Company News
2024 was a year of growth for Tower Mains, with our team expanding to 14 and the addition of a new Associate strengthening our Investigator Site Auditing services. We also enhanced our training offerings and deepened our engagement with industry and academia to support quality understanding at all levels.
We look forward to continuing this momentum in 2025 and supporting you in the year ahead!
Regulatory News
New MHRA Chair Anthony Harnden sets key priorities for his tenure
On 2nd January, Professor Anthony Harnden officially began his role as Chair of the MHRA. In his first 100 days, he plans to engage with staff and partner organisations to understand how to advance the MHRA’s strategic direction, aiming to maintain the UK’s status as a global leader in life sciences for the benefit of patients and the public.
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New EU rules for health technology assessments take effect to accelerate patient access
On 12th January, the EU’s new Health Technology Assessment Regulation (HTAR) became effective, aiming to accelerate and broaden patient access to innovative health technologies. This regulation establishes a unified framework for assessing the clinical effectiveness and safety of new medicines and high-risk medical devices across EU member states.
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EMA recommends 114 human medicines in 2024, boosting access to oncology and biosimilars
On 16th January, the European Medicines Agency (EMA) revealed that in 2024 it recommended 114 new medicines for marketing authorisation, with 46 featuring new active substances previously unauthorised in the European Union. The oncology sector led with 28 recommendations, and 28 new biosimilars were approved, enhancing accessibility to treatments for various conditions.
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FDA unveils IT operating plan to enhance public health through technology and innovation
On 17th January, the FDA unveiled its IT Operating Plan for Fiscal Years 2024 – 2027, outlining a strategic approach to advancing public health through technology and data. The plan emphasises global collaboration, the use of emerging technologies like AI, data sharing, and robust cybersecurity practices. Developed with input from workshops, listening sessions, and a Federal Register Notice, it focuses on modernising IT infrastructure, optimising investments, and creating a unified “OneFDA” digital ecosystem to enhance efficiency and innovation in public health.
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EMA recommends 25 veterinary medicines in 2024, including treatments for companion animals
On 23rd January, the EMA revealed that in 2024 it recommended 25 veterinary medicines for marketing authorisation, the highest number in a single year. Among these, two featured new active substances previously unauthorised in the EU. Fourteen were vaccines, including seven developed through biotechnological processes. Thirteen of the recommended medicines were intended for food-producing animals such as chickens, pigs, and cattle, while eleven were for companion animals like dogs and cats.
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European Commission releases 2023 annual report on medicines for children
On 24th January, the European Commission released the “Medicines for Children 2023 Annual Report,” highlighting progress in paediatric medicine development. The report underscores the importance of ensuring that children have access to safe and effective medicines tailored to their specific needs. It details initiatives aimed at enhancing the availability of paediatric medicines, including the implementation of the Paediatric Regulation, which encourages the development of medicines for children.
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European Shortages Monitoring Platform fully operational to track medicine supply issues
On 29th January, the EMA announced that the European Shortages Monitoring Platform (ESMP) is now fully operational. The platform enables the monitoring and reporting of medicine shortages across the EU, enhancing coordination to prevent and manage supply issues. From 2nd February, it will be mandatory for marketing authorisation holders and national authorities to report shortages through the platform, with publicly available information accessible via the ESMP.
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UK launches integrated pathway to speed access to transformative medicines for patients
On 30th January, the MHRA, in collaboration with UK Health Technology Assessment bodies and the NHS, unveiled an enhanced Innovative Licensing and Access Pathway (ILAP). This integrated pathway aims to expedite the delivery of transformative medicines to NHS patients by fostering early collaboration among developers, regulators, and healthcare providers.
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EU Clinical Trials Regulation now fully applicable, streamlining processes across member states
On 31st January, the European Medicines Agency (EMA) announced that the Clinical Trials Regulation (CTR) is now fully applicable across the European Union (EU). This milestone concludes a three-year transition period during which over 5,000 clinical trials were integrated into the Clinical Trials Information System (CTIS), the unified platform for submitting and assessing clinical trial applications in the EU. As of this date, all clinical trials, including those approved under the earlier Clinical Trials Directive, are governed by the CTR.
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MHRA seeking Feedback on draft guidelines for individualised mRNA cancer immunotherapies
On 3rd February, the MHRA launched a consultation on a draft guideline on individualised mRNA cancer immunotherapies, aiming to streamline the approval process for these innovative treatments. This guidance addresses regulatory classification, product design and manufacture, evidence of safety and effectiveness, post-approval safety monitoring, and information for patients and the public. The consultation period is open until 31st March 2025 and invites feedback from manufacturers, developers, patient organisations, and other stakeholders to ensure robust safety standards while facilitating patient access to these novel therapies.
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Published Guidance
Medicines and Healthcare products Regulatory Agency (MHRA)
- 15th January: Medical devices: post-market surveillance requirements. How to interpret post-market surveillance (PMS) requirements for medical devices in Great Britain.
- 15th January: Medical devices: periodic safety update report (PSUR). Information and recommendations on the presentation and review of a periodic safety update report (PSUR).
- 15th January: Breast implants: reporting adverse incidents. Guidance for manufacturers on reporting adverse incidents involving breast implants under the vigilance system was issued.
- 30th January: Innovative Licensing and Access Pathway (ILAP). Guidance on the Innovative Licensing and Access Pathway (ILAP), an initiative by the MHRA designed to expedite the availability of transformative medicines and drug-device combinations to patients.
- 31st January: UK-wide licensing for human medicines: supplementary guidance. Information on the licensing of medicines for human use in the UK following the agreement of the Windsor Framework.
- 3rd February: Current approaches to product marking. Guidance on product marking, including the continued recognition of certain EU requirements, such as CE marking, for specific product sectors.
- 3rd February: Digital mental health technology: qualification and classification. To help manufacturers define the characteristics of their devices and determine the appropriate regulatory qualification and classification.
US Food and Drug Administration (FDA)
- 6th January: Recommendations to Reduce the Risk of Transmission of Human Immunodeficiency Virus (HIV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps). Draft guidance on donor eligibility recommendations to reduce the risk of transmission of Hepatitis B Virus, Hepatitis C Virus, and Human Immunodeficiency Virus by HCT/Ps.
- 6th January: Considerations for Complying with 21 CFR 211.110. Guidance, which when finalised, will describe considerations for complying with the requirements in 21 CFR 211.110 to ensure batch uniformity and drug product integrity. In addition, this guidance discusses related quality considerations for drug products that are manufactured using advanced manufacturing.
- 7th January: Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products. Recommendations to sponsors and other interested parties on the use of artificial intelligence (AI) to produce information or data intended to support regulatory decision-making about safety, effectiveness, or quality for drugs.
- 7th January: Artificial Intelligence-Enabled Device Software Functions: Lifecycle Management and Marketing Submission Recommendations. Recommendations about the contents of marketing submissions for devices that include artificial intelligence (AI)-)-enabled device software functions including documentation and information that will support FDA’s evaluation of safety and effectiveness.
- 7th January: Developing Drugs for Optical Imaging. Recommendations to sponsors regarding clinical trial design features that support the development and approval of optical imaging drugs that are used in conjunction with imaging devices and intended as intraoperative aids for the detection of pathology such as tumours or to enhance the conspicuity of normal anatomical structures.
- 7th January: Considerations for Including Tissue Biopsies in Clinical Trials. Recommendations to industry, investigators, institutions, and institutional review boards (IRBs) regarding considerations for tissue biopsies that may be conducted in adults and in children as part of clinical trials that evaluate investigational medical products and/or that are conducted or supported by the Department of Health and Human Services (HHS).
- 7th January: Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway. Guidance, which when finalised, will describe the FDA’s interpretation of the term “underway” and policies for implementing this requirement, including factors the FDA intends to consider when deciding whether a confirmatory trial is underway before an accelerated approval action.
- 17th January: Prevention and Treatment of Chemotherapy-Induced Peripheral Neuropathy: Developing Drug and Biological Products in Oncology. Recommendations for sponsors for the clinical development of drug and biological products intended for the prevention and treatment of chemotherapy-induced peripheral neuropathy in oncology patient populations.
Updated Guidance
MHRA
- 20th January: Blood establishment authorisation application form. The MHRA updated the form to apply for blood establishment authorisation and guidance on completing the form.
- 24th January: MHRA phase I accreditation scheme. A list of Accredited Units 24 October 2023 was added to guidance on how to join the phase I accreditation scheme.
- 29th January: Supply unlicensed medicinal products (specials). A new guidance document, complementary to MHRA Guidance note 14, was included linking different government pages of interest to the supply of unlicensed medicinal products in the UK.
FDA
- 7th January: Interim Policy on Compounding Using Bulk Drug Substances Under Section 503B of the Federal Food, Drug, and Cosmetic Act. Describes the FDA’s interim regulatory policy concerning compounding by outsourcing facilities using bulk drug substances while the FDA develops the list of bulk drug substances that outsourcing facilities can use in compounding under the applicable section of the Federal Food, Drug, and Cosmetic Act (FD&C Act).
- 7th January: Interim Policy on Compounding Using Bulk Drug Substances Under Section 503A of the Federal Food, Drug, and Cosmetic Act. Describes the FDA’s interim policy concerning compounding by human drug product compounders that are not outsourcing facilities using bulk drug substances while the FDA develops the list of bulk drug substances that can be used in compounding under the applicable section of the Federal Food, Drug, and Cosmetic Act (FD&C Act).
- 13th January: Premarket Approval Application and Humanitarian Device Exemption Modular Review. Information on the Premarket Approval Application (PMA) and Humanitarian Device Exemption (HDE) modular review program and the procedures for submitting or reviewing a modular PMA or HDE.
- 31st January: Recommendations to Reduce the Risk of Transmission of Mycobacterium tuberculosis (Mtb) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps). To assist establishments making donor eligibility (DE) determinations in understanding the requirements in 21 CFR part 1271, subpart C (21 CFR part 1271, subpart C).
- 31st January: Recommendations to Reduce the Risk of Transmission of Disease Agents Associated with Sepsis by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps). To aid establishments making donor eligibility determinations in understanding the requirements in Title 21 Code of Federal Regulations, part 1271, subpart C (21 CFR part 1271, subpart C).
Industry News
Neumirna Therapeutics secures €20 million in Series A financing to advance RNA therapies for epilepsy and other neurological disorders
On 9th January, Neumirna Therapeutics announced the first closing of its €20 million Series A funding round. The investment, co-led by Angelini Ventures and Invivo Partners, will support the development of NMT.001, a promising RNA-based therapy for drug-resistant epilepsy. The funding will help expand R&D and progress the therapy through key development stages.
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Biofidelity and CellCarta partner to expand lung cancer testing in global trials
On 9th January, Biofidelity and CellCarta announced a partnership to deploy Biofidelity’s Aspyre® Lung assay in global clinical trials. The assay enables efficient detection of lung cancer biomarkers using DNA and RNA from tissue and blood. CellCarta will validate and implement Aspyre Lung in its European and U.S. labs, supporting clinical trials with advanced genomic profiling for liquid and tissue biopsy samples. This collaboration aims to streamline genomic testing and accelerate clinical trial enrolment.
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FDA spotlights CDER’s new method for streamlining nitrosamine safety assessments
On 10th January, the FDA released a Spotlight on CDER Science, highlighting CDER’s collaboration with international regulators to create a methodology called the Carcinogenic Potency Categorisation Approach (CPCA). This approach evaluates the chemical structure of nitrosamine impurities to recommend acceptable intake (AI) limits by categorising them into one of five predicted potency levels, based on carcinogenic risk. The use of the CPCA has streamlined the regulatory review process for drug safety assessments, enabling more efficient identification of AI limits with improved transparency and predictability.
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GlobalData report highlights AI’s potential to transform pharmaceutical R&D and reduce costs
On 13th January, GlobalData issued a report highlighting the significant impact of artificial intelligence (AI) on pharmaceutical R&D. The report indicates that AI is set to improve productivity, reduce costs, and streamline drug discovery and clinical trials. AI has already proven effective in lead generation and optimisation, particularly in early-stage drug development. The report emphasises the importance of AI adoption for pharmaceutical companies to stay competitive and keep up with digital trends.
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Datapharm partners with phactMI to enhance US access to medicines information
On 14th January, Datapharm announced a partnership with phactMI to enhance the accessibility of medicines information in the US. This collaboration aims to integrate Datapharm’s advanced search technology into phactMI’s Drug Information Database, providing healthcare professionals and patients across the U.S. with comprehensive and up-to-date information on medicines. This partnership marks a significant milestone in Datapharm’s mission to support healthcare professionals in delivering informed and effective patient care globally.
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FDA highlights medical device supply chain vulnerabilities
On 16th January, the FDA highlighted the impact of medical device supply chain vulnerabilities on vulnerable patient populations, particularly children. Shortages of appropriately sized devices, such as those for paediatric use, have led to safety risks and suboptimal care. The FDA pointed out several contributing factors, including manufacturing limitations and natural disasters. Unlike the European Union, which now requires mandatory reporting of shortages, the US lacks such a requirement, further exacerbating the issue.
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EMA celebrates three decades of advancing medicine and public health in the EU
On 26th January, the EMA marked its 30th anniversary, celebrating three decades of advancing public and animal health within the EU. Over the years, EMA has expanded its role in evaluating and ensuring access to high-quality, safe medicines, especially during the COVID-19 pandemic. EMA’s future continues to focus on innovation, open collaboration, and centralising the evaluation of new medicines to meet evolving healthcare challenges.
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MHRA opens applications for Its 2025 Graduate Scheme
On 28th January, the MHRA opened the applications for the MHRA Graduate Scheme, a three-year graduate development scheme that provides an exciting opportunity to play a critical role in protecting public health and positively influencing patient safety. The scheme provides a fully financially supported study package towards a Level 7 (degree level) apprenticeship in Regulatory Affairs, with 20% of your time dedicated to this course.
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MHRA cracks down on illegal medicine trade, seizing £40M in drugs and £7.5M in criminal assets
On 29th January, the MHRA and its law enforcement partners seized over 17 million doses of illegally traded medicines, including painkillers, sleeping tablets, and erectile dysfunction treatments, with a potential street value exceeding £40 million. The MHRA’s Criminal Enforcement Unit (CEU) also denied criminals access to £7.5 million in assets by freezing bank accounts, intercepting digital currencies, and confiscating luxury goods.
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Aplagon secures €7 Million funding to advance APAC Therapeutic into Phase 2a trials
On 3rd February, Aplagon, a clinical-stage biopharmaceutical company developing a first-in-class therapeutic for thrombo-inflammatory diseases, announced a €7 million financing round to advance its APAC therapeutic into Phase 2a clinical trials. The funding, led by Fåhraeus Startup and Growth AB (FSG) and the European Innovation Council (EIC) Fund, will support trials in Finland for peripheral arterial occlusive disease and chronic limb-threatening ischemia.
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A QA Perspective on the Annual Tower Mains Get-Together
The Tower Mains get-together was a night filled with celebration, team spirit, and festive fun—but no event escapes a Quality Assurance review! As auditors at heart, we couldn’t resist assessing the evening, highlighting key findings and areas for improvement.
From overflowing festive “refreshments” to undocumented casino procedures, our audit revealed key areas for improvement. Click here to read the full report in our latest blog, featuring root cause analysis and recommendations to make this year’s celebration even more seamless!
Issue Number: WN00194
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