Case study

Agile, responsive regulation helps early breast cancer patients to benefit from a study to potentially improve survival outcomes

Following MHRA approval of a clinical trial, early breast cancer patients on endocrine-based therapy can benefit from a study with camizestrant.

Background

Breast cancer is the most common cancer and is one of the leading causes of cancer-related deaths worldwide.

Data published between 2016 to 2018 suggests that close to 56,000 new cases of breast cancer are diagnosed in the UK, mostly affecting females, and with 86% presenting with early disease.

Most breast cancers have proteins (receptors). These include oestrogen receptor-positive (ER+), where breast cancers have receptors for the hormone oestrogen, and HER2-negative, where the protein is absent – human epidermal growth factor 2.

Hormones, particularly oestrogen, can attach to these receptors and encourage the cells to grow. A pathologist can identify the receptors during biopsy or surgery, helping to determine treatment.

Recent research has led to the development of new candidate drugs known as SERDS (selective oestrogen receptor degraders). These drugs are designed to attach to and disrupt these oestrogen receptors, preventing the growth of cancer.

A new SERD molecular entity, camizestrant, is under clinical development by biopharmaceutical company AstraZeneca and is currently in a Phase III study. The drug candidate is administered orally in the form of a film-coated tablet and acts by targeting oestrogen receptors.

This Phase III open-label study in early breast cancer enrols 5,500 patients worldwide. It aims to assess if camizestrant improves survival outcomes compared to standard adjuvant endocrine-based therapy for patients who are ER-positive and HER2-negative.  

Good practice clinical trials application

The MHRA’s agile regulatory framework for clinical trials supports innovation in the life sciences sector by assessing and approving safe, effective, and efficient trials that benefit patients in the UK and around the world.  

AstraZeneca submitted a compliant application for the clinical study to the MHRA in September 2023. Their cover letter addressed the recommended points of reference for the MHRA assessors, including signposting and highlighting certain areas that have changed, and cross referencing to other studies and guidance.

The company focused on the quality, effectiveness, and safety profile of camizestrant and the other investigational medicinal products relevant to the patient population. The protocol and documents appropriately reflected the risks and mitigation measures, making the task as efficient as possible for the MHRA’s assessors. 

An agile and responsive regulator  

The MHRA’s expert assessors took a risk-proportionate, targeted approach to reviewing the camizestrant clinical trial application. This medicine had already been used in people with cancer, so evidence was available to support the application.

Whilst acknowledging that this study design was simple, the protocol was well written and clearly presented. The agency was adequately assured of the pharmaceutical quality, risk mitigation measures and safety reporting to support a favourable risk/benefit balance for the trial.  

Decision

The MHRA issued a decision in 25 calendar days. The final outcome (acceptance) for the study was in 32 days. No grounds for non-acceptance were issued from any MHRA assessors, and the responses from the Research Ethics Committee and Health Research Authority were received promptly.  

Since 1st September 2023, the MHRA has assessed all initial applications within the statutory timeframe of 30 days.

“This continually improving performance in regulatory timescales, along with a holistic national health service, an active academic research community and an ethnically diverse population, makes the UK a globally competitive location for clinical research,” comments Andrea Manfrin, the MHRA’s Deputy Director, Clinical Investigations and Trials.

“Our experts are dedicated and pragmatic in their approach to assessing trials. We are committed to providing predictable and reliable timescales for Clinical Trial Authorisation applications, so that sponsors can plan with confidence their studies in the UK.”

The MHRA receives between 950 and 1,000 clinical trial authorisation (CTA) applications for investigational medicinal products (IMPs) per year, with more than half requiring additional information to be submitted before they are considered approvable.

To assist applicants, the MHRA has published online guidance that identifies common issues with the validation and assessment of clinical trial applications and how to avoid them throughout the process, helping to ensure that clinical trials are assessed efficiently.

Applicants can also ask for scientific advice from the MHRA at any stage of the development of their medicine.

Advice on specific or regulatory aspects of a clinical trial can also be obtained via the agency’s Clinical Trials Helpline at clintrialhelpline@mhra.gov.uk.

See Common issues identified during clinical trial applications.

References

About the MHRA

An executive agency of the Department of Health and Social Care (DHSC), the Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All the agency’s work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks. 

About AstraZeneca

AstraZeneca is a biopharmaceutical company which is focused on the discovery, production, and commercialisation of a range of prescription drugs. It develops products related to therapy areas such as respiratory, cardiovascular, renal, and metabolic diseases, as well as cancer, autoimmune, infection, and neurological diseases.

The company’s product portfolio includes biologics, prescription pharmaceuticals, and vaccines. AstraZeneca sells its products through wholly-owned local marketing companies, distributors, and local representative offices. The company markets its products to primary care and speciality care physicians and operates in Europe, the Americas, Asia, Africa, and Australasia. AstraZeneca is headquartered in Cambridge, Cambridgeshire, the UK.

Published 28 March 2024