The Elephant in the Partnering for Cures 2012 Room

Last week I attended the Partnering for Cures 2012 meeting in New York. I sat through four roundtable type discussions in which multiple stakeholders made up each panel. e.g. there were multiple NIH participants including Dr. Francis Collins, Dr. Janet Woodcock from the FDA, Big pharma and smaller pharma executives, academics, not for profit CEOs etc..The discussion topics were interesting, however apart from a few topics I sensed a real lack of urgency. The meeting also introduced me to many more parent or patient advocates specifically those focussed on ultra rare diseases (just think a handful of patients in a country like the USA – to low thousands). Several of these patients/ parents had a small discussion towards the end of the meeting (perhaps next year this could be more organized to share experiences?). It was pretty clear they were the minority group of attendees at the meeting and their diseases were predminantly dwarfed by the likes of Cystic Fibrosis, cancers etc that seemed to get most if not all of the attention.

I was also recently made aware of some testimony on Capitol Hill from Roy Zeighami. I have spoken to Roy a few times but I was not aware of the magnitude of the situation he was facing until watching this video. In it Roy describes how a pharmaceutical company can develop a molecule in the USA but pursue clinical trials in Europe and at the same time exclude patients from the USA in participating in the trial. Roy and his employer Cisco have gone to enormous lengths to try to enroll his son Reid who has Sanfilippo Syndrome type A, in the trial with no success. Interestingly this type of situation was not raised at the meeting last week. Also, I am not aware that anyone even brought this kind of issue up.

Why do I think this is important…Lets just imagine hypothetically a best case example. The NIH funds researchers in the USA to discover a cure for an ultra rare disease. The preclinical work is successful and the company (assuming a company discovered or licensed it) now wants to pursue clinical trials. They want the fastest route to market. Perhaps they find the FDA to be too demanding and opt to have the trials in Europe in which perhaps it is more preferable from an ease of regulatory rules etc. The molecule undergoes clinical trials in Europe but may exclude patients in the USA. The bitter irony of this of course is that tax dollars went in to the discovery of the molecule and that the patients that benefit in Europe did not have to pay for it. If the disease is severe, by the time the molecule ultimately (hopefully) makes its way back to the USA, patients will have progressed in their disease and it may be too late. This type of situtation is worthy of much more visibility and should have been discussed. If it is widely known to be taking place, then possibly this was the elephant in the Partnering for Cures 2012 room.


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  1. Roy Zeigham says:


    Flattered that the talk had an impact on you. Hope you thought it was delivered well.


    1. sean says:


      I think anyone watching your testimony would be affected by it emotionally but also by how incredibly brave you are to have stood up for your son and done it so eliquently. I had no idea what you were up against – You should be on platforms like this Partnering for Cures meeting alongside the NIH and FDA, because it does not matter what they say about any changes they lead, they cannot happen fast enough.


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