Regulatory news - August 2018

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


European Medicines Agency (EMA)

EMA to suspend or reduce certain activities in view of Brexit

EMA have announced that in the lead-up to 1st October 2018,it expects to launch the next phase of its business continuity plan which includes suspending or temporarily reducing activities to focus on its essential public health activities. The reduction of activities will allow the EMA to intensify preparation for its relocation to Amsterdam in 2019, as well as manage significant EMA staff loss (estimated to be 30%) and train EMA staff who will be reassigned to new duties.

In consequence, the EMA will temporarily:

  • Limit collaboration at international level – unless these relate to product-related requests, supply-chain integrity and procedures under Article 58
  • Limit development and revision of guidelines unless required as part of an urgent public/animal health need or to support preparations for Brexit
  • Reduce meeting frequency of non-product-related working parties
  • Limit organisation attendance at stakeholder meetings unless Brexit related
  • Suspend clinical data publication – started as of 1 August 2018

Click here to view the press release for information.

United Kingdom (UK)

Brexit – Guidance published on preparing for a ‘no-deal’ Brexit scenario

The UK government has published the first series of advice documents to inform stakeholders of the implications in the event of a no-deal Brexit scenario. Several of these documents consider the implications for the regulation and supply of medicines.



How medicines would be regulated if there’s no Brexit deal

The document describes the arrangements that will come into force for human medicines regulation currently subject to EU rules. ATMPs are authorised via centralised procedure. In case of no deal, the below will apply:

Converting centrally authorised products (CAP) to UK MA

On 29th March 2019 all CAP MAs will automatically be converted into UK MAs. Marketing Authorisation Holder (MAHs) will have a period of time from exit day to provide MHRA with baseline data for CAPs that are converted into UK MAs, the exact requirements to be communicated at a later date

Initial MA applications

An initial MA application will need to be submitted to the MHRA and will go through a national assessment. The UK will no longer be a part of the EU centralised, mutual recognition and decentralised procedures

In-progress licensing procedures at time of exit

Any EU procedures that have not reached the decision phase at the time that the UK exits the EU will not be valid in the UK. For centralised procedures in progress at time of EU exit the application will need to be submitted to the MHRA.

MHRA will take into account any CHMP assessment that had already taken place.

Submitting regulatory information on medical products if there’s no Brexit deal

This notice provides the UK Government’s position on how regulatory information will be practically submitted to MHRA, particularly for processes currently managed by shared EU wide systems (e.g. Common European Submission Portal(CESP), Eudravigilance, EudraCT and new CTR portal).

The government confirm that the UK would need its own national portals. The portal systems would adopt the following principles:

  • Continue to accept EU application forms and EU standards for submissions where possible
  • Accept eCTD submissions in relation to MAs
  • Systems to be live by March 2019 but to be developed further over time

Stakeholders would be asked to test the systems prior to March 2019 and further information will be provided at a later date.

Developing genetically modified organisms (GMO) if there’s no Brexit deal

Regulatory decisions on proposed GMO trials will continue to be made within the UK on a devolved basis

Ensuring blood and blood products are safe if there’s no Brexit deal

It is recommended that the MHRA is consulted prior to importing or exporting blood or blood components to or from the EU.

  • Import from EU: establishments would be required to add a description of activity to cover import to their blood establishment authorisation
  • Export to the EU: establishments may need to certify that any products comply with EU standards

The current blood safety and quality standards for blood and blood components would not change

Quality and safety of organs, tissues and cells if there’s no Brexit deal

UK licensed establishments would continue to work to the same quality and safety standards as they did before exit, but some would need new written agreements with relevant EU establishments.

Click here for additional information.

MHRA publishes new guidance for patients previously treated with ATMPs, joining new ATMP clinical trials

Patients previously treated with an Advanced Therapy Medicinal Product (ATMP) have been prohibited from participating in new clincial studies in the United Kindom (UK). However, based on recent data, MHRA publishes a guidance to allow patients which may be amenable to a new therapy to enter new clincial trials if certain criteria are met.

The guidance provides information on a number of relevant topics to consider in these cases for a new Clinical Tral Authorisation (CTA) application including the trial rationale and population, the Investigational Medicinial Product (IMP) characteristics, risk mitigation strategies, benefit-risk assement, scientific validity of the trial, blood sample collection and ethical issues.

The guidance generally points sponsors towards a rigorous and data-driven examination of the safety and efficacy assessment of a new IMP in relation to any previously administered ATMPs based on any relevant data available. In the absence of information about the persistence and long-term safety of previous ATMPs the eligibility criteria should initially be conservative and modified subsequently only if accumulated data support a broadening of the trial population.

Given the increasing number of ATMPs being investigated clincial trials, this new guidance helps provide greater treatement options to patients with a high unmet medical need.

Click here to view the document in full.


Food and Drug Administration (FDA)

Draft FDA Guidance on use of expansion cohorts in first-in-human clinical trials to expedite development of cancer drugs and biologics

The targeted treatments in oncology over the last decade have shown great potentials to treat patients. The FDA published a draft guidance to help advance effective and innovative clinical trial designs early in drug development to help bring new cancer therapies to patients as quickly as possible. The document provides advice on designing and conducting adaptive trial designs in which the sponsor can assess many different aspects of a drug in development in a single clinical trial while enrolling the minimum number of study participants necessary to obtain this information. The real benefit of the use of the expansion cohort resides in the efficiency in drug development. The document includes:

  • characteristics of drug products best suited for consideration for development under a multiple expansion cohort trial;
  • information to include in investigational new drug application (IND) submissions to support the use of individual cohorts;
  • when to interact with FDA on planning and conduct of multiple expansion cohort studies; and
  • safeguards to protect patients enrolled in First In Human (FIH) expansion cohort studies

Click here to view the draft guideline.

US works towards streamlining review of gene therapies

Given the recent scientific and clinical breakthroughs in gene therapy, the National Institutes of Health (NIH) and FDA are working together to streamline the oversight for human gene transfer clinical research protocols while reducing unnecessary duplicative reportive requirements. As a consequence, the NIH will

  • no longer accept new human gene transfer protocols,
  • no longer accept annual reports, safety reports, amendments or other documentation for any previously registered human gene transfer protocols
  • Restore the Recombinant DNA Advisory Committee (RAC) initial vision to focus on emerging biotechnologies, such as gene editing, synthetic biology, and neurotechnology

Click here to view more information.

FDA to run workshop on “Quantification of AAV-based Gene Therapy products

FDA has announced it will hold a public workshop on the 7 December 2018 on “Quantification of AAV-based Gene Therapy products”. AAV-based gene therapies are promising treatment approach for a variety of genetic diseases. The objective of the workshop is to bring all stakeholders together and discuss best practices when measuring the concentration of AAV vectors including:

  • Overview of AAV concentration-determining assays
  • Critical elements for assay optimization and qualification
  • FDA perceptive on qualification and validation of AAV concentration assays
  • Development of AAV concentration assays: Case Studies

Click here to view more information.

Public consultations




Consultation period



Good Clinical Practice for Advanced Therapy Medicinal Products

01 Aug to 31 October 2018

Draft guidance




Consultation period



Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018

Draft guidance