Tower Mains Weekly Newsletter
Regulatory Updates
MHRA guidance updates
- Innovative Devices Access Pathway (IDAP). On 29th February, the Medicines and Healthcare products Regulatory Agency (MHRA) added Data Protection and Privacy information to guidance on the Innovative Devices Access Pathway (IDAP), a pilot designed to accelerate the development of innovative medical devices that meet an unmet clinical need in the NHS and support their integration into the UK market. Click to view source
- Established Medicines marketing authorisation application process changes. On 7th March, the MHRA updated details of the process changes for “Established Medicines”, effective from 1 March 2024, which apply specifically to ‘chemical’ products. Click to view source
FDA guidance updates
- Key Information and Facilitating Understanding in Informed Consent Guidance for Sponsors, Investigators, and Institutional Review Boards. On 29th February, the US Food & Drug Administration (FDA) published draft guidance providing recommendations related to two provisions of the revised Federal Policy for the Protection of Human Subjects (the revised Common Rule) by the U.S. Department of Health and Human Services (HHS) and identical provisions in FDA’s proposed rule “Protection of Human Subjects and Institutional Review Boards.” It addresses the provisions of the revised Common Rule that require informed consent to begin with key information about the research and to present information in a way that facilitates understanding and identical provisions in the FDA’s proposed rule. Click to view source
- Q2(R2) Validation of Analytical Procedures. On 6th March, final guidance was published providing a general framework for the principles of analytical procedure validation, including validation principles that cover the analytical use of spectroscopic data. Click to view source
- Q14 Analytical Procedure Development. On 6th March, final guidance was published providing harmonised guidance on scientific approaches for analytical procedure development and describing principles to facilitate more efficient, science-based, and risk-based post-approval change management. Click to view source
European Commission updates
- Guidance for the Transition of Clinical Trials from the Clinical Trials Directive to the Clinical Trials Regulation. The European Commission published the latest version of the Guidance for the Transition of Clinical Trials from the Clinical Trials Directive to the Clinical Trials Regulation, which has been updated to align with the most recent guidance from the Clinical Trials Coordination Group (CTCG) on transitions. It includes information on the consequences of not complying with transition obligations, stating that sponsors may face corrective measures from Member States as per Article 77 of the Clinical Trials Regulation.
- Clinical Trials Regulation Q&A. The European Commission published the latest version of the Clinical Trials Regulation Questions and Answers (CTR Q&A), which has been updated to version 6.8. Changes include revisions to questions 1.17, which now aligns with the updated guidance on auxiliary medicinal products, and 1.19, referencing an upcoming recommendation paper from the Clinical Trials Coordination Group (CTCG) on the principles of good laboratory practice (GLP) relevant to clinical trial authorisation.
ICH Q12 IWG “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management”
On 4th March 2024, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) announced the publication of the ICH Q12 “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management Introductory Video” following the publication of the ICH Q12 Module 8.
Industry Updates
FDA Voices: Helping deliver meaningful advances for rare disease patients
On 29th February, the FDA published the FDA Voices: “FDA Helps to Deliver Meaningful Advances for Rare Disease Patients,” by FDA Commissioner Robert M. Califf, M.D., and Sandy Retzky, D.O., J.D., M.P.H., director, Office of Orphan Products Development. Advancing the understanding of rare diseases and innovative tools to support product development is part of the FDA’s commitment to promoting safe, effective treatment options for patients. The agency strives to help include patient experience into development programs for all medical products, and considers the patient experience, as appropriate, when it reviews the safety and efficacy of these products.
MHRA grants first approval via the new International Recognition Procedure
On 1st March, the MHRA authorised a new formulation for XGEVA (denosumab), a treatment used in adults to prevent serious bone-related complications caused by bone metastasis and to treat giant cell tumours of bone in adults and adolescents. This is the first product to be authorised by the MHRA via the new International Recognition Procedure (IRP), and was authorised in 30 days, providing UK patients with earlier access to this treatment. IRP allows the MHRA to accelerate the assessment of new medicines by taking into account the expertise and decision-making of trusted regulatory partners in the authorisation process.
Johnson & Johnson secures FDA approval for RYBREVANT to treat lung cancer
On 1st March, Johnson & Johnson announced that following a priority review, the FDA has approved RYBREVANT® in combination with chemotherapy for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test.
FDA clears first over-the-counter continuous glucose monitor
On 5th March, the FDA cleared for marketing the first over-the-counter (OTC) continuous glucose monitor (CGM). The Dexcom Stelo Glucose Biosensor System is an integrated CGM (iCGM) intended for anyone 18 years and older who does not use insulin, such as individuals with diabetes treating their condition with oral medications, or those without diabetes who want to better understand how diet and exercise may impact blood sugar levels.
Rubidium (Rb82) Generator approved as a diagnostic tool for people with heart disease
On 6th March, the MHRA approved the Rubidium (Rb82) Generator (RUBY-FILL) as a diagnostic tool for imaging of the heart, to evaluate blood flow and aid in the diagnosis or assessment of suspected or known coronary heart disease.
DARWIN EU® to continue to expand its capacity to deliver real-world data studies
On 6th March, the European Medicines Agency (EMA) announced that the Data Analysis and Real World Interrogation Network (DARWIN EU) will be working towards higher capacity for real-world data (RWD) studies in 2024 and seeks to add ten new data partners in 2024. The network has now completed its establishment two years after the Coordination Centre was set up in February 2022. Since then, 14 RWD studies have been completed and eleven are ongoing. They cover drug utilisation, vaccine effectiveness, disease epidemiology and patient characterisation and help EU regulators to better understand diseases, populations and the uses and performance of medicines.
MHRA approves Ganaxolone as first anti-seizure medication to treat patients with rare epileptic seizure disorder
On 7th March, the MHRA approved the medicine ganaxolone as the first anti-seizure medication in the UK to treat cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD). Ganaxolone is a steroid that attaches to specific receptors in the brain and aims to stop epileptic seizures and is taken orally through a dosing syringe. The approval is supported by evidence from a phase 3 randomised, double-blind, placebo-controlled clinical trial involving 101 patients with CDD aged 2 to 19 years.
Issue Number: WN00152
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