Paediatric Clinical Trials: Update on Legal Requirements

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Barbara Sickmüller

Barbara Sickmüller, Senior Scientific Advisor for the German Pharmaceutical Industry Association speaks to Pharma IQ on the legal requirements for paediatric clinical trials.

Pharma IQ: Have we come a long way with improved monitoring of regulations around paediatric clinical trials?

Barbara Sickmüller: I think with the paediatric regulations there has been a change in culture and in the paediatric development in the EU because, clinical trials that include children, are an integral part of the clinical development for new pharmaceutical products, but I think, the five years recent European Commission Research is too short a time to draw conclusions because the development of a new pharmaceutical product takes about 10 to 12 years.

Pharma IQ: What exciting results are coming out of paediatric clinical trials?

Barbara Sickmüller: Since 2008 more than 18,000 study reports on about 2,200 medicine products have been submitted to the competent authorities as part of the Articles 45 and 46 of the Paediatric Regulation. I think it’s important that these results are incorporated and put in the information of products. Pharmaceutical companies are working on this.

Pharma IQ: What are the newest regulatory developments around paediatric clinical trials that we should be aware of?

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Barbara Sickmüller: The European Commission has just released a very interesting report: “5-year Report to the European Commission - General report on the experience acquired as a result of the application of the Paediatric Regulation,” prepared by the European Medicines Agency with its Paediatric Committee. A second report will be released in another five years. This report gives a lot of information, and I think companies active in this area should take an in-depth look at it.

There is in addition a public consultation on this report; the deadline for this is the end of November. Both papers are available online through this link: http://ec.europa.eu/health/human-use/paediatric-medicines/developments/index_en.htm.

Pharma IQ: Barbara, can you tell us a little bit more about the Standard PIP for Allergen Products for specific Immunotherapy and the challenges around this? This is something that you’re going to be presenting on at the conference.

Barbara Sickmüller: I will be looking at the problems companies have with the “standard PIP” for Allergen Products for Specific Immunotherapy. These products have been on the market for many years, because they had been used under a ‘Named-Patient’ basis. Under the enactment of the German Regulation for Therapy Allergens (Therapieallergene-Verordnung), companies had to submit applications for new marketing authorisations by 1 December 2010 to the German authorities.

Consequently, for all those products that are subject to the Regulation for Therapy Allergens in Germany, EMA decisions on agreed PIPs must be included in the marketing authorisation applications. To our understanding it is important that the experiences from the market are accepted in the regulatory requirements with regard to the PIP.

Pharma IQ: What in your opinion are the current challenges around paediatric clinical trials in general?

Barbara Sickmüller: The main problem that we are seeing in clinical trials in Germany is that parents have to be convinced about the importance for their children to take part in a clinical trial. Another more practical but still important problem is that there are many patchwork families, or families where the couples are divorced but, clinical trials require the allowance from both parents and this is not always possible. However, I think this is improving.

Pharma IQ: Are there regions, globally, where paediatric regulations do really need to be reviewed?

Barbara Sickmüller: One problem we see is getting data on products which are already on the market. Part of the EU paediatric regulation is the “Paediatric Use Marketing Authorisation (PUMA)”. This regulates that companies get ten years regulatory protection for older products, if they are doing research and get authorisation for children. Unfortunately this does not work because it’s not accepted in the reimbursement situation and companies don’t get rewarded enough to really work in this area.

So in the five years since the Paediatric regulation, there’s only one PUMA application which has gone through, but I think it’s necessary to really work on that. With regards to the Allergen products, we have the problem that the regulation was set out with the idea of “carrot and stick”. Companies getting a “stick” to do clinical trials with children and the “carrot” is that they get an extension of the Supplementary Protection Certificate (SPC), but this does not work, for instance, with Allergen products. They are on the market, and they are not patent protected, and so they have to make these clinical trials with children, which is necessary I think.