Orkambi: Will you email your MP?

On Monday 12th August the Scottish Medicines Consortium (SMC) announced that the cystic fibrosis drug Orkambi would not be made available to Scottish patients on the NHS because of the high price tag. This announcement was yet another blow to patients and parents across the UK, some of whom had even considered moving to Scotland to access this medicine.

In England, for four agonising years patients have waited while drug company Vertex have refused to offer a fair price - even turning down the “largest commitment” the NHS has ever made of £500m per year over 5 years.

Negotiations have failed, and with no end in sight we are demanding that the government overturns Vertex’s monopoly so that patients can finally get access to the medicine they need.

Emily, whose son Jack has cystic fibrosis, has written to her MP asking for answers from Health Secretary Matt Hancock. Please join us and contact your MP too. You can use the email below as a template - feel free to add your own words.


Some pointers to help you:

  • Don’t know who your MP is? Just visit www.theyworkforyou.com

  • Remember to insert your own MP’s name at the start, and replace Emily’s name with your own at the end!

  • Remember to include your name and address so they know you’re a constituent.

  • When you get a reply please send it to organise@justtreatment.org

Here is the letter:

Dear MP,

We've been in contact a number of times regarding Orkambi and Symkevi, drugs which treat the root cause of Cystic Fibrosis but which are not available on the NHS.

You may have seen the news recently that the Scottish Medicines Consortium, having recently reviewed all of the latest evidence for both Orkambi and Symkevi, decided not to recommend either drug for general use in Scotland. This was despite applying extra flexibility around pricing given that these medicines are for a rare condition.

Let's be clear about what this means. The detailed SMC advice published shows that these drugs have clear clinical benefits. They calculated, for example, that Symkevi would generate more than 5 additional quality-adjusted life years (QALYs) over the current standard of care for some genotypes, and that Orkambi reduces the annual rate of pulmonary exacerbations in patients. However, even at the "discounted" price offered by Vertex they are simply too expensive to be recommended for routine use.

While this decision is incredibly disappointing, it is not surprising given Vertex's extreme pricing. What it does show is that NHS England are incredibly unlikely to be able to reach a deal with Vertex, and that the protracted negotiations which have been ongoing for almost 4 years are all but guaranteed to be unsuccessful. If a deal could not be reached in Scotland, where additional pricing flexibility was awarded, and where the patient numbers - and therefore total budget impact - is ten times smaller than in England, what hope is there that a deal can be reached in England?

There is, however, another option. Not being able to secure a deal with Vertex does not mean that patients should have to suffer untreated. There are companies in other countries making generic versions of these drugs, and our government should be urgently exploring how to get these generics to patients. Driven by desperation, a group of parents has set up the Cystic Fibrosis Buyer's Club which has extensively researched these options, and which has proposed 3 separate ways in which generics could be made available to all patients:

  1. The NHS could undertake a large scale clinical trial to generate additional important evidence about the efficacy of the medicine, utilising research exemptions which allow a generic version to be used.

  2. The government could invoke Crown Use licensing to effectively set aside Vertex's patent, demonstrating that they prioritise patient welfare above the greed of a pharmaceutical company described as an "extreme outlier" in pricing and behaviour.

  3. The NHS could provide personal budget payments to all Cystic Fibrosis patients, to enable them to privately purchase generics from abroad - rather than accepting that this is only an option for the most wealthy of patients.

Speaking in parliament in June the Health Minister, Seema Kennedy, rightly stated that the government has a moral obligation to explore all options that could ensure patient access, including these proposals. In light of the recent developments in Scotland, this must now be done with extreme urgency, so that patients are not left to suffer any longer.

I would very much appreciate your help in raising this with the Health Secretary Matt Hancock, and in asking for an update on what progress has been made in exploring each of these options.

Many thanks,


Elizabeth Baines