In The end, Rare Drug Pricing Will Hurt Pharma Just As Much As Patients

Radical reform is needed to break the vicious circle

There’s been an odd-sounding word circulating in the English headlines recently.

The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation.  True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein[1]. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.

Orkambi has been licensed by the EMA since 2015. It’s available to patients in the US and 11 other EU countries. In Britain, it is available on compassionate use only.

The latest spate of headlines came on Thursday, as even investors became uneasy over reputational damage, after seven executives awarded themselves paypackets big enough to cover one year’s treatment for 369 eligible patients.

I have cystic fibrosis, and I’m tired.

Not on this occasion, because of my condition. I’m tired of the combative exchanges between campaigners, NHS England, Vertex and the CF Trust, that have still not led to access.

I’m tired of reading that Vertex’s stock has ‘tripled since 2015’, and that every second headline is not about lives to be saved, but a hot investor tip. I’m tired of the paradox thrown up repeatedly, that tomorrow’s R&D demands today’s executive remuneration. With automation speeding up high-throughput screening exponentially, that won’t be true for long.

I have accepted the fact that I may never swallow a pill created by Vertex. But I cannot accept the pricing precedent set by them. It is a precedent, yes, because although not the first time an expensive orphan drug has come to market, Orkambi is the first to demand figures over £100,000 - $240,000 in the US - per patient, per year for what is an incremental outcome[2]. Impressive benefits reported by patients have come from the follow-on drug, Symkevi, and the triple-therapy modulators currently in phase III trials. These are expected to be priced higher still.

As one of approximately 30 million EU citizens with a rare disease, I am keenly aware of this human cost. Many rare diseases, like CF, are debilitating and life-shortening. According to Eurordis, rare diseases are estimated to be 80% genetic in origin, meaning they hold enormous therapeutic, and commercial, potential.

Curing diseases may make Wall Street nervous, but so will the alternative
Potential that will be wasted if the goal of treatment is never to cure, but to prolong monthly scripts. Thanks to the Gilead parable wherein the company became less profitable when it created an effective cure for Hepatitis C, the 'treat not cure' model remains infallible. That may change when it comes up against competing research models and motivations in the future.

A CF example again: in February this year, researchers at Edge Hill University quietly announced a world-first in nanoparticle gene therapy. Their aim is to reduce treatment burden dramatically and to one day offer a realistic ‘cure’. It's easy to see, when the time comes, which approach payers will support.

Nationalised services likes the NHS may be the canary in the coalmine when it comes to reimbursement, but healthcare providers across Europe and the US are struggling. What’s required is less a Marshall Plan, more an approach of symbiotic survival. A fundamental, voluntary, and no doubt painful, overhaul of rare drug pricing. With the view of keeping the payers - whether health service or insurance company - of tomorrow healthy and viable customers long into the future. How else will pharma keep their customers in business?

This isn’t just about looking within; bringing drugs to market at lower initial prices or in managed portfolio agreements means co-operative, faster decision making from regulators. Together with a desperately needed re-evaluation of HTAs, that could mean thousands of patients accessing drugs months and even years faster. And that means quicker returns for manufacturers.

From uncomfortable bedfellows to partners
As a rare disease patient, I’d like a stronger relationship with pharma. Empathy on both sides is not as good as it could be, and for that to change we need closer collaboration.

We, as a cohort, remain more motivated than your average Type 2 diabetes sufferer to get involved in the care and research of our condition. Industry must use that desire. The reasons are many; the discovery of more relevant, accessible primary endpoints in trial co-design. Improved patient access and recruitment. Increased understanding and patience from patients when reimbursement problems arise. And when patients finally access treatments, they are likely to be more trusting of and more adherent to their medication.

I believe that isn’t just good treatment, but good business.