EMA and FDA aim to alleviate burden of paediatric Gaucher trials

The challenges of designing and conducting clinical trials in children suffering from LSD’s, and the attendant burden on their families, are well known.

The EMA and the FDA have recently jointly published a strategy document on drug development research in paediatric Gaucher disease. The key recommendations are:-

  1. That specific paediatric trials no longer be carried out. Instead, data from adult trials and paediatric populations can be modelled to be applied to paediatric patients.
  2. Safety and efficacy be studied in the same trial.
  3. There may be a case for conducting multi-arm, multi-company trials.

These are important and welcome recommendations which, if formalised, will achieve two important outcomes:-

  1. Reducing the need for affected children to participate in clinical trials (and the attendant burden on their families).
  2. Considerably reducing the time and cost involved.

Although the recommendations apply to Gaucher disease, one can see no reason why it should not be possible to include other LSD’s in due course.

Here is the link to the EMA website from which the document can be downloaded.

Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration


Genomic testing; UK’s chief medical officer calls for making it as common as blood tests

The Chief Medical Officer to Her Majesty’s Government, Dame Sally Davies, has today published her annual report. In it, she has outlined the importance of genomic testing, and recommended that it become part of routine NHS care.

Make DNA tests routine, says UK’s chief medical officer 

Coming from the country’s senior most advisor on health, this is an important statement, and acknowledges the importance of the genomic revolution. It is likely to have a significant impact on the management of LSD, for example by facilitating newborn screening; early diagnosis will undoubtedly improve the prognosis for many LSD’s that are currently treatable, but where the diagnosis is often made at an advanced stage, when such treatment is likely to be less effective.

It may also open up new and exciting avenues of research. For example, patients with LSD’s that affect the brain are often diagnosed too late to benefit from clinical trials of newer treatments. By providing earlier diagnosis, patients who previously might have been excluded from such trials may now stand a good chance. It will also surely be an incentive to the industry to develop clinical trials, knowing that there may well be larger numbers of suitable patients.

There are tight regulatory processes in place that ensure good research governance; the Medicines & Healthcare products Regulatory Agency, research ethics committees, and so on.

Of course, there are wider implications for society as a whole. There will also undoubtedly be financial implications for an already cash-strapped NHS. New treatments for LSD’s are invariably expensive. A discussion of these issues, while important, is outside the scope of this post.

At the very least, however, by placing this important item high on the government agenda, and thereby raising public awareness, the CMO has encouraged healthy public debate.  She has done the right thing.