Understanding the New EU Clinical Trial Regulation (CTR)

Understanding the New EU Clinical Trial Regulation (CTR)

On the 31st January 2022, The Clinical Trials Regulation ((Regulation (EU) No 536/2014)) became effective and replaced the previous Directive (EC) 2001/20/EC and corresponding national legislation. The Clinical Trials Regulation (CTR) coordinates the review and regulatory process for drug clinical trials across the EU. Prior to the implementation of the new CTR, clinical trial sponsors were required to submit clinical trial applications separately to national competent authorities and ethics committees in each country to gain regulatory approval to run a clinical trial.


The new CTR enables sponsors to submit a single application via an online platform known as the Clinical Trials Information System (CTIS) for approval to run a clinical trial in up to 30 EEA countries. The goal is to optimise the clinical trial process and make it more efficient to conduct pan-European trials. The regulation seeks to provide a single, unified portal and database for both trial sponsors and regulatory agencies in each member state. For sponsors, the portal will be the main platform to submit applications and notifications allowing regulators to conduct their assessments and supervise the trial. During a clinical trial, users of CTIS can collaborate with national regulators while recording the results. In addition to this, the CTIS enables the monitoring of results and assessing safety-related data in this single integrated online platform.


The CTR and CTIS aims include the implementation of clinical trial regulations, to improve the efficiency and transparency of drug clinical trials and ensure the highest safety standards for trial participants. Under the new CTR, clinical trial sponsors can use the Clinical Trials Information System (CTIS) from 31 January 2022, but are not obliged to use it immediately, in line with a three-year transition period. National regulators in the EU Member States and EEA countries will use CTIS from 31st January 2022. The Clinical Trials website can be visited here and the CTIS can be visited here where you can find out more information.


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Cell and Gene Therapies: 3 Key Supply Considerations

Cell and Gene Therapies: 3 Key Supply Considerations

Personalised medicines involve an increased degree of complexity when it comes to manufacturing and supply in comparison to traditional pharmaceuticals. The Horizon 2020 Advisory Group defines personalised medicine as ‘a medical model using the characterisation of individuals’ phenotypes and genotypes (e.g. molecular profiling, medical imaging, lifestyle data) for tailoring the right therapeutic strategy for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely and targeted prevention.’ Personalised medicine focuses on treating patients based on their individual clinical characteristics, the diversity and severity of symptoms, and genetic traits. Cell and gene therapies (CGTs) are an increasingly important segment of personalised treatments. CGTs are typically expensive treatments manufactured in low volume. Due to the time-sensitive nature and challenging temperature conditions associated with manufacturing, handling and treating patients with cell and gene therapies, this article outlines some considerations for the handling and supply of CGT products.


Consideration 1: Transportation

Many CGTs are transported and stored at ultra-low temperatures including cryogenic temperatures below -80 °C. Primary materials and therapies may be required to be shipped from the site of manufacturing to the clinical setting within 40 to 50 hours typically. Maintaining the integrity of cell and gene therapy products during shipping, as well as ensuring that they arrive on time and within the appropriate temperature constraints, is a major challenge. CGT companies should consider:

  • Has the optimal courier been selected who has experience with transporting high-value, temperature-sensitive cell and gene therapies?
  • Is the selected shipping system suitable and validated for transporting the materials?
  • Has a risk assessment been performed to mitigate any delays or incidents that may arise during transportation?
  • Is the necessary documentation available to successfully export and import the CGTs across territories?
  • What processes are in place at the receiving site to ensure the CGTs arrive undamaged, viable and fit for administration?

Issues experienced during transportation can be possibly devastating for patients and highly costly for clinical trial sponsors as CGTs can cost $200,000–2,000,000 per dose. A commercially viable supply chain is recommended to be in place at the start of clinical trials (even in Phase 1) as many of these therapies are given orphan (US) and prime (EU) status which could allow them to gain approval in a shortened timeframe should the clinical trials be successful.


Consideration 2: Sourcing of Materials

Materials and equipment used in the manufacturing and supply of cell and gene therapies may be more challenging to acquire in contrast to traditional pharmaceutical products. For example, high quality specialised reagents are critical to maintaining healthy cell cultures that perform optimally and consistently. In many cases, companies may only have single-source suppliers and this may increase the risk of supply chain disruption. With CGTs requiring specialised raw materials and unique manufacturing equipment, CGT companies commonly chose to outsource manufacturing to CDMOs with experience in small volume, high value manufacturing and the ability to manage specialised packaging and labelling.

CGT companies may take into consideration:

  • Has an inventory management system been developed to ensure there are optimal levels of materials available?
  • Are there alternative suppliers of materials and equipment that may be qualified as a back-up source?
  • Have your key suppliers been qualified for use and audited?
  • Have Critical Quality Attributes (CQA) and Critical Process Parameters(CPP) been defined and agreed with suppliers?
  • Has a Business Continuity Plan (BCP) been developed and implemented?

Selecting and qualifying the optimal vendors to support your clinical and commercial needs is a crucial activity. Identifying risks to the provision of materials and implementing mitigation strategies could prevent detrimental impacts to product supply and ultimately to patients. Furthermore, effective communication with suppliers to coordinate and schedule activities is essential to avoid disruption.


Consideration 3: End-to-end Visibility

End‑to‑end traceability from manufacturing to final delivery of CGTs to patient treatment centers is an important consideration for successfully providing personalising treatments. A CGT supply chain may involve many different entities including contract development and manufacturing organisations (CDMOs), thermal shipping system suppliers, diagnostic companies, specialised couriers and distributors. Ensuring there is alignment between all entities should be a key area reviewed during the pre-clinical and clinical development phases.

A Chain of Identity (COI) linked to the unique identifier should be generated at patient enrolment. This identifier should be visible and traceable throughout a patient’s personalised treatment. For example, as part of the COI, an autologous donor’s patient number should be associated with their unique donation number (e.g. Donor identification number/DIN) and the manufacturing batch number. Chain of custody (COC) refers to the ownership/stakeholder ‘in‑custody’ of the cells or drug product, at any stage of the value chain. COC typically incorporates numerous critical parameters to ensure high quality standards and integrity of the cells at every step. COI and COC are key components to ensure traceability over the course of the personalised treatment and to safeguard patient safety. This can present more challenges that the traditional biopharmaceutical product supply model as adequate infrastructure and systems must ensure the correct CGT is available in time for the scheduled administration at the correct treatment site for the correct patient. Autologous cell therapies, for instance, must have robust controls in place to ensure patients receive the CGT from their own donor cells and not another patient’s cells. Important areas to closely examine in relation to end-to-end visibility include:

  • Have the COI and COC requirements been reviewed and defined?
  • Is there full traceability from the start to the finish of the CGT product lifecycle?
  • If using a CDMO, how will manufacturing activities be monitored and how will key updates be provided?
  • What processes are in place to ensure the correct CGT reaches the correct patient?


Advanced technologies including software systems can support the coordination of last mile deliveries with treatment centers. As the CGT industry continues to grow rapidly, so too will the development of technologies to provide end-to-end traceability.


To learn more about HiTech Health CGT services –  contact us.


EMA Regulation of Cell and Gene Therapies (CGTs)

EMA Regulation of Cell and Gene Therapies (CGTs)

Cell and Gene Therapy products and other Advanced Therapy Medicinal Products (ATMPs) have to comply with European Medicines Agency (EMA) legislation at different stages of the development process, including Good clinical practice (GCP), Good manufacturing practice (GMP) and Good Laboratory Practice (GLP) requirements. Potential new Cell and Gene Therapies or Advanced Therapy Medicinal Products firstly require laboratory tests and clinical trials to evaluate their benefits and side effects. Understanding the EU legal and regulatory framework, providing details of the benefits and risks of the products and obtaining approval from regulatory authorities for their use is therefore essential to providing the best possible treatments for end users. So this article will cover the basic EMA regulation of Cell and Gene Therapies (CGTs).

Research and development of Cell and Gene Therapies:

Tissue procurement, CMC and GMP manufacturing:

Marketing authorisation and Post-authorisation (Pharmacovigilance for ATMPs):


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Cell and Gene at the Abbey 2021 – Galway, Ireland

Cell and Gene at the Abbey 2021 – Galway, Ireland

From 10th to 12th November, HiTech Health hosted ‘Cell and Gene at the Abbey’, a world-leading conference focusing on Production, Scale-up and Globalisation of cell and gene therapies. We would like to sincerely thank all the speakers, sponsors and attendees of this year’s conference and are already looking forward to hosting the event in 2022.

Over the course of 3 days at the beautiful setting of the Glenlo Abbey in Galway, there were amazing insights from global leaders in the cell and gene therapy space. Overcoming production and supply chain challenges was a key focus with speakers from around the world attending the event. The exciting list of speakers included Power List 2020 in Advanced Medicines featured experts Bruce Levine, Massimo Dominici and Qasim Rafiq who spoke about development and regulatory approvals, challenges in scaling up, supply chain and manufacturing and the opportunities to solve them. An update on the European regulatory environment for Advanced Medicines from Niamh Curran of the HPRA was of benefit to all.

Attendees were able to see first-hand some of the latest technologies in cell processing technologies from many of  the event’s sponsors Cellexus, ChemoMetec, Miltenyi Biotec, Sexton Biotechnologies, Terumo, VivaBioCel, AccuScience and MeiraGTx. Cell and Gene therapies are at the forefront of regenerative medicine and HiTech Health are continuing to grow rapidly in this sector. Find out how we can support you on your development and commercialisation journey by contacting us today.

HiTech-Health awarded with government funding

HiTech-Health awarded with government funding

HiTech Health awarded with €7m government funding for ‘disruptive’ project


HiTech Health and our partners, Relevium Medical and Professor Garry Duffy’s Laboratory at NUI Galway are honoured to receive €7 million from the Disruptive Technologies Innovation Fund (DTIF), administered by Enterprise Ireland. This award will support the development of a novel treatment for Knee Osteoarthritis with the potential to regenerate the damaged knee joint whilst providing long-lasting pain relief.

Dr Brian Harrison, MD Hi-Tech Health, “We are excited to work with Relevium and Prof Garry Duffy’s team on the process development, scale up and GMP manufacturing of this novel therapeutic candidate at our Galway facilities. With all parties in the local area, we believe this will strengthen our partnership and help with developing this novel Knee Osteoarthritis therapy.”

Dr Alison Liddy, Founder and CEO Relevium, “This funding will enable Relevium and our partners to develop this ground-breaking treatment that has the potential to transform the lives of people living with Knee Osteoarthritis.”

Professor Garry Duffy, NUI Galway, “My team at NUI Galway are delighted to be part of this project and to work with industrial leaders to develop an impactful solution for Osteoarthritis.”

Relevium Medical awarded with government funding

HiTech Health awarded with government funding

NUI Galway awarded with government funding

Read the full Irish Times article here: Government to provide €95m in funding for ‘disruptive’ projects

Find out more about Relevium: www.relevium-medical.com

Find out more about NUI Galway: www.nuigalway.ie/medicine-nursing-and-health-sciences/

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HiTech-Health awarded with government funding

HiTech Health awarded with €7m government funding for 'disruptive' project   HiTech Health and our partners, Relevium Medical and Professor Garry Duffy’s Laboratory at NUI Galway are honoured to receive €7 million from the Disruptive Technologies Innovation Fund...

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HiTech Health and Anthony Nolan announce collaboration

HiTech Health and Anthony Nolan announce collaboration

HiTech Health and blood cancer charity Anthony Nolan have entered into an agreement to facilitate the development of novel cell and gene therapies.

Anthony Nolan Cell & Gene Therapy Services will support HiTech Health’s supply chain through the provision of a reliable, scalable source of high-quality starting material. Initially provision of cellular material will be for research and development (R&D) programmes with a view to scale up in the future to clinical application and manufacturing. The parties are open to collaboration with cell and gene therapy developers and will work with them to provide bespoke offerings specific to the client’s requirements.

Anthony Nolan Cell & Gene Therapy Services brings expertise in cell sourcing, selection and provision of high-quality starting material to complement HiTech Health’s end-to-end service from product development to manufacturing and launch of life-changing treatments.

In partnering we aim to support cell and gene therapy researchers and developers to bring life-saving treatments to patients who need them.

Diana Hernandez, Head of Immunotherapy Research at Anthony Nolan says: “This is a significant step towards establishing a sustainable chain in cell and gene therapy development as we move towards allogeneic therapies. This collaboration ensures supply of ethically sourced starting material to developers of new therapies, which we hope will improve the treatment and care of thousands of patients in the future. It also allows the parties to share information regarding quality attributes of the material, which can help inform future improvements.”

Aoife Duffy, Cell and Gene Therapy Operations Manager, HiTech Health says: “HiTech Health’s collaboration with Anthony Nolan will be a great benefit to our clients. Anthony Nolan’s reputation for supply of high quality starting materials provides assurance to our clients and collaborators that a robust supply chain is in place for the manufacturing and supply of Cell and Gene Therapy Products. With this collaboration with Anthony Nolan, we now have a supply of starting material that is crucial to enable manufacturing of these novel therapies. This collaboration adds to our current R&D experience and future expansion into GMP manufacturing. Our expertise in quality, QP approval, and management of product supply and logistics will also be an important role in this collaboration. We are very excited about this collaboration and are looking forward to the possibilities that we can achieve together with the ultimate goal of treating patients who have an unmet medical need.”

About Anthony Nolan Cell and Gene Therapy Services

Anthony Nolan is the pioneering charity that saves the lives of people with blood cancer and blood disorders who need a stem cell transplant. Anthony Nolan’s Cell and Gene Therapy Services aim to facilitate ethical research and development through the sourcing and supply of donor starting materials to researchers and developers. With a register of over 850,000 donors, a cord blood bank, and four decades of experience providing stem cells, Anthony Nolan has the infrastructure, the expertise, and the skills to support the needs of the cell and gene therapy industry

Website: www.anthonynolan.org/clinicians-and-researchers/cell-and-gene-therapy-services
LinkedIn: www.linkedin.com/showcase/anthony-nolan-cell-&-gene-therapy-services/
Contact: Katie Griffee, Business Development Manager – katie.griffee@anthonynolan.org

About HiTech Health

HiTech Health bring an in-depth knowledge and experience of Cell and Gene Therapy products, We offer a range of services to help develop and supply life-changing treatments. Our experts can provide comprehensive solutions and strategies for process development, supply pre-clinical products and quality control assay development. We also have the GMP experience and expertise including QP support to ensure GMP compliance and product release. We also have the demonstrated supply chain and logistics expertise to enable cell and gene therapy products to be imported into Europe and the UK.

Find out more about Cell & Gene Therapy: www.hitech-health.com/cell-and-gene-therapy/
Find us on LinkedIn: www.linkedin.com/company/hitech-health
Contact: Aoife Duffy, Cell & Gene Therapy Operations Manager – aduffy@hitech-health.com

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