Industry Insights for Q2 – 2019

The second quarter has flown by and we are still living with the uncertainty of Brexit. However, Christina Hägglund can be relied upon to bring the latest updates from the global regulatory authorities. If you want to find out more about our products or services, please don’t hesitate to contact us.


The European Medicines Agency has released updated guidance for industry on preparing for Brexit.

The EMA has also published a Q&A document on preparatory work that European Union authorities are doing to prevent medicine shortages which you can read here.

Access to Medicines

UK and Compliance with Clinical Trial Transparency Rule

The Transparency Rule demands that clinical trial results are recorded in a publicly accessible registry, that summary results are published within a set time after trial completion and that trial results are reported in full. The Health Research Authority (HRA) acknowledges that not all studies are registered and that the results of many are not reported publicly. Non-publication of results means information on the efficacy of new drugs or other medical interventions cannot be used. A new strategy of clinical trial transparency is being developed, with sanctions for non-compliance a possibility.

You can read the Government response to the House of Commons Science and Technology Committee report on research integrity: clinical trials transparency here. 

Download the EudraCT Reporting Tips here.

FDA – Improve Efficacy of Drug Development

New FDA guidance could improve the efficiency of drug development and support precision medicine, by using a specific patient characteristic to select a study population. The detection of a real drug effect (efficacy or safety) may be more likely in this selected study population compared with an unselected population.

FDA – Breakthrough Therapy Designation (BTD)

The BTD is a regulatory process designed to expedite the development and review of drugs that are intended to treat a serious condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints.

Sponsors granted this designation can take advantage of compressed clinical trials, advice and input from dedicated FDA personnel and a streamlined FDA evaluation process.

Europe – clinical trial regulation (CTR – 536/2014)

Progression to the EU CTR

The EC maintains Volume 10 of ‘The Rules Governing Medicinal Products in the European Union’ on its website.

Volume 10 has been updated to bring it in line with changes required by the EU CTR. Inevitably there will be a period when some clinical trials in Europe are being performed under the current EU Clinical Trials Directive (CTD – 2001/20/EC) whilst others are initiated under the CTR. In order to distinguish between these two guidance documents the EC now has two complementary pages on the Eudralex Volume 10 website (Page 1) and (Page 2) is here.

CTR and General Data Protection Regulation (GDPR)

The EC has released a Q&A document explaining the interplay between the CTR and the GDPR. The document reviews the data protection requirements of the CTR in the context of GDPR. It acknowledges that, in the short term, trials in Europe will continue to be performed under the EU Clinical Trials Directive.

EU Guidance Documents/Recommendations

Recommendations for Complex Clinical Trials

This document provides recommendations for sponsors regarding the authorisation and conduct of complex clinical trials. The document describes the current perspective of the Clinical Trials Facilitation and Co-Ordination Group (CTFG) on these trials and outlines the major issues that sponsors should address in the process of initiating and conducting complex clinical trials in the EU/EEA.

Sub-Group Analysis

This is the latest EMA Guideline on the investigation of subgroups in confirmatory clinical trials.

Guidance on New EU Regulations of Medical Devices

EMA has published the first of a series of guidance documents to help applicants prepare for obligations stemming from the new EU regulations on medical devices.

Questions & Answers on Implementation of the Medical Devices and In Vitro Diagnostic Medical Devices Regulations ((EU) 2017/745 and (EU) 2017/746). This Question and Answer (Q&A) document provides practical considerations concerning the implementation of the medical devices and the in vitro diagnostic medical devices regulations

FDA guidance documents

The Effect of Food on Medicines

Assessing the Effects of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations

Regenerative Medicine Products

Evaluation of Devices Used with Regenerative Medicine Advanced Therapies

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions

Improving Representation in Oncology Studies

Cancer Clinical Trial Eligibility Criteria: Minimum Age for Pediatric Patients

Cancer Clinical Trial Eligibility Criteria: Patients with HIV, Hepatitis B Virus, or Hepatitis C Virus Infections

Cancer Clinical Trial Eligibility Criteria: Patients with Organ Dysfunction or Prior or Concurrent Malignancies

Cancer Clinical Trial Eligibility Criteria: Brain Metastases

Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials

Updated Guidance on Risk-Based Monitoring

A Risk-Based Approach to Monitoring of Clinical Investigations Questions and Answers


EU – New EudraVigilance System Improves Detection of Safety Signals

The assessment of safety signals, a routine part of pharmacovigilance, establishes whether there is a causal relationship between the medicine and the reported event.

In the European Medicine Agency’s (EMA’s) 2018 annual report, it is acknowledged that the new EudraVigilance system (launched in November 2017) has encouraged early detection and evaluation of possible safety signals, leading to a safer and more effective use of medicines in Europe.

UK – MHRA GCP Data Integrity Requirements

Compliance with GCP, including compliance with standards relating to data integrity, is typically verified through audit and inspection. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) Inspectorate has shared answers to some of the key questions it has received on data integrity in the context of GCP. The MHRA published a much broader guidance on GxP data integrity just over 12 months ago.

FDA – Patient Experience Data

The FDA is catching up with other regulatory agencies by embarking on efforts to incorporate meaningful patient experience data into its regulatory decision making. Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.

Contact our Regulatory Affairs team, if you require any further guidance or support on any regulatory matters.