Regulatory News - December 2021

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


Medicines and Healthcare products Regulatory Agency (MHRA)

Use of Real-World Data in Clinical Research

The MHRA have released guidance on the use of real-world data (RWD) in clinical studies to support regulatory decisions. The guidance provides information for sponsors proposing to conduct clinical research using RWD to aid the design of studies, including points to consider when evaluating sources of RWD and assessing the quality of a source for its intended use.

Sponsors interested in using RWD in their development programmes are encouraged to engage with the MHRA for further advice.

For further information and to read the MHRA guidance, see here.

Updated Brexit Guidance for Businesses

The MHRA have updated the Brexit guidance on their website for businesses to capture changes to post-Brexit protocols. Updated sections include:

See the links above for the latest updates.


European Commission (EC)

EC Proposal to Ensure Supply of Medicine Between UK and EU

The Commission has released information on how it plans to prevent Brexit from disrupting the supply of medicines in certain markets. The plan builds on the Protocol on Ireland/Northern Ireland, which was proposed in October 2021 as part of negotiations with the UK. The proposals ensure continued medicine supply from Great Britain to Northern Ireland, and address outstanding supply concerns in Cyprus, Ireland and Malta.

In the context of the Protocol on Ireland/Northern Ireland, this means that the same medicines will continue to be available in Northern Ireland at the same time as in the rest of the United Kingdom, while specific conditions ensure that UK-authorised medicines do not enter the Single Market.

See here for the full press release.

European Medicines Agency (EMA)

EMA Data Standardisation Strategy

The EMA Management Board adopted a set of principles for data standardisation and have released the ‘European Medicines Regulatory Network Data Standardisation Strategy’. The strategy sets out recommendations for the development and adoption of standards across four domains:

  • Medicinal products
  • Healthcare and study data
  • Safety and risk management
  • Submissions

Historically, regulatory procedures have been mainly documentation submission based with assessment of information contained within documents, rather than assessing the underlying data used to create those documents. The need for standardisation has become more apparent with regulatory processes shifting to assessing data rather than documents and the potential for secondary uses of data collected to drive better regulatory decision making.

The EMRN Data Standardisation Strategy is available here.


Food and Drug Administration (FDA)

FDA Guidance on ‘N of 1’ Gene Therapies

The FDA has released two draft documents aimed at sponsors of individualised investigational antisense oligonucleotide (ASO) therapies for ultra-rare diseases. The draft guidance provides clinical, and chemistry, manufacture and control (CMC) recommendations for investigational new drug application (IND) submissions to support initial and continued administration, dosing, and clinical monitoring of these therapies for people with severely debilitating or life-threatening diseases.

The guidance for clinical investigators and sponsors provides clinical considerations for a range of topics including the following:

  • Ethical and human subject protection
  • Diagnostic and genetic considerations
  • Dosing considerations
  • Drug product administration procedures
  • Safety assessment
  • Assessment of clinical response

The CMC guidance provides recommendations regarding the CMC information that should be provided in an IND application submitted by a sponsor, topics include:

  • Regulatory considerations
  • Drug substance description, including physical and chemical characteristics
  • Method of preparation of drug substance
  • Characterisation, control and stability of drug substance
  • Components of the drug product and quantitative composition
  • Description of manufacturing and packaging procedures for the product
  • Control and stability of drug product
  • Container closure system
  • Immediate packaging and labelling
  • Environmental exclusion

To read the clinical guidance see here. To read the CMC guidance, see here.

Public Consultations

No relevant public consultations.