Regulatory news: August 2016

European Medicines Agency (EMA)

EMA publishes adaptive pathways report: key learnings and next steps

The EMA has issued a summary report on the practical experience gained from the adaptive pathways pilot, launched in March 2014.

EMA’s adaptive pathway is a product development concept aimed at providing timely access to promising medicines that address an unmet medical need. It makes use of existing approval tools, in particular those implemented during conditional marketing authorisation and post-marketing pharmacovigilance monitoring. Importantly, it also involves cooperation between various key stakeholders involved throughout the life-span of a medicine (including Health Technology Assessment (HTA) bodies) with a view to incorporating these requirements at an early stage of development.

EMA received 62 applications during the pilot, 18 were selected for in-depth, face-to-face meetings with the participation of other stakeholders. Six applications progressed to receive formal parallel advice by EMA and HTA bodies and one to benefit from simple scientific advice. The majority of the proposals received were considered not suitable for adaptive pathways, and the companies were advised to pursue traditional development routes. Adaptive pathways is still a developing concept and a workshop is being run on 8 December 2016 to gather views and proposals from stakeholders on the approach.

Click here for the full report.

EMA publishes initial statistics from new PRIME procedure

Results from the intitial series of applications received for the PRIME scheme by EMA have been published their website. The PRIME procedure aims to accelerate the development and assessment of therapies that address serious unmet medical needs. The main difference between PRIME and the adaptive pathways (mentioned above) is that the former builds on the agency’s exisiting accelerated assessment procedure whilst the latter is based on the conditional marketing authorisation procedure.

From a total of 9 recommendations for eligibility to PRIME in July 2016, 2 applications were granted (both were ATMPs) and 7 were denied. To-date, the majority of recommendations for PRIME eligibility have been for indications in oncology (4 applications accepted, 8 denied).

Click here for more information.

Committees

CAT publishes July 2016 report of application procedures, guidelines and related documents on advanced therapies

The Committee for Advanced Therapies (CAT) held its 84th meeting on 13- 15 July 2016.

Highlights:

• 5 scientific recommendations finalised on the classification of ATMP products

• Presentation given regarding a public CAT workshop on cell-based cancer immunotherapies which will take place on 15 – 16 November 2016

CAT highlights for 2016:

• 1 MAA submission

• 2 positive draft opinions from CAT

• Scientific advice procedure for ATMPs: 43 discussed, 31 procedures

• PRIME Eligibility for ATMPs: 9 discussed

Click here for the full report.

Newsletters

SME newsletter: August 2016

Guideline implementation for Nov 2016 - Process validation for the manufacture of biotechnology-derived active substances and data to be provided in the regulatory submission

This guideline (EMA/CHMP/BWP/187338/2014) comes into effect on November 1^st 2016, covering process characterization and verification requirements for submission of a marketing authorisation or variation.

Click here to view the guidance.

UK

MHRA

Good clinical practice (CGP) inspection checklist for clinical trials

The MHRA have updated the GCP inspection checklist for organisations to ensure all the relevant documentation is included in submissions.

Click here to view the GCP Inspection Dossier checklist.

For more detail on the MHRA’s expectations for GCP standards and the inspection process click here.

MHRA and making a success of Brexit

Following the outcome of the EU referendum, the MHRA is working closely with the UK government to analyse the best options and opportunities available for the safe and effective regulation of medicines and medical devices in the UK. The MHRA will be engaging widely with stakeholders to fully understand and maximise the opportunities of Brexit.

Clinical Trials Regulations – have your say. Deadline for feedback 31 Aug 2016

The MHRA is assessing the potential impact on our regulatory framework of the decision to leave the EU. At present, it is business as usual and the MHRA invites feedback on Clinical Trial Regulation guidance documents.

There are currently four consultations on guidance documents that have been developed in preparation for the implementation of the Clinical Trial Regulation (EU) No 536/2014:

• “Risk proportionate approaches in clinical trials” - providing further information on how a risk proportionate approach can be implemented in clinical trials and highlighting areas within the clinical trials Regulation which support such adaptations;

• Revision of the “Definition of Investigational Medicinal Products (IMPs) and use of Auxiliary Medicinal Products (AMPs)” (previously called “Guidance on Investigational Medicinal Products (IMPS) and Non-Investigational Medicinal Products (NIMPs));

• Revision of “Ethical Considerations for Clinical Trials on Medicinal products conducted with Minors”; and

• “Summary of Clinical Trial Results for Laypersons”.

The documents for feedback are located on the EC website.

Good pharmacovigilance practice (GPvP)

The GPvP compliance report requirements have been updated. Over the last seven years the MHRA GPvP inspectorate has requested organisations with UK marketing authorisations to periodically complete and submit GPvP compliance reports (also known as Risk-Based Inspection questionnaires). The data in these reports was used to support MHRA inspection scheduling and planning activities and to understand the number of pharmacovigilance systems in operation in the UK. As of 2016, the MHRA no longer requires routine compliance reports to be submitted. Organisations will be contacted directly by the GPvP Inspectorate in the event that a specific request to provide information relating to authorisations and pharmacovigilance systems are required.

Click here for details on compliance report.

Medical devices: UK notified bodies

The MHRA provides a list of UK notified bodies. Links are now provided to the European Commission's NANDO database where the Notified Body's full designated scope is publicly accessible and downloadable.

Click here for details.

Information on when software applications are considered to be a medical device and how they are regulated

Many manufacturers, software developers, academics, clinicians, patients and organisations use software applications (apps) for both healthcare and social care needs. The MHRA have issued guidance which explains how this type of technology is regulated and includes examples of what can be considered a medical device.

Click here for the guidance.

Human Tissue Authority (HTA)

New HTLV-1 Policy developed for donors of tissue human application

The HTA has issued a Human T-lymphotropic Virus, type I (HTLV-1) policy to further clarify and to promote consistency in how licensed establishments apply the mandatory minimum testing requirements for HTLV-1. The policy explains the purpose for testing and identifies when and to which donor population mandatory testing applies, the scientific basis for repeat testing, alternative testing methods and the HTAs recommendations for testing.

Click here for details.

Health Research Authority (HRA)

Revised Department of Health contract will benefit researchers and patients

Clinical research funded by an NIHR research programme will be able to receive payments for start-up in advance of ethical approval. This will enable more rapid, efficient and streamlined set-up of research and quicker translation of research into patient benefit. This change will enable the appointment of skilled research staff earlier in the project leading to better quality applications for HRA approval, fewer protocol amendment applications early on in the project, and a quicker and more efficient start up process. This change will support researchers as part of the HRA’s system wide approach to work with its partners to streamline research in the UK. This change will enable CTUs to assemble the multidisciplinary teams required to deliver clinical trials research at the start of the set-up process so facilitating the approvals process and hence impacting on the timely and efficient delivery of clinical trials research.

Click here for further details.

USA

FDA Upcoming Workshops, Meetings & Conferences

Public Workshop 8th September 2016; Scientific Evidence in the Development of Human Cells, Tissues, and Cellular and Tissue-Based Products Subject to Premarket Approval.

The purpose of the public workshop is to identify and discuss scientific considerations and challenges to help inform the development of human cells, tissues, and cellular and tissue-based products (HCT/Ps) subject to premarket approval, including stem cell-based products.

Click here for details.

Recently Issued Guidance Documents (Biologics)

Early Clinical Trials with Live Biotherapeutic Products (LBPs): Chemistry, Manufacturing, and Control Information

The US Food and Drug Administration has announced the availability of a document entitled “Early Clinical Trials With Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information; Guidance for Industry.” The guidance document provides investigational new drug application (IND) sponsors with recommendations regarding IND submissions for early clinical trials with live biotherapeutic products (LBPs) in the United States. This document updates the guidance of the same title dated February by addressing when the label on the commercially available products(s) would be considered adequate to satisfy the purpose of the chemistry, manufacturing, and control (CMC) information requirements.

An LBP, for the purposes of this guidance document, is a biological product that: 1) contains live organisms, such as bacteria; 2) is applicable to the prevention, treatment, or cure of a disease or condition of human beings; and 3) is not a vaccine. For the purposes of this document, LBPs are not filterable viruses, oncolytic bacteria, or products intended as gene therapy agents and, as a general matter, are not administered by injection.

To view the guidance click here.

Australia

Presentation by Therapeutic Goods Administration (TGA) - Regulation of autologous cells and tissues