4:51 PM
Feb 23, 2018
 |  BioCentury  |  Strategy

Patients as pawns

How Vertex and France's health authority actions penalize patients

In an apparent game of chicken between Vertex Pharmaceuticals Inc. and French health authorities over the price of the cystic fibrosis drug Orkambi, patients say the decision to use access to investigational therapies as a negotiation chip goes too far.

Orkambi ivacaftor/lumacaftor was approved in the EU in November 2015 to treat CF patients 12 or older who have two copies of the delta F508 mutation. Since then, the biotech has been locked in negotiations with France’s Comité Economique des Produits de Santé (CEPS) over the drug’s price.

Negotiations came to a head this month when Vertex sent a letter to French clinical trial sites canceling three planned Phase III trials to evaluate double- and triple-combinations from the biotech’s next-generation CF portfolio. The first trial started on Feb. 21 at sites outside of France, according to the company’s press release.

Vertex pulled the trials because CEPS wanted an 80% discount to the biotech’s latest offer on Orkambi. The company is not disclosing the price it offered.

“If countries can’t recognize the innovation that we can bring -- and an 80% discount isn’t recognizing innovation -- then it is not a viable business option for our other medicines,” the biotech told BioCentury.

France’s CEPS and Health Ministry have not responded to BioCentury’s requests for an interview or comment.

“It is not right to use the most vulnerable party in the whole discussion as a pawn.”

Ulrike Pypops, CF patient

But CF patients and other patients who have benefited from clinical trials see the actions by both parties as punitive. The perception is that each side is waiting for the other to back down.

“Patients and families are being used as a trade in negotiations between a company and health authorities,” said Ulrike Pypops, a CF patient who lives in Belgium. “From a patient perspective, it is not right to use the most vulnerable party in the whole discussion as a pawn.”

Based on Vertex’s account of the negotiations, both the company and French authorities are violating the social contract described in BioCentury’s 25th annual Back to School Essay. The contract obligates drug companies to help patients live better and longer, and society to pay enough for medicines so that investment in innovation continues.

Above all, for the contract to work, patients must have access to medicines that will improve their lives at prices they can afford.

While Vertex might have a case for pushing back on the size of the price reduction, patients think it’s wrong to couple pricing negotiations for an approved therapy with access to other novel and potentially beneficial experimental agents.

In CF -- a progressive disease where the majority of patients lack disease-modifying treatments -- any decision to refuse access means some patients will suffer irreversible declines.

“I’m concerned that they’re making this trade off in reimbursement and withholding the opportunity to participate in a trial. They are playing with patients’ lives,” said Bray Patrick-Lake. Patrick-Lake is not a CF patient, but did suffer from a debilitating disease until she received an investigational therapy as part of a clinical trial.

According to at least one expert on market access, Vertex has alternatives apart from simply agreeing to the price proposed by CEPS. But if it wants to gain any revenue or the chance to gain additional evidence that could support a higher price for Orkambi, it will have to court a patient community it has angered -- a tough prospect without reinstating the trials.

The French way

The deadlock...

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