European Regulatory Roundup: UK Plans Off-Label, Experimental Drug Database (4 February 2016)

Regulatory NewsRegulatory News
| 04 February 2016 | By Nick Paul Taylor 

Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.

UK Government Backs Plan to Create Database of Off-Label and Experimental Treatments

The United Kingdom government has thrown its weight behind legislation that would result in the creation of a database of innovative treatments being carried out by doctors in England, including both off-label uses of existing drugs and tests of unlicensed, experimental therapies.

Officials are proposing to task the Health and Social Care Information Centre with the construction and maintenance of a database that would give doctors access to details of experimental therapies, as well as the outcomes generated using these novel approaches. The idea is to broaden access to knowledge about experimental treatments, whether they be off-label uses of existing drugs or new, as-yet-unlicensed therapies. Predecessors of the bill, notably a proposal by Lord Saatchi dubbed a “quack’s charter,” were controversial, but the government is willing to back the latest proposal.

George Freeman, the UK minister for life sciences, has committed government resources, as well as his support, to the legislation, which was first proposed by politicians from outside of the upper echelons of government. “I felt their objectives were so supportable that I have been happy to put officials in the department to work to help come up with amendments to produce a bill that the government could support,” Freeman told The Guardian. In doing so, the odds of the bill becoming law have improved, although it could still face opposition in the House of Lords.

The involvement of the government has coincided with the removal from the bill of some of the more contentious proposals. When first introduced, the text stated: “It is not negligent for a doctor to depart from the existing range of accepted medical treatments for a condition if the decision to do so is taken responsibly.” The line, like others in Saatchi’s bill, raised concerns that the legislation would serve to protect negligent doctors from punishment. Now, the diluted text is less likely to trigger vehement opposition, although concerns about its effect on health research and care remain.

In drafting a bill that has a chance of becoming law, those involved with its creation and amendment have left out sections that would have reshaped aspects of the regulatory landscape. The latest round of proposed amendments included a section to legislate for the establishment of a pathway for approving the routine use of off-patent drugs in new indications. Another section proposed putting the National Institute for Health and Care Excellence (NICE) in charge of assessing the evidence to support off-label uses. Neither proposed amendment is in the latest version of the text.

Latest Legislation,Proposed Amendments, The Guardian

EMA Makes Recommendations for the Labelling of Pandemic Vaccines

The European Medicines Agency (EMA) has posted recommendations for the labeling of pandemic vaccines. EMA has based its advice, which is designed to ensure labels provide current information without slowing access to vaccines, on the experience it gained during the 2009 pandemic outbreak.

In 2009, the fast-changing nature and on-going scientific evaluation that characterized the response to the outbreak rendered the information provided on vaccine labels obsolete quickly. To counter this problem, EMA is recommending manufacturers of pandemic vaccines put a notice at the start of their package leaflets directing readers to up-to-date sources of information, specifically by linking to EMA’s website. In addition, EMA wants manufacturers to “optimize the time to implement in the printed material the authorized changes made by the competent authorities.”

The implementation of changes was an issue in 2009. In stockpiling supplies of pandemic vaccines, manufacturers also produced labels ahead of their submission to EMA. This resulted in companies being unable to fully address changes proposed by the regulator. As such, EMA now wants firms to supply mock-ups for review before they start commercial-scale printing. EMA would aim to review the mock-ups quickly, positioning manufacturers to ship pandemic vaccines with labels that met its requirements without delaying the supply of products.

Access to clear, accurate and up-to-date information is particularly important given the potential for confusion during a pandemic situation. In 2009, EMA found the outer labeling used by companies increased the odds of medication errors, specifically because it gave insufficient prominence to the administration instructions. Other medication errors stemmed from the illegibility of vial labels that featured lots of very small text, sometimes in multiple languages. EMA lays out how companies can avoid these problems in a future pandemic in its review document.

EMA Recommendations

UK Clears Researchers to use CRISPR to Edit the Genes of Human Embryos

The Human Fertilisation and Embryology Authority (HFEA) has cleared a research team in the UK to edit the genes of human embryos. Having secured the approval, a team at the Francis Crick Institute will use CRISPR–Cas9 to investigate the role genes play in the first days following fertilization.

On one level, the approval is a relatively small event. The research team now has clearance to run tests to see what happens when certain genes are switched on or off during the first seven days of the development of an embryo, work that could lead to improved treatments of infertility. However, because of the opportunities presented by the application of gene editing to humans — and the potential for regulators to stymie them — the approval of the project is a notable event.

"It’s an important first,” George Daley, a stem-cell biologist at Boston Children's Hospital told Nature News. “The HFEA has been a very thoughtful, deliberative body that has provided rational oversight of sensitive research areas, and this establishes a strong precedent for allowing this type of research to go forward.” Now, attention will shift to how gene editing will be handled by regulators outside the UK, which is known for its liberal approach to fertility science.

Press Release, Nature News, NHS Choices

CVMP Releases a Clutch of Veterinary Drug Development Texts for Consultation

The Committee for Medicinal Products for Veterinary Use (CVMP) has released five draft documents for consultation. Across the texts, CVMP lays out its current thinking on multiple topics related to the development of veterinary medicines for minor use or minor species (MUMS).

Each of the four draft guidance documents relates to MUMS, the veterinary equivalent of an orphan designation. The texts cover CVMP’s proposed stance on various types of data, including efficacy and target animal safety, quality, immunological and residue. CVMP last addressed each topic from 2006 to 2007, since when the regulatory committee has gained understanding of how best to handle the data requirements for MUMS products.

“These MUMS guidelines have been reviewed and revised with the aim of updating the acceptable data requirements in light of experience gained and clarifying, where appropriate, the applicability of the MUMS data requirements,” CVMP wrote in the draft. Each of the four draft guidelines are open for comment until 31 July.

CVMP has given the industry a narrower time window in which to comment on its fifth new text, a draft concept paper on the revision of the guideline covering the conduct of pharmacokinetic studies in target animal species. The concept paper, which is the start of the process of bringing guidance in line with the rise in population pharmacokinetic studies and pharmacokinetic/pharmacodynamic (PK/PD) modelling in veterinary drug development, is open for comments until 30 April.

Efficacy, Immunological, Quality, Safety, Pharmacokinetic


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