Monthly Archives: January 2013

A plea to fund a drug

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

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: 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.


* 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.

Lives to be saved on March 14th 2013

Kidney’s are being attacked every day.  Yours could be next.  So common and deadly are kidney attacks that the theme for this year’s World Kidney Day is “Kidneys for Life: Stop Kidney Attack!


Kidney Attack, or as Physicians and scientists call it “Acute Kidney Injury,” is a syndrome which affects several thousand people a year here in New Zealand.  It is notoriously difficult to detect and can be deadly.  For more than 5 years now I have been researching how better to detect, and ultimately to treat, Kidney Attack.  Over the past 12 months I have posted several times about this – here are links to just a few of the previous posts:

There will be more as we lead up to World Kidney Day 2013.

Time to get rid of drug advertising

New Zealand is one of the few countries in the world where pharmaceutical companies are allowed to advertise to consumers directly.  I believe this is detrimental to our health and I call on the government to ban the practice immediately.

Drugs are not like any other consumer product. There is one reason and one reason only why you should be prescribed a particular drug and that is because the scientific evidence states this is the “best bet” for you.  I freely admit that there are often issues with the scientific evidence, this is partly because of pharmaceutical companies obfuscation of the evidence.  It is also because of the lack of money put into independent clinical trials by the governments who are meant to be promoting the best health care.  That aside, the social contract with the medical system we have is that the tools, training, information and lists of drugs provided to the doctors is based on the best available evidence.  The drug companies spend an awful lot of money trying to persuade doctors that their drug is best (something I also don’t think they should be allowed to do, but that is for another post), why then do they also spend money directly marketing the drug to you.  It’s simple really, they want you to go to your doctor and bring up the name of your drug.  They know that this will end up with doctors, on average, prescribing more of their drug.  Doctors deny they are influenced by advertising or “consumer demand” but that is a load of hogwash.  Drug companies don’t spend such big bucks without expectation of bigger returns.  There are also trials which show that a patient mentioning a particular drug are more likely to be prescribed that drug. *

Before you dismiss this saying that you never mention a drug to a doctor, let me remind you that your taxes are paying for these drugs and that these drugs are much more expensive because of the advertising spend of the manufacturers.  So even if it is only your neighbour who is reading the adds and getting the prescriptions, you are paying for it.

The issue is greater than the money.  If the drug company with the best advertising campaign manages to have more prescriptions than their competitors in the same class of drug then they win.  However, it is quite possible that you and I lose.  If it is really unknown which drug is best then we lose, perhaps more of us die, if one of the other competitor’s drug is better than the one with the best advertising campaign.  What is often missing is the comparative trial evidence from trials conducted by scientists with no ties to the pharmaceutical companies.

* Kravitz RL, Epstein RM, Feldman MD, et al. Influence of Patients’ Requests for Direct-to-Consumer Advertised Antidepressants: A Randomized Controlled Trial. JAMA. 2005;293(16):1995-2002. doi:10.1001/jama.293.16.1995.

Ben Goldacre in his book “Bad Pharma” describes this trial nicely.  I think that book should be compulsory reading for all new medical students.


Just to let you know how slick things are in New Zealand I thought I’d take you through a case study.  On the advertising part of my Facebook page came up:

Facebook Advertisement

Facebook Advertisement

The first thing to note is that cholesterol is NOT A DISEASE.  It does not, should not, and cannot be treated. Sorry for shouting, but this is maddening  – too often we are being fooled into thinking that some treatment is great for our health simply because it changes the levels of something in our blood (normally) that is merely associated with a possibly bad outcome.  What really matters is that the drug lowers the incidence of the outcome without unduly increasing the incidence of other outcomes, and that the outcome in question really matters.

Anyway, I clicked on the advert and it took me to this site:

Drug company advertising site

Drug company advertising site

Clicking on any links took me to “” pages which were all about the drug Crestor.  They were pages directly aimed at getting people to go to the doctor and ask for Crestor.  This includes a “CRESTOR CHALLANGE – Try CRESTOR for Two Weeks Free”  ( ). They talk of a maximum monthly recommended retail price of $36 and provide a table in which they “Compare CRESTOR with the cost of other health products” – these being Omega 3 Fish Oils, Resveratrol, and Co-Enzyme Q10.  Other than the obvious problem with “cherry picking” some products that are more expensive to compare with, this gives the impression that CRESTOR is simply another health supplement.  It is NOTHING OF THE SORT (there I go shouting again). Crestor is a prescription medicine that contains a statin – rosuvastatin.  This is stated in the small print on the bottom of the page (and elsewhere on the site).  It is unfunded by the NZ government, hence the cost to the consumer.  However, it requires a doctor’s prescription, hence the “Talk to your doctor to see if Crestor is suitable for you” message.

Pharmac, New Zealand’s drug buying agency, have chosen not to fund Rosuvastatin.  They fund three other statins which give the doctors plenty of choices.  My guess is that the reason they fund these three is that they are all “off-patent” which makes them considerably cheaper than on-patent statins (Rosuvastatin is one). Also, they will not have evidence that Rosuvastatin offers a health benefit over and above the other statins. That is not to say that it is not appropriate for a doctor to prescribe Rosuvastatin in certain circumstances.  They may do so when, for example, a patient has had an adverse reaction to other statins.

Finally, in the buyer-beware category, I checked out the papers the website referenced in relation to their product.  They rely primarily on three papers (and use an adapted graph from #2), namely:

1. Ridker PM et al NEJM 2008;359(21):2195-2207

This was a large study comparing Rosvustatin with a placebo. It was sponsored by the manufacturer (AstraZeneca) and the principal authors received grant support, lecture and consulting fees from AstraZeneca.  The participants were healthy men older than 50 and women older than 60 with an LDL cholesterol of <130.  That is a low level.  The purpose of the trial was to see if Rosvustatin could prevent cardiac events in a normal cholesterol population. The trial reported a 44% reduction in risk in cardiovascular events in the Rosvustatin group.  Sounds great.  This is the problem with Relative Risk reduction when the incidence of the outcome is very small.  The results showed 109 fewer events out of 8901 participants.  In other words 82 people would have to be taken the drug for there to be 1 less event.  In the meantime, all 82 are exposed to potential side effects.  For example in the trial if 164 took drug, there would be two less cardiovascular events, yet 1 more diagnosis of diabetes.  Also, unfortunately, the trial was terminated early with less than half of the expected events.  From a statistical perspective this increases the risk that the results could be misleading.

2. Jones P et al Am J Cardiol 2003;92:152-160 (STELLAR).

AstraZeneca sponsored this study and had their own writers involved.  It compares Rosvustatin (their product) with three other statins (incidentally the ones Pharmac funds).  They produce nice graphs which show that Rosvustatin in increasing doses reduces LDL cholesterol and that in the same doses it reduces it more than the other statins.  Sounds good?  Buyer-beware – they were not comparing apples with apples.  A relevant comparison would be to compare the manufacturers recommended doses with each other.  The dose is not the same for all statins.  Perhaps not surprisingly when they presented their comparative data in adverts in the US the FDA jumped on them – see

3.  Nicholls SJ et al Am J Cardiol 2010;105:69-76 (VOYAGER)

Another AstraZeneca funded and co-authored study.  This time it is a metanalysis comparing effects of dose of various statins on cholesterol.

An implantable artificial kidney

$100 Dialysis is my vision.  University of California San Francisco researchers are doing some exciting research that may well be a step on the way.  They are developing an implantable artificial kidney .  They have a facebook page for those wishing to follow progress.  $13M is needed to bring it clinical trials – I think they should try Kickstarter.  With 400,000 on dialysis in the US alone there will be plenty of contributors (If you are feeling generous now you can contribute by going to their website).  Alternatively, $13M is a drop in the bucket of the $29Bn that Medicaid (6% of its budget)* spends on dialysis every year!

A few interesting facts about UCSF’s artificial kidney:

  • No pumps and no power supply.  It relies on the body’s blood pressure to perform filtration.
  • The key to cleaning the blood is a good filter – they have developed nano technology to produce a silicon based filter with pores small enough and a structure that does not induce blood clotting.  See
  • Beyond simply filtering the nasties, the kidney’s role is to regulate salt and water reabsorption and blood pressure.  This is being achieved by using bioengineered grown tissue – that is real cells from inside the kidney. They call this the bioreactor.
  • They are currently in the “pre-clinical” stage where they are miniaturising the machine and preparing it for implantation into humans.

* If you want to know why this is so high and the practices in the US then see  You may be shocked (a must read for every for anyone in the US making choices about dialysis).