Some patients with a rare disease face hurdles getting the only treatment

Victor A. Mejias had been taking a drug to treat a rare skin disease for about two years when its manufacturer effectively cut off his supply.

Mejias was switching dermatologists and needed to find someone approved to administer the treatment by Clinuvel Pharmaceuticals Ltd, which makes the drug. But he says the company wouldn’t help him find a new doctor. Mejias was enrolled in a study for another treatment and the drugmaker told him it would give him the name of a center only after he was done with the trial.

“I was so flabbergasted,” Mejias said. “This deep depression sets in. What am I going to do?”

Clinuvel’s drug, known as Scenesse, is the only US-approved drug for erythropoietic protoporphyria, or EPP, a genetic disorder that causes intense pain when sunlight hits a person’s skin. It’s been on the market since 2020.

The Melbourne-based company is an outlier in the pharmaceutical industry in that it keeps tight control over who can administer its drug. It doesn’t publish a list of the doctors it’s trained and approved and declined to give one to Bloomberg.

It also discourages patients like Mejias from participating in trials of other treatments, and has without warning barred a hospital from administering Scenesse.

Clinuvel’s business practices are highly unusual. In the American heath care system, drugmakers generally don’t get to choose which doctors can provide their medicines.

“Pharmaceutical companies don’t practice medicine,” said Ryan Nash, the director of the bioethics center at the Ohio State University. “It would be inappropriate if they tried to move into the space of taking away a clinician’s ability to make clinical judgment.”

Steven Joffe, chair of medical ethics and health policy at the University of Pennsylvania’s medical school, said it could be an intrusion into the doctor-patient relationship. “When my doctor and me go into the clinic room together and we close the door, a company’s commercial interests don’t belong in that room with us,” he said.

Mejias was participating in a trial of cimetidine, which has been sold as the cheap over-the-counter acid reflux medication Tagamet for decades. The sticker price of Scenesse, administered as an implant, is about $57,000, according to the consultancy 3 Axis Advisors. Depending on need, patients can get a new implant every two months.

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