The Orkambi Debate at Westminster Hall

Introduction

On 19th March 2018, a debate was held in Westminster Hall to debate Orkambi’s®¹ availability on the NHS. Watch the debate here (Parliamentlive.tv, available until approximately 26th March 2018). [A transcript is also available] (https://hansard.parliament.uk/commons/2018-03-19/debates/41D2017B-E026-49FC-B94E-BCDB566AF1D6/OrkambiAndCysticFibrosis) (Hansard.parliament.uk. I believe this link will work permanently). The debate was attended by approximately 60 MPs, but attendance at Westminster Hall debates are normally attended by under 15 MPs. The debate was in response to ePetition 209455 (parliament.uk) which was calling for Orkambi to be made available on the NHS, which following NICE (National Institute for Health and Care Excellence. Web link nice.org.uk) recommendations in July 2016 was not funded by the NHS “on grounds of cost-effectiveness and a lack of long-term data."² . As health is a devolved issue, Scotland, Wales and Northern Ireland are handled separately. At the time of writing, Orkambi® has not been recommended by any of the devolved equivalents to NICE.

The debate and its results

The debate was lead by Paul Scully MP (Sutton and Cheam, Conservative. Web link Parliament.uk), with significant input from Ian Austin MP (Dudley North, Labour. Web link Parliament.uk) and closing arguments from Steve Brine MP (Winchester, Conservative. Web link Parliament.uk), the Parliamentary Under-Secretary for the Department of Health and Social Care.

Many Members who spoke at the debate repeatedly stated that the system NICE is using to decide on the efficacy of new targeted drugs is no longer fit for purpose. This affects other targeted treatments, not just those for CF, which as Sharon Hodgson MP (Washington and Sunderland West, Labour. Web link Parliament.uk) said includes “access to cancer drugs”. This is something both she, and the Minister have worked on access to in the past.

Below I quote part of the Minister’s (Steve Brine) response to the debate:

“It seems to me that the main ask I have heard from pretty much every hon. Member speaking today is, “Get involved.” I am not the Minister directly responsible. That falls to Lord O’Shaughnessy, the Parliamentary Under-Secretary who sits in the other place, but I can say on his behalf and on mine that we are involved, and you can bet your life that we will continue to be involved. I do not think I can be clearer."³

“The other place” is a parliamentary term for the House of Lords/House of Commons.⁴ It is a convention that neither House in the Palace of Westminster refers to the other House directly when speaking in the Chamber. As a member of the House of Lords, Lord O’Shaughnessy (Conservative) the Life Sciences Minister, was precluded from attending the debate.

The Minister also mentioned that as NICE is independent of Government, the Government cannot and should not mandate NICE (Or any of the devolved equivalents) to fund Orkambi on the NHS. To do this would undermine NICE’s independence from Government; something I agree it should maintain. The Minister pointed out that NICE have a very taxing job, and new targeted treatments represent an immense challenge to the NHS. He goes on to say that medicines aiming at treating CF get “the same quality, safety and efficacy”⁵ as other medicines while representing “the best possible use of funding for patients and the NHS."⁶. In summarising his closing arguments, he points out NHS England put a counter offer to Vertex last week (Week ending Friday 16th March 2018) which was rejected by Vertex. A copy of the CF Trust’s tweet (Twitter) showing Vertex’s statement can be found on Twitter. The Minister did not reveal the offer that was rejected, but he does state that there are more negotiations to be had, as the current offer is still far from cost-effective. He encourages all sides to get back to the negotiating table (And not via email, which Vertex state in their statement is how negotiations are/were being conducted). Both the Minister and Lord O’Shaughnessy believe a mutually satisfactory deal is achievable, and he finished by assuring the House, that he and Lord O’Shaughnessy had heard the message of “Get involved” loud and clear.

My reaction

In my opinion, reviewing NICE’s criteria for acceptance of targeted drugs for all currently licensed and future treatments for illnesses that could benefit from targeted drugs is the way we will get treatments such as Orkambi® funded by the NHS, irrespective of where you live. This must not be a “postcode lottery” as many MPs said.

Moving forward Ian Austin MP will be writing a cross-party letter to Jeremy Hunt MP (South West Surrey, Conservative) The Secretary of State for Health and Social Care, and Lord O’Shaughnessy which needs support from as many MPs as possible. Therefore, I urge you to write to your own MP asking them to sign this letter in support of reviewing NICE’s criteria for funding new specialist treatments. You can find your MP here and entering your postcode, constituency or MP’s name.

Unfortunately, my own MP, Jonathan Djanogly MP (Huntingdon, Conservative. Web link Parliament.uk) was unable to attend the debate, so my story was not able to be represented. Given this, I offer my own story below, and also expressly thank all MPs who attended and represented their own constituents and outlining their own personal experiences with CF during the 3-hour debate.

My story

I started Orkambi® (Orkambi.com) in July 2017, under a special access program run by Vertex (The manufacturer). The special access program is designed to gather more information on the effects of Orkambi® so NICE have a better range of data demonstrating Orkambi’s® effectiveness (And therefore it’s cost efficiency). As we enter Spring 2018, I have managed to go just under 6 months without IVs. This is the longest period I have gone without IVs for a considerable time, especially over a winter period; where I would normally need 4 - 5 sets of IVs.

Orkambi has not increased my lung function much (I’ve gained an average of 1%), but it has massively improved my quality of life. I’ve been able to continue working full time (Something I was close to giving up), and even started working for myself. While I have no figures as to the exact money my being on Orkambi has saved the NHS, I estimate it will be the cost of at least 3 more sets of IVs and associated clinic appointments and hospitalisations. In addition I have generated additional revenue for the Exchequer in taxes paid. This is something NICE do not take into account. Their decisions are based solely on costs to the NHS, and other areas where costs could be offset, such as taxes paid, or lower/stopping benefit payments are ignored. A significant number of businesses have been started by CF patients since starting Orkambi® or Kalydeco®, as they now feel healthy enough to work. Kalydeco® is a targeted treatment by Vertex for a different CF mutation, which is funded by the NHS. It works on approximately 4% CF patients in the UK. Orkambi could help approximately 50% of UK based CF patients.⁷ I am hoping in an upcoming review of assessment criteria for NICE (Scheduled for Summer 2018) all aspects of potential cost offsetting is taken into account, not solely those directly benefitting the NHS.

To summarise Orkambi hasn’t given me the lung function benefits it might have. I know of one person who went from 35% lung function up to 75%, but it has given me my life back. If you had told me 9 months ago I’d have gone through a winter season with only 2 sets of IVs, 6 months apart, and I’d have a significant improvement in my quality of life, I wouldn’t have believed it possible. Although I am a little disappointed not to have gained much lung function back.

How can people help?

As many of us as possible need to write to their MP. If you would like further information about the CF Trust’s Life Unlimited campaign can be found on the Your voice pages of the CF Trust’s website.

Thank you for taking the time to read this summary of the debate in Westminster Hall.