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

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