Marcus Evans - Conference Report
Medicines for children London, Jan 14-15, 2008
After an interesting opening by the chair Frances Crawley, we moved the first main section of the conference entitled “Overcoming the challenges of specific indications”. This section was opened by Dr. Paul Wang whose presentation gave a clear and concise overview to the challenges of all the aspects of running a paediatric trial; from the efficacy and safety considerations that need to be thought about as part of the initial planning stages and into the actual running of the study. Dr. Suyash Prasad took the latter half of this section and presented his ‘learning points’ as a result of a trial in the ultra orphan area of Pompe disease. He also showed two videos showing the before and after enzyme replacement therapy which showed how much impact a trial can have on the lives of people. Babies who were expected to have a on to two year life span are now living relatively normal lives into later childhood and perhaps beyond.
This section covered the pharmacokinetic aspects of children in trials- this I think lead to two conflicting views on … is a child a small adult or just a small child?
One view was that any child aged above 2 was in fact a small adult and the other was a child was either a neonate, infant, child or adolescent depending on age and more importantly weight…once they are above 50Kg, then they can be considered an adult. The PK section was presented by Dr. David McIntosh and Dr. Willi Weber.
The third section of the day was entitled ‘Addressing the challenges of recruitment and retention’. This section was opened up by Lisa Shafe presenting on the challenges of assent requirements across Europe and the role of the child vs the parent. along with the age considerations and levels of understanding that need to be considered. This section was closed by Dr. Paul Wang and Dr. Suyash Prasad presenting on ‘Strategies for overcoming recruitment and retention challenges’ this lead into the panel discussion. The discussion is generally focussed on …if you have a good, clear, concise and workable protocol; chose your sites well, and train the site teams then you should have no problems!
Day 2 started with a complete breakdown of all the components of the PIP; the paediatric investigation plan, by Dr Birke Lehmann. She also discussed the applicable timelines and the process for waivers, deferrals and appeals. Just in case you were wandering 3785 CTAs were presented between 1 August 2004 and 1 October 2007, of which 259 presented trials in children. The remainder requested waivers or deferrals.
The post presentation discussion pointed to the fact that the PIP is very restrictive with any changes to protocols and programmes by ECs and CAs need to go back to the PDCO as part of a modified PIP. It may be better in the future that the PIP is more of a concept document in that going into the specific of individual trial. Dr. Birke Lehmann pointed out that we are all in the learning stages at the moment and there may be changes in the future but this cannot be confirmed.
The PIP section was closed with Dr. Penelope Ward presenting a view from industry. She felt as do most people in industry that the stage at which the PIP is requested, is much too early. Most companies will not have completed the full reproductive toxicity programme prior to completion of phase 1 studies, the current point of submission of the PIP.
The second half of the morning brought some insight into designing effective protocols for paediatric studies by Dr. Philippe Auby. This looked at the ethical, scientific and clinical aspects as well as the consideration that paediatricians are used to using drugs off label in children, utilising a certain amount of guess work. The last session of the morning was on pharmacovigilance and best practice in paediatric trials, stressing that the requirements are not going to be very easy to follow.
In the afternoon Prof Terence Stephenson brought us a presentation on the EU legislation and UKCRN; along with the opportunities it will bring both for now and in the future. This was followed by an informative presentation from Jane Lamprill on ensuring high ethical standards in paediatric trials, something we all I am sure strive for.
Upcoming Conferences
The group have given details of forthcoming conferences that they are aware of:
Paediatric Drug Development and Clinical Trials 30 September - 1 October, please click here for Agenda.
EFGCP 08 - paediatric oncology - please click here for the programme
Meeting on Training in Childrens Research Ethics - 10 December 2008
Paediatric Regulation Seminar and MHRA Paediatric Survey 2008
http://www.mhra.gov.uk/ConferencesLearningCentre/Conferences/CON026011
Please see the link to the forthcoming EMEA workshop.
http://www.emea.europa.eu/htms/human/paediatrics/workshops.htm
