Applied Clinical Trials
The right-to-try gale is now blowing at full strength in the US and is likely to prove a straw in the wind that could become a haystack.
“We have to do something, we have to do it quickly,” said the UK's top health minister, Jeremy Hunt, just hours after the government reversed its approach to the use of cannabis oil in the treatment of epilepsy. "It does take time, because we’ve got to not only look at the law, we’ve got to look at the clinical evidence and make sure there are no unintended consequences", he went on.
Hunt was speaking in mid-June in the midst of a storm of public outrage over the UK's denial of treatment to a boy afflicted with repetitive seizures and whose supplies of cannabis oil-which reportedly eases the frequency and intensity of his attacks-were confiscated days before by government authorities.
Bad cases make bad law, as the axiom goes. But this case-indubitably bad for the boy and his family-has thrown the spotlight even more sharply onto questions of drug authorization. The outcry of sympathy that it has provoked inevitably focuses public reflection on the rules that governments use to permit or prohibit therapy, in the name of protecting the public. That reflection is unlikely to be guided by the most informed voices, and may struggle with the inevitable complexity of the underlying issues. But it would be foolish for any in the health community-in the UK or more widely in Europe-to dismiss it entirely.
This case relates, of course, only to one treatment and one country-cannabis oil is available legally in some other EU countries, as well as in Canada and the US. But when the right-to-try gale is now blowing at full strength in the US, it is likely to prove a straw in the wind that could become a haystack. In the UK alone, a third of the 63,000 children with epilepsy do not respond to the prescribed medication and resort to additional or substitute therapies. What will it mean for European regulators if more cases of this sort hit the headlines? Dan Poulter, a former UK health minister who has been backing the boy and his family, downplayed the challenges: in his view, all that would be needed is "a simple tweak to the law".
But Pandora's box was opened by only "a simple tweak", too. And in the US, where right-to-try is now the law of the land, companies are already preparing for a flood of patient requests for experimental therapies. Among the numerous problems identified already by firms, some are very practical. One is that they simply may not have enough volume to respond to requests-which threatens extravagant disappointment in the face of the increased expectations. Another is the amount of time and resource that will be required by companies as they cope with patient requests. And a third is who will pay for products provided.
But a wider issue will arise, relating more to regulators than to companies. In making their decisions to authorize or not life-saving treatments, regulators are bound to become increasingly sensitive to public assumptions that a chance is better than no chance. Because their very existence and power ultimately depends on public trust, they will have to become more adroit both in striking the balance between protection and permission, and in explaining what they do to volatile public opinion.
Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium