Tuesday, July 08, 2014

Pomalidomide update: a question of statistics

Information from the Scottish Medicines Consortium website, published 7 July 2014:

"pomalidomide (Imnovid)

following a full submission

pomalidomide (Imnovid®) is not recommended for use within NHS Scotland.

Indication under review: in combination with dexamethasone for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy.

Pomalidomide plus dexamethasone significantly increased progression-free survival compared with high-dose dexamethasone in patients with refractory or relapsed and refractory multiple myeloma.

The submitting company did not present a sufficiently robust economic analysis and in addition their justification of the treatment’s cost in relation to its benefits was not sufficient to gain acceptance by SMC."

I read the full adjudication.  The facts are fairly clear:  the drug is too expensive and its benefits are insufficient to warrant the cost.  It didn't help that the drug company prepared their stats on the basis of a 25-year life expectancy, when the median age of diagnosis for myeloma is 70.  Quite rightly, the SMC queried this, and adjusted the cost / benefit analysis to reflect a more realistic 10 years.  Of course, if you are the statistical anomaly who develops myeloma in your 20's or 30's, you would argue that 25 years was just about reasonable... but that's not how statistics work!

The drug company estimated that 132 people in Scotland would have been eligible to receive the drug in its first year, if approved, for a total cost of over £6 million in that first year.
That is an insane amount of money!

FL has always been clear that he didn't want the NHS to squander its resources on "an old man" when there are younger people out there in greater need, in his words "more deserving".  But 3 months on Pomalidomide have hammered his cancer. His kappa score has plummetted.  So it is hard to accept that at next week's hospital appointment he will more than likely be taken off the drug that has helped him because it does not represent "value for money".

"The greatest good for the greatest number" - we still believe in it!  But FL has a book to finish.  And he's still the man I fell in love with all those years ago.  And I don't want to lose him sooner than I have to.

More and more I wonder - who sets the cost of the drugs in the first place?  How can those tiny little capsules possibly be worth so much money?  And if the drug company can't sell them to the NHS, they have no market in the UK.  So why not cut the price to attract the target customers?  Buy one get one free.  Isn't that how business works?

So even though Pomalidomide has brought my husband back from the brink of death, neither he nor anyone else in Scotland can have that same opportunity to stay alive because of an "insufficiently robust cost / benefit analysis" by some poor sod in the finance department of Celgene.

The cited (only?) study of this drug indicates that it increased the duration of survival without progression of cancer by an average of around 7 weeks in the (small) group of patients studied.
132 people could live for 7 weeks for £6 million.
I start to understand why relatives and friends of cancer patients become obsessed by fundraising.
It sounds plausible: you can buy survival. 

But then you read on, and you realise that it is just a temporary fix.  Pomalidomide is NOT a cure for myeloma, it just holds it at bay for a few short weeks.  And if you had £6 million to spend on drugs,  wouldn't you rather save it for the cure?

So could someone do some work on that, please?  And could you be quick about it - we're running out of time here.


Wakeymakes said...

Stay strong sweetie you are not alone. My dad was given 3 to 5 7 years ago and has had 3 new treatments develop in that time xXx

Sew little time said...

oh roo, what a horrible situation. i applaud FL's determination not to "squander" resources on an "old man" but that's so hard for you both. drug companies can charge what they want if the drugs work i guess. Big Pharma (as per ben goldacre) is not really focused on the patients as much as the money - as seen by the amounts they try to charge the third world countries for HIV/ AIDS drugs.

will be thinking about you both.

Sarah said...

You analyse the simultaneous and conflicting reactions really clearly. I feel for you both.

Sarah said...

You analyse the simultaneous and conflicting reactions really clearly. I feel for you both. Sarahel

ambermog said...

Damn:( I believe everyone should have access to necessary drugs regardless of age. mr Mog is 70 next month and neither he nor FL are past their best or old. They are human beings who deserve ANY help there is available to enable them to enjoy the rest of their lives as much as possible. Hugs xx

beate grigutsch said...

i wish you strength - and tons of luck for FL. strength he has, the wonderful man :-)

Linda C said...

Oh Roo, too hard. I am so sorry. We are getting more into that sort of thing over here--who is worth more to society, which can be so subjective. Who know how valuable FL's book could be to society or (just as important to me) how much pleasure could he give to how many people, or how much education for young people?

I am not sure who to blame about the high cost of drugs - but I do think Big Pharm and the Insurance Companies are in bed with government officials and their advisors.

Small comment- Husband went to get his cloresteral med refill this weekend, and sudden it is $700.00 and this is the generic.

Prayers and strength for you and FL!


Terrij said...

My daughter was diagnosed at 32 with Myeloma & she would like that 25 year chance. She is on maintenance therapy after transplant with Revilmid. It costs $13,000 a month. No one can afford that but her insurance & a grant pay for most of it. My son works in India & Revilmid ,same dosage, costs $500 a month. Pharms in US are outrageous.

Lynn Barnes said...

Oh, damn it all. There is a little boy in our community who is one of only about 100 people in the world with his condition. There is nearly no research on any medicines at all, because the costs so outweigh the risk of being born with his condition. I know whereof you speak, and it is just not fair. Not fair, not fair, not fair. Prayers for you and FL from here.

Lynn said...

Wish there waas a cure. Wish there were not such difficult decisions to be made. Wishing you strength and peace during this very difficult time.

Sew Brunswick said...

I'm a paediatric pharmacist and we battle from the other side. No evidence in kids. No company wants to invest in drugs for kids because the market is so small. Not enough money in it. I do know that it costs a hell of a lot of money to develop a drug from scratch to general release. So much money! We couldn't live without big pharma. Much love to you all. Thinking of you at this difficult time. L x

Janine said...

This is heart wrenching. I admire how brave you seem and how you and your beloved make the most of your lives. Take good care of yourself.

MaryinTN said...

Oh Roo. Dratted cost benefit analyses strike again! Very sad to hear that news. Anyone who is sick and wants the treatment should have the opportunity in my book

seashell bluez said...

Please remind your husband that the resources they use to maintain his health add to the data about the efficacy of the medicines in prolonging life.

In the US, drug companies put out ads saying to call them if you cannot afford the drugs. They have plans to help people. Does that happen where you are?

Roobeedoo said...

Thank you all for your supportive comments - every one is very much appreciated.
Seashell bluez - In Scotland, the National Health Service pays for all medicines it approves. There is private healthcare available, if you are willing and able to pay for it, but FL does not support the concept. I am not aware of drug companies themselves paying the cost of treatment, other than through clinical trials, which is how FL was funded for previous treatments.

liza jane said...

I just started reading a little bit of you and your husband's story. My husband was diagnosed with chronic myeloid leukemia almost 18 months ago. His life is totally dependent on a pill now. An extremely expensive pill! It's scary. So far our costs have been relatively low with insurance (employer insurance here in the US) and a patient assistance program through the drug company. I have a hard time rationalizing my extreme gratitude that the drug even exists over my disgust at the cost. Nothing is black and white. We just take it one day at a time. Best wishes to you and yours. I hope he gets to take it for the next 25 :)