Regulatory news - June 2017

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 delays implementation of new Clinical Trial Regulation to 2019

Due to technical difficulties concerning the development of new IT systems for the European Clinical Trial Portal, the ‘go-live’ date for the new regulation has been postponed by the EMA until 2019. The agency had previously indicated that the new clinical trial regulation, finalised in May 2014, would come into force by October 2018.

The EU clinical trial portal and database supports the modernisation of the processes for authorisation and oversight of clinical trials in the EU. This new system is a significant shift from the current process involving independent clinical trial authorisation (CTA) by each member state, into a single portal for submission and maintenance of applications and authorisations, coordinated assessment and supervision.

The EMA is working closely with its IT service provider to ensure that corrective measures are implemented and will closely monitor its progress.

The meeting note goes on to say:

EMA’s priority is to ensure that a high quality and functional system is delivered to the EU regulatory network and its stakeholders…This is the most ambitious IT system required by the EU legislation in the last decade, involving a complete EU-wide system to be used for clinical trial applications, urgent safety measures and other notifications to regulators before, during and after the conduct of clinical trials.

The EMA management board will provide a progress update at its next meeting in October 2017.

Click here to view the press release from the agency.

Before Brexit, EMA tasks Human Medicine Working Group with redistributing the regulatory workload

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) currently handles a large portion of EMA’s regulatory workload, including processing of initial marketing authorisations, scientific advice and pharmacovigilance procedures. Whilst the MHRA’s relationship with the EU post-Brexit is still to be determined, the EMA still stands to lose access to that work capacity on 30th March 2019.

In order to manage a smooth transition and ensure resources are adequately managed to provide an uninterrupted regulatory service, the EMA announced its working group on operational preparedness on the 14th June which aims to:

  • Ensure business continuity and proportionality in the distribution of workload taking into due account other elements including but not limited to the deadline of 30 March 2019 and the average timelines of the various procedures
  • Ensure knowledge retention, either by building on existing knowledge, or through knowledge transfer (if the latter applies this should be accommodated)
  • Allow to comply with the legally required timelines and maintain the quality of the output
  • Be as easy as possible to implement and, in addition, should be sustainable (both short/medium term to address the more immediate Brexit consequences, as well as longer term)
  • Strive to allow all NCAs [National Competent Authorities] to participate in EMA activities, as per the capacity and capability of each NCA, so as to ensure an optimised and robust allocation of the workload across the Network.

The working methodology for this initative involves four key stages beginning with an initial mapping of capacity and expertise in the agency, implementing general principles and objectives, decision-making processes and finally communicating to stakeholders.

Click here to view the press release from the agency.



Enhancing patient engagement across the FDA

In March 2017, the FDA proposed a new Office of Patient Affairs (OPA) as part of an effort to offer "a single, central entry point to the Agency for the patient community," as well as “triage and navigation services for inbound inquiries from patient stakeholders." The concept was out for public comment until June 12th 2017, reciving 89 statements from various stakeholders including patient organisations, industry and charitable group.

The Biotechnology Innovation Organization (BIO), the world’s largest biotechnology trade association commented:

BIO commends the Agency’s commitment to advancing the systematic integration of patient perspective data to inform drug development and regulatory decisionmaking. Enhancing staff capacity and developing guidance and tools that will better enable the incorporation of the patient voice throughout the lifecycle of product will support our shared goal of ensuring that medicines are truly meeting the needs of patients and their families.

GSK similarly backed its support for the creation of OPA, adding that the office could take on responsibilities developing relevant FDA Manuals of Processes and Procedures, coordinating shared objectives across various FDA offices, linking up with other government agencies and serving as a regulatory center of excellence on patient engagement to promote the adoption of best practices and harmonization of guidance documents.

Click here to view the stakeholder comments.

CBER director highlights growing activities around cell and gene therapy

Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the FDA provided a few examples of changing landscape for regulating cell and gene therapies including regenerative medicines, at DIA’s annual conference this month.

In particular, the rapidly growing field of chimeric antigen receptor-t cell therapy was touched on, including challenges for process development, logistics and safety. Marks discussed the CAR-T cell database set up by the agency last year to monitor the safety of this technology across all open INDs. There are currently more than 100 INDs being tracked, many of which target the CD19 antigen.

Click here for more on FDA’s CAR-T database.

FDA Commissioner to eliminate backlog of 200 orphan drug designation requests within 90 days

At the latest Senate hearing on the 2018 budget request for the US FDA, Commissioner Scott Gottlieb has vowed to eliminate a major backlog of orphan drug designations. “We have a backlog of about 200 orphan drug designation requests where we haven't responded to sponsors…" Gottlieb said. “As part of our new plan we are committing today that in 90 days we will completely eliminate this backlog of requests and provide an answer to sponsors…We will never again develop a backlog”.

Gottlieb announced a new orphan designation review template to enable more efficient reviews and timely responses in order to meet a 90 day timeline for all future requests.

Within the statement, a number of further actions were announced including work on rare diseases.

Gottlieb stated:

We’ll be issuing a new guidance document within the next 6 months on the clinical evaluation of targeted therapies for rare disease subsets. This new policy will address targeted drugs and how we can simplify the development of drugs targeted to rare disorders that are driven by genetic variations and where disease all have a similar genetic fingerprint, even if they have a slightly different clinical expression.

Click here to watch the full FDA hearing at the Senate.

Draft Q&A guidance on electronic systems in clinical investigations published

Regulation 21 CFR Part 11 on Electronic Records; Electronic Signatures was first published in 1997 and was intended to permit the widest possible use of electronic technology. Advances in technology have expanded the uses and capabilities of electronic systems in clinical investigations. Electronic systems and technologies are used and managed in novel ways including how services are shared or contracted between organizations, and electronic data flow between parties is more efficient and more prevalent.

The standards and capabilities of electronic systems have improved, and features such as audit trails, automated date-and-time stamps, appropriate validation, and the ability to generate copies and retain records are standard components of many electronic systems.

This latest guidance addresses the applicability of part 11 requirements for systems used to create, modify, maintain, archive, retrieve, or transmit an electronic record as applied to:

  • Electronic systems, including commercial off-the-shelf (COTS) and customized electronic systems owned or managed by sponsors and other regulated entities;
  • Electronic services, outsourced by the sponsor or other regulated entities;
  • Electronic systems primarily used in the provision of medical care;
  • Mobile technology; and
  • Telecommunication systems

Regarding the use of mobile technology, the FDA says:

Sponsors and other regulated entities may use mobile technology during the course of a clinical investigation to capture, record, or transmit data directly from study participants. The recommendations in this section apply to mobile technology used in a clinical investigation whether that technology is provided by the sponsor or owned by the study participant (i.e., bring your own device (BYOD)). For the purposes of this guidance, mobile technology refers to portable electronic technology used in clinical investigations that allows for off-site and remote data capture directly from study participants and includes mobile platforms, mobile applications (mobile apps), wearable biosensors and other remote and ingestible sensors, and other portable and implantable electronic devices.

Click here to view the draft guidance document.

Public consultations



Consultation Period



Post-implementation Review of the Human Medicines Regulations 2012

15 Jun 2017 to 6 July 2017

Draft Review of regulations





Consultation Period



Toward Mandatory Reporting of Serious Adverse Drug Reactions and Medical Device Incidents by Health Care Institutions

28 Jun 2017 to 11 August 2017

Draft Consultation Paper