Industry Insights for Q4 – 2019

Our latest regulatory updates for the final quarter of the year covers news from the FDA, EMA, important news about nitrosamines impurities, and much more. Christina Hägglund compiles the latest regulatory updates from around the globe. Need support? Get in touch.


As a consequence of the recent discovery that certain EU medicines contained nitrosamine impurities, which are probable human carcinogens, EU Marketing Authorisation Holders are being requested to conduct a risk evaluation for the presence of nitrosamine impurities in their medicines. EMA have published an updated Question & Answers document to provide additional guidance on the steps that companies should take. You can read full details here. The initial risk evaluation need to be completed by end of March 2020. Our team of experts at S-cubed can support you to fulfil the requirements. Get in touch for advice.

TOPRA Top Team Winners 2019

TOPRA CRED LogoS-cubed is proud to announce that Stephen Thompson, Director of Regulatory Affairs, is part of the CRED (Continuing Regulatory Education and Development programme) Clinical Trials Working party which has been recognised as this year’s TOPRA, Top Team. Alongside, Shaila Choi (Chair), Chris Parkinson and Pierre Omnes, Stephen and the team have ensured that the courses are of a consistently high standard and that all post course queries are answered. Congratulations to team for this highly deserved recognition.

Global Drug Development

Food and Drug Administration (FDA) and European Medicines Agency (EMA) Marketing Applications

Drug Development is a global enterprise and alignment between agencies for new drug approval is vital.
The results following a 3 year study have been published and show that the FDA and EMA agreed 91% of the initial decisions for marketing applications for new drugs.

Patients Preference

The results of a survey evaluating the view of nearly 200 patients shows a preference for the use of mobile technologies in clinical trials. Mobile technologies may also increase the quality and efficiency by improving participation rates and participants’ experience, as well as capturing more informative real-world data. (Read our previous article on Real World Evidence)

Securing a Healthier Future in Europe

The European Federation of Pharmaceutical Industries and Associations (EFPIA) represents the European pharmaceutical industry. One of its missions is to create a collaborative environment that supports a healthier Europe based on prevention, innovation, access to new treatments and better outcomes for patients. Read A Healthier Future – No Quick Fixes.

UK – Pilot to Encourage Development of New Antibiotics

Antimicrobial resistance (AMR) means antibiotics should only be used when needed. Drug companies are paid by the volume of antibiotics sold and may therefore be reluctant to invest in the development of new antibiotics. A new system is therefore being piloted to encourage development of new antibiotics, where drug companies are paid for the drug even though it may be stored and held in reserve until needed. Find out more from GOV.UK.

Expedited Drug Development (& potential issues)

EMA and FDA Aim to Support Sponsors Seeking Early Access Approaches

In 2018, the EMA and FDA held a workshop to identify approaches to facilitate the development and preparation of robust quality data packages for medicines of the early access programmes. The meeting report (published July 2019) captures the workshop’s conclusions, reflecting the views of industry and key regulatory agencies.

FDA Expedited Development Programmes

The FDA has five programmes aimed at expediting patient access to important drugs that treat serious or life-threatening conditions.
There has been a misconception that these programmes are increasingly used because they have reduced approval standards. An article in FDA Voices, ‘Perspectives from FDA Leadership and Experts’ reports however that all FDA drug approvals receive the same high safety and efficacy approval standards.

EMA Expedited Development Programmes

The EMA offers several expedited approval pathways to facilitate the development of medicines that address unmet medical needs and have shown to reliably reduce the time to marketing authorisation. A review of medicines authorised in the EU between 2011 and 2018 suggests, however, that the EMA’s expedited pathways are over-reliant on non-validated surrogate endpoints, rather than clinical outcomes, and that this may not be obvious to prescribers and patients.

UK Speeds Access to Pioneering Treatment

The UK’s Accelerated Access Collaborative (ACC) is designed to select the most promising new treatments, tests and medical devices at an early stage, and to steer them through the clinical development and regulatory approval process faster. New improvements to the ACC are intended to ensure that pioneering treatments are fast-tracked into the National Health Service (NHS) in order to reach patients more quickly. Full details from Health Data Research UK here. 

Clinical Data & Post-Marketing Studies

EMA – Public Access to Clinical Data

The EMA publishes clinical trial data submitted by pharmaceutical companies to support marketing authorisation applications made under the centralised procedure. The EMA has suspended all new activities related to clinical data publication under its business continuity plan. The Agency has, however, co-signed a letter with the European Commission and the Heads of Medicines Agencies (HMA) reminding clinical trial sponsors of their obligation to provide results of their EU trials on the EU Clinical Trials Database.

FDA to Expand Sentinel System

The Sentinel System enhances the FDA’s ability to proactively monitor the safety of marketed medical products and complements other systems that focus on spontaneous safety reporting. The FDA now plans to broaden Sentinel’s capabilities and ongoing value in regulatory decision making, particularly through the use of real-world data and real-world evidence.

FDA – Post-Marketing Requirements

The FDA is increasingly adopting a lifecycle evaluation process. More emphasis will be placed on evidence generated in the post-marketing environment, as part of an overall therapeutic evaluation.  A recently published study has examined how often post-marketing commitments, agreed by pharmaceutical companies at the time of first FDA approval, actually lead to new clinical trials being performed.

UK – Data Hubs to Harness Data and Support Clinical Development

Health Data Research UK has announced seven new digital innovation hubs that will help the UK to make the most of health-related data for future research and development into new medicines. The seven hubs are: Acute Care; Cancer; Clinical Trial; Eye Health; Inflammatory Bowel Disease; Respiratory; Use of Real-World Data.

Guidelines & Guidance Documents

ICH Releases E17 Guideline Training Material

In November 2017, E17 guideline ‘General Principles for Planning and Design of Multi-Regional Clinical Trials’ was finalised. ICH has now developed a comprehensive set of training materials to promote the efficient and consistent implementation of the guideline.

FDA – ICH E8(R1) ‘General Consideration for Clinical Studies’ Draft

Read the full document here.

FDA – ‘Patient Engagement in Design and Conduct of Medical Device Clinical Investigations’ Draft Guidance

Read the full document here.

FDA – ‘Population Pharmacokinetics’ Draft Guidance

The revised draft guidance reflects significant improvements in PK analysis methods since the original 1999 guidance.

FDA – Patient Focused Drug Development Guidance Documents

Patients who live with a disease understand the benefits and risks throughout the drug development process. The documents are likely to take 5 years to develop, but aim to show sponsors how best to integrate the patient voice into their drug development programmes.

FDA – ‘Placebo and Blinding in Randomised Controlled Cancer Clinical Trials for Drug and Biological Product’ Guidance for Industry

Read the Guidance for Industry here.

FDA – ‘General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products’ Draft Guidance

Read the Draft Guidance here.

UK – Joint Working Toolkit Guidance

Joint working projects will enable pharmaceutical companies, the NHS and others to pool their skills, experience and resources to jointly develop patient-centred projects. A 10-step process for scoping, setting up and monitoring has been generated.

UK – Medicines and Healthcare product Regulatory Agency (MHRA) Medical Device Guidance

The MHRA has updated its 2013 Guidance ‘Clinical Investigation of Medical Devices – Biological Safety Assessments’ for consistency with the EU medical device regulation.

UK – MHRA Inspectorate

Electronic Health Records (EHRs) are increasingly being used within the NHS. A recent blog post from the MHRA Inspectorate outlines common inspection findings with EHRs and provides guidance on how to ensure GCP compliance.

If you need to know more about how we can assist you with our services or productsfeel free to get in touch. And we hope you find our updates useful and informative.