Monday, 7 March 2016

Guideline on the clinical development of medicinal products for the treatment of Autism Spectrum Disorder

Most readers of this blog are in North America and I think this will be by far the largest market for any new drugs approved for autism.

An even bigger market by population (508 million vs 354 million) is the European Union, where the drug regulator is now developing guidance for those developing new treatments for autism.  They are asking for comments.

The only people really qualified to give comments are those with some experience of treating autism, very few of whom live in Europe.

Regardless of where you live, I would suggest that the doctors and researchers who read this blog take a look at the short guideline document and pass on any comments they may have to the European Medicines Agency.  

For everyone else, I do not suppose they expect to get comments from lay people, but why not go ahead and surprise them?

The obvious comment would be to hurry up, but there are many more constructive comments that can be made. 

The Press Release:

The Draft Guidance Document:- 


  1. Thanks for this Peter, it is definitely worth writing to them - while some of the suggestions are decent some are very, VERY bad and will make the terrible situation and lack of treatment options even worse.

    The authors of the draft proposal obviously have their hands up in the clouds of the La-la Land of when they say that "It is important to demonstrate that the effect of the medicinal product is specific for ASD and is not (164) due to secondary therapeutic effects on psychiatric co-morbid conditions..."

    As every parent knows if ask your GP or (or indeed a specialist consultant) to prescribe something for your child’s say aggression or extreme anxiety or irritability you will be turned down on the basis of the meds you ask for 'not being proven to work for aggression SPECIFIC to autism'. This happened to us many times, this “sorry Sir, we doctors cannot extrapolate to autism”. Diagnosis of autism is the magic word that shuts the door to every and any treatment right now, and this guideline will shut the door to the only solution, which is to actually research things specific to autism. Ironic, isn’t it?

    The other big negative is the authors’ obsession with addressing the Core Symptoms of Autism – while they are being somewhat reasonable and state that “Due to the heterogeneity of the disease it may not be possible to achieve a significant effect on all core symptoms with a single compound.”(no kidding!) they still go on to conclude “Therefore short term efficacy has to be demonstrated on at least one core symptom, supported by a positive effect on global function.” BUT it should be the other way around – we should be looking to improve global functioning first and foremost, with improvements in core symptoms being a very welcome secondary outcome.

    Rather than being obsessed with correcting the core symptoms I much prefer your suggestion Peter of looking at and addressing cognitive dysfunction in autism as a PRIMARY ENDPOINT – it would be easier to measure and likely much easier to tackle, but most importantly cognitive (dys)ability in autism is much more relevant and predictive of quality of life and future independent living (read economic cost of autism to society) than the core symptoms of autism.

  2. Autism is not a homogeneous disorder.There are those,like my sister,and others diagnosed with so-called high functioning autism,who have no obvious cognitive dysfunction,in terms of intellectual or learning disability,but have profound psychiatric disability as well as autism.In my sister's case,it is probably folate related,given my own diagnoses,and what I have read about CFD/FRAs in schizophrenia,and other psychiatric disorders.

    It really is a shame Vincent Ramaekers was not called in to be an adviser to these guidelines.

    It is obvious,to me,this that these guidelines are mostly talking about nonsyndromic or isolated autism.I do wonder just how much of the autism population have this type of autism.It has only been relatively recently that American researchers have begun to accept that autism is a heterogeneous condition,with different presentations and causes.With a handful of exceptions,such as Dr. Ramaekers,Europe has been even slower in accepting the idea that what we call the "core symptoms of autism" can often be only one feature of any number of complex syndromes.Syndromes that can be due to immune or metabolic dysfunctions,rare or novel genetic mutations,or chromosomal deletions/duplications,or any combination of these.A serious researcher or clinician needs to do extensive testing on each patient,to learn if there are underlying medical and genetic conditions,that might be causing these "core features".Antipsychotics can be very dangerous,if not life-threatening,for people with certain metabolic,or genetic disorders.


Post a comment