Quite a coup this morning for a group of 8 sufferers of a rare disease to get a plea for Pharmac to fund a new drug in to both the Herald and the Press.  I’ve since discovered that Radio NZ and TV3 have picked up the story.  There are also some slick videos on vimeo.com

These people are evidently very ill with a debilitating and very rare disease (less than a hand-full of cases per million).  Paroxysmal Nocturnal Hemoglobinuria (PNH) does nasty things to one’s red blood cells. Blood clots which may result in death are a serious complication of the disease.

The campaign, backed by one haemotologist, is directly aimed at getting Pharmac to fund a drug.  The drug is called eculizumab and is produced by the Pharmaceutical company Alexion under the name Solaris.  Apparently the cost is $500,000 per year per patient every year for life.  This is information in each of the very similar media articles.  Apparently Pharmac thought the evidence limited last year, but sought the opinion of a specialist heamotology committee who are due to report back to Pharmac soon.

The Herald (only!) reports

“A support group for patients with PNH said Pharmac was taking too long to decide, has begun a lobbying campaign, whose costs are met in part by Alexion.”

Alexion are engaging in a very common practice to promote their own drug by engaging the media through support groups.  That is not to say their drug is somehow “bad”, merely that they see it reasonable to use the media to raise sympathy in an attempt to put pressure on an independent committee which is supposed to be making decisions entirely based on the scientific evidence and a cost-benefit analysis.  I don’t believe they will be swayed by this sort of campaign – and nor should they.  It is not in the patients’ interests for them to be so.  Having said that we have seen before in New Zealand how such a campaign can influence the Pharmac process and that was with Herceptin prior to the 2008 elections when the National party made policy promises*.  I do hope there won’t be a politician who speaks out in support of this current campaign, but I don’t hold my breath.

Having bagged Alexion’s tactics I must also praise them.  Not many drug companies seem to bother with rare diseases.  The risks of not getting a return are greater than for common diseases.  Good on them for developing this.  With small numbers it is difficult for quality efficacy or safety trials to be done, therefore difficult for the evidence to be gathered.

Pharmac need to assess the evidence.  I had a look on PubMed for trials of PNH with eculizumab.   There is precious little. The FDA summarised the evidence in a freely available publication: http://theoncologist.alphamedpress.org/content/13/9/993.long in which they discuss one and one only randomised controlled trial of just 87 patients (with a positive result) along with some other “open label” studies. I could find no trials that provide evidence of extension of life (not unusual). What is often the case is that a surrogate marker (eg measurement of some substance in the blood) which is thought to relate to the risk of death is measured instead and a change in risk of death is then inferred from this. Not the best kind of evidence, but often all there is. The studies all seem to say something positive.

The difficulty Pharmac has to deal with is that nearly all the trials are sponsored by Alexion and the principal authors receive consultancy fees etc from Alexion.  This does not automatically mean the trials are biased, but it does mean that Pharmac should proceed very cautiously given the history of  Pharmaceutical companies (and I am not pointing at Alexion here) tendency to publish only the positive results.

A very tricky decision for Pharmac- I don’t envy the decision makers.  A very difficult disease to live with for the patients – I wish them all the best.

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* Disclaimer – I was a candidate for another Party in that election.  When I told a soon to be National MP in a debate that decisions on drugs should not be made by Politicians he turned around and accused me of wanting to kill women!  For the record, I don’t, and I did lose my rag.  Ho hum.

Tagged: Alexion, evidence, health, media, Paroxysmal Nocturnal Hemoglobinuria, Pharmac, pharmaceutical companies, PNH, Solaris