakeholders may be organised to discuss the learnings.

Potential developer candidates can contact their national competent authority or EMA via advancedtherapies@ema.europa.eu to express their interest in participating in the pilot or to receive more information.

Tecvayli was supported through EMA’s PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs. EMA’s human medicines committee (CHMP) reviewed the application for marketing authorisation under an accelerated timetable to enable faster patient access to this medicine.

The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

For more details and related content, please click here.

• First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder: Xenpozyme (olipudase alfa), a therapy for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disease. Xenpozyme is indicated for use in children and adults of all ages with type A/B or type B ASMD. Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder.

• First treatment for children with Progeria or progeroid like syndromes (rare premature aging syndromes): Zokinvy (lonafarnib), a treatment for patients with a genetically confirmed diagnosis of Hutchinson-Gilford Progeria Syndrome or progeroid laminopathies. Zokinvy is indicated for use in children one year of age and older.Hutchinson-Gilford Progeria Syndrome is an ultra-rare multi-systemic ‘premature aging’ disease.

• First therapy to treat rare genetic nervous system disorder AADC deficiency: Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and paediatric patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis. AADC deficiency is an ultra-rare, inherited genetic disease which typically manifests within the first year of life.

• First gene therapy to treat severe haemophilia A: Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5). Patients with haemophilia A cannot produce factor VIII (an essential protein required for blood to clot and stop bleeding); they are more prone to bleeding and have prolonged bleeding, e.g. after injury or surgery. Haemophilia A is a rare debilitating disease affecting approximately 0.7 in 10,000 people in the EU.

This workshop seeks to engage stakeholders early in the drafting process of an EU Network data quality framework, to ensure a robust starting point, in line with their expectations and needs.

The main objectives of the workshop are to:

• Engage stakeholders in a discussion on the content (e.g.: topics, table of contents) and approach to a Data Quality Framework, according to their needs
• Collect input from experts in the field and learn from existing experiences
• Prioritise use cases to focus an in-depth analysis

The meeting will be broadcast live, and a video recording will be made available afterwards. No registration is required to view the live broadcast.

Watch the broadcast here: https://www.ema.europa.eu/en/events/data-quality-framework-medicines-regulation

To support not-for-profit organisations and academia ahead of the upcoming deadline for submission (February 28th), the EMA is hosting a “walk-in” clinic webinar on February 17th from 17.00 – 18.00 CET.

The webinar will:

• provide a brief reminder of the repurposing pilot
• provide information on how EMA real-world evidence initiatives can support the pilot
• have an open Q&A session to answer any questions

The deadline for webinar registration is February 14th.

More information about the pilot: https://www.ema.europa.eu/en/news/repurposing-authorised-medicines-pilot-support-not-profit-organisations-academia

Webinar registration: https://ec.europa.eu/eusurvey/runner/2aad8a2c-8707-64bd-1b4a-239f000dd854

CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union. The Clinical Trials Regulation and CTIS will strengthen clinical trials in the EU, ensuring better outcomes for patients and supporting the attractiveness of the European Union as a location for clinical research.

Sponsors can visit the public Clinical Trials website at https://euclinicaltrials.eu/home. The “CTIS for sponsors” page provides key information and links for sponsor users.

Patients, healthcare professionals and the general public can also access the public Clinical Trials website. When clinical trial applications have been submitted through CTIS and authorised by Member States,they will be openly accessible via the “Search clinical trials page”. The public searchable database will gradually contain more information as sponsors and Member States use it to initiate and oversee clinical trials.

The webinar will take place on January 18th 2022 from 10.00 – 12.00 CET.

Gaps exist in regulatory science that need to be addressed to improve medicine development and evaluation to enable access to innovative medicines that address patients’ needs. The EMA identified around 100 such topics and will publish them on December 15th 2021 as the “Regulatory science research needs” list.

Thus, this virtual event aims to launch the Regulatory science research needs initiative of the EMA. With this initiative, the EMA seeks to stimulate researchers and funding organisations to consider addressing these needs in their research work and funding programmes, respectively.

Researchers and stakeholders are invited to raise questions and to comment during the event; the agenda can be found here.

More information and registration here: https://ema-europa.webex.com/ema-europa/j.php?RGID=rb39c319843e8c9c7ff344ac294a2bfb1

As part of the pilot, EMA and the national medicines agencies will provide regulatory support, primarily scientific advice, to help these stakeholders generate a data package robust enough to support a future application by a pharmaceutical company.

The pilot is open to not-for-profit stakeholders and academia (institutions and individuals) who have a particular interest in repurposing in an area of public health interest, have a scientific rationale for their repurposing programme and would like to seek scientific advice from a regulatory authority.

The EMA is accepting submissions from sponsors until February 28th, 2022.

For more information: https://www.ema.europa.eu/en/news/repurposing-authorised-medicines-pilot-support-not-profit-organisations-academia

gene therapy SKYSONA

SKYSONA, developed by bluebird bio, is a one-time treatment which can only be given in a specialised hospital by doctors who are experienced in treating patients with CALD, transplanting bone marrow, and using gene therapy medicines. This therapy was accepted into PRIME, a support scheme developed by EMA for promising new medicines that address an unmet medical need.

Path to patient access: The EMA opinion will now be sent to the European Commission (EC) for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State in the context of the national healthcare system.

More information here: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-children-rare-inherited-neurological-disease