A Periodical for the Sarcoma Community

Letter to the Editor - October 2011

The Creating Hope Act – Some Perspectives from Europe

Editor’s Note: Some background to the letter - On October 5, 2011, I posted the following note to the various sarcoma support groups on ACOR

"The Creating Hope Act is innovative legislation that will incentivize drug companies to invest in childhood cancer research in return for a voucher that will let them speed other, more profitable drugs to market. It is incentive at no cost to the American taxpayer. More childhood cancer research means better treatments and moving closer to the cure for childhood cancer. 

"Nancy Goodman is the architect of the Creating Hope Act, the founder of Kids v Cancer and, forever mom to Jacob. On September 23, 2011 Congressmen Michael McCaul (R-TX), G.K. Butterfield (D-NC), Sue Myrick (R-NC), and Chris Van Hollen (D-MD) introduced the Creating Hope Act. Senators Bob Casey (D-PA) and Scott Brown (R-MA) introduced a companion bill, S. 606. Congressman McCaul, the founder and co-chairman of the House Childhood Cancer Caucus, said 'The Creating Hope Act offers the best chance of encouraging pharmaceutical companies to develop treatments for children at no cost to taxpayers.'

"Now we must advocate for passage of the Creating Hope Act. The kids need your help! You can help by contacting your representatives seeking their support of the CHA. Please click the link below to easily send a note to your reps. The main note is already written for you, and the reps are pre-selected by your address. You simply fill in your contact information, add a personal note if you like, and hit send. Click here to send your note."

I received a number of responses to this post including some asking if there were similar efforts in the UK. I asked Roger Wilson, President of the Sarcoma Patients EuroNet Association (SPAEN) and Honorary President of Sarcoma UK to reply to this question. His response follows.

Hi Bruce,

What is happening over here is a pan-European initiative.

In essence, virtually all teenage/children's anti-cancer medication was 'off-label' and there were no pharmaceutical company-led trials, just a few academic trials looking at dosing regimens of standard drugs, etc. Some years ago the European Parliament agreed that there should be incentives for pharmaceutical companies to trial new drugs in the children's setting. These incentives include extension of patents by several years (including the patent regarding adult use), reduced bureaucracy in licensing applications, including standards of evidence based on smaller numbers, and reduced bureaucracy in set-up of studies. We have seen some movement, though not a lot. Among the good moves has been a Pfizer study of Sunitinib in paediatric GIST.

Interestingly the greater freedoms are also being exploited by academic studies. Our latest first-line Phase 3 study in soft tissue sarcoma (GEDDIS) is available from teenage (13yrs +) through to elderly (based on performance status). It is recruiting well. We have two soft tissue studies active in children (one for rhabdo and one for non-rhabdo) available at diagnosis, and we have osteosarcoma and Ewings studies active, with new ones coming through shortly. However these do not have significant new agents in them - they are all looking at various dosing/combinations of standard agents, and maybe adding a bisphosphonate.

The failure of manufacturers to take forward the IGF1-R drugs, which had a good response in Ewing’s sarcoma a couple of years ago, is most depressing. The arrival of mifamurtide in osteosarcoma is controversial scientifically, even though the drug is approved in Europe and funded in some countries (including the UK). The indication is however limited, and we will be getting a new study co-funded by the drug company and the European Commission to look at extending the indication to metastatic disease.

We can predict further issues of a not dissimilar kind with new agents. Cediranib for ASPS is in worldwide trials at the moment.  Many of these patients are young adults. However even if these studies show benefit (and they are doing so), the manufacturer will not seek licensing for this disease unless the drug gets a licence for something more common. So at best it will be off-label use, and worst it will get withdrawn.  However Denosumab, which is in a study for giant cell tumour of bone (with 100% response rate), is already licensed for osteoporosis so there will be no problems over getting hold of it if the study is positive. Again the patients are mostly young adults.

While these last two are not paediatric, I think we can predict that the disadvantage that childhood cancer has faced is extending itself to adult rare cancers. So any initiative to open things up and incentivise pharmaceutical companies to take small groups seriously is welcome.