Base camp

A cold snap in the November of 2011 and poor plumbing in a Canary Wharf hotel conspired to make a frosty affair of the first of a series of European meetings on drugs development for children with cancer. The suspicion that characterised this first attempt to bring together doctors, drug companies, regulators and families of patients thawed somewhat at a warmer venue in Paris in 2013, which was an altogether more collaborative affair. But if Paris was about cooperation, the meeting in Vienna last week of the newly-named Cancer Drugs Development Forum was, if anything, about the need for action.

The message from more or less all of the parents’ representatives was that if changes were needed to the EU PaediatricMedicines Regulation, then now was the time to get started on pushing them through. This view came from other sides of the table too. Christina Bucci of the drug company, Novartis, argued that we could not wait another year for reform, while clinician, Peter Adamson, of the US Children’s Oncology Group put it succinctly, in what was to become a mantra for the meeting: waiting is not an option.

In the closing session of the gathering, it was therefore agreed to set up an action group to frame necessary reforms to the PMR, based on conclusions that had been reached in both Paris and Vienna.  The group will have its work cut out, though, as it has become clear that this is not simply a matter of narrowing the opportunities drug companies currently have to waive their obligations under the Regulation to develop Paediatric Investigation Plans (PIPs).

As Pam Kearns of the Birmingham Clinical Trials Unit pointed out, the unnecessary red tape associated with the Regulation can not only act as a disincentive to research for children but for adults also. Comparable drugs are often developed by different companies and separate PIPs for each, perhaps competing for recruits from a tiny patient population, make no sense for business, researchers or patients.  Paediatric plans also need to begin earlier in the development process, avoiding the delays companies may make until they are sure an adult product will go to market. Moreover, there needs to be a more flexible definition of what a PIP is. If an adult illness such as melanoma also affects a small number of adolescents then lowering the age of entry makes more sense than creating an additional and unviable trial.

These are just a few examples of how the Regulation could be made more ‘intelligent.’ Sensible amendments like these, however, need to be placed in a wider context. The Regulation does not only relate to cancer, after all, and any changes would need to take into account the knock-on effect with other childhood illnesses. Beyond this, how would a reformed PMR relate to other legislation, such as that for ‘orphan drugs,’ or indeed to the new initiatives discussed in Vienna for creating further incentives for pediatric research?

If the work ahead seems daunting, the message of Vienna was that important inroads have now been made. In the world of health, there is much that simply cannot be legislated for, an obvious example being that of willing cooperation between the many different parties involved. Maoxia Zheng of Genentech / Roche set out how this corporation had developed ‘matrix’ studies in which drugs from different companies have been combined to develop potentially more effective therapies. Pam Kearns countered that an independent ‘broker’ was more appropriate for bringing private companies together and outlined how ITCC itself had created such ‘matrices.’ Either which way, what was demonstrated was that partnerships between commercial competitors are possible and, given the complexity of cell biology and the urgent need for progress, of vital importance.

Regulators, too, demonstrated a spirit for innovation. The Paediatric Committee of the European Medicines Agency are reviewing waivers of PIPs made under the current framework, and their report due next month may well revoke some of them.

I may still not have come down to earth from flight BA0707 from Vienna, but I wonder if when the action group makes its push for change, it may be at an open door. 

We hope to report back at the Childhood Cancer International meeting in Malmo in May and hopefully this will be an opportunity for wider parent / patient input. In the meantime, do please make comments at the foot of this blog.