The impasse over access to Vertex’s Orkambi for cystic fibrosis patients in England and Wales has revealed the cracks in the NHS’ regulatory system. Matt Fellows explores where the core issues lie in the debate, and what can be done to prevent future breakdowns in communication for the good of patients.
Pricing is an issue that is rarely out of the news, and it’s not difficult to see why: health is an issue for literally every single person on the planet, and to bring money into the equation as a requisite for accessibility was never going to go well. But medicines must be developed and delivered within the existing national or international framework, and that naturally renders any access efforts vulnerable to conflicts of interest and financial concerns.
The issue inspires intense debate for this reason alone, but on a deeper level, requiring stakeholders to debate and affix a price tag on a medicine only invites or even requires them to court the unavoidably subjective notion of the intrinsic value of a drug. What value can it bring? How many lives will it save? How can its benefits for patients be neatly quantified and monetised? How can it make a return on investment? It’s seems an almost inherently self-defeating discussion when you have two parties on either end of the negotiating table whose primary [modus operandi] is to protect their own interests within strict red lines.
Of course, this is perhaps slightly more applicable to the system in the UK than in, say, the US, which faces entirely unique problems within its own highly-publicised debate on pricing and the value of drugs. While some of the most prominent Big Pharma CEOs lined up in February before US Congress to justify the high prices of their medicines – a line-up which included AbbVie’s Rick Gonzalez, Pfizer’s Albert Bourla, Bristol-Myers Squibb’s Giovanni Caforio, MSD’s Kenneth Frazier, Sanofi’s Oliver Brandicourt, and Johnson & Johnson’s Executive Vice President Jennifer Taubert, who stood in for Chief Executive Alex Gorsky – the UK’s cystic fibrosis (CF) community has been grappling with a crisis of its own, and one that is emblematic of the wider pricing and access debate.
NICE has been locked in negotiations with Boston-based Vertex for a number of years, negotiations which have starkly highlighted the distance between the two organisations’ stances on the perceived valuation of the medicine. And with these discussions at a stalemate, approximately half of the 10,000 eligible patients in dire need of an effective treatment for the debilitating condition are denied access until the stand-off is resolved.
But the story stretches back even further than that. [Pharmafocus] spoke to Nick Medhurst, Head of Policy and Public Affairs at the Cystic Fibrosis Trust, to get the full picture that has led to this point.
"The story really starts back around 2012 with Kalydeco, Vertex's first medicine in CF and the first drug in CF to target the root cause by modulating the cystic fibrosis transmembrane-regulating (CFTR) protein. It's why this category of drugs is so innovative in CF: Kalydeco when it was first licensed was only available for one specific CF-causing mutation, which only 5% of the UK CF population had," he explained. "We could probably get bogged down in the detail of how that got reimbursed but it basically rose up through the gap when the Health and Social Care Act came in, and specialised medicine commissioning moved from being the domain of the Advisory Group on National Specialized Services (AGNSS) to NICE. It was effectively approved within six months for routine commissioning by NHS England, and then in 2015 Orkambi was licensed, and Orkambi expanded the treatable population to more than 50% of people with cystic fibrosis, because it targets people who have two copies of the most common CF-causing mutation.
"In 2018, a new iteration of that medicine was licensed: Symkevi, which slightly expanded the treatable population again, but is effectively a demonstration of a rolling pipeline of medicines being released by Vertex, but we hit the buffers back in 2015; we're expecting in the middle of this year data for a Phase 3 trial to be released by Vertex on what is known as a triple combination therapy – the drug compounds that are in Symkevi, which are Kalydeco, ano...
(All Rights Reserved)