Medicine

Drugs for rare disease out of reach for Bend patient

Orphan drugs tend to carry high price tags

By Tara Bannow, The Bulletin, @tarabannow

Wanda McTee has more hope these days than she’s had in a long time.

That’s despite the fact that the 73-year-old Bend resident is at the five-year mark on an incurable disease that typically kills people within five years. Despite that her lungs are covered in irreversible scarring. Despite that she’s mostly bedridden and can’t speak for more than few minutes at a time.

She has hope, her husband Chester McTee says, because the U.S. Food and Drug Administration last fall approved this country’s first two medications for her condition, called idiopathic pulmonary fibrosis, a progressive scarring of the lung tissue that usually kills patients from respiratory failure.

“She is willing to fight it, even knowing what they say about the five years,” he said.

The FDA approved the drugs, nintedanib — brand name Ofev — and pirfenidone — brand name Esbriet — on the same day in October 2014. They were fast-tracked through the typically expensive and lengthy drug approval process because of their so-called orphan status, a title the FDA grants to products for diseases that affect fewer than 200,000 people in the U.S. About 140,000 Americans have pulmonary fibrosis, according to the American Lung Association.

But it’s still expensive and time-consuming to develop the drugs, and manufacturers expect to recoup those costs when they bring them to market. Since there are a comparatively small number of people to sell them to, orphan drugs tend to be more expensive than non-orphans. Some experts say orphan drugs have been getting even more expensive in recent years.

“We need better systems to think about ways to create orphan drugs that don’t require such higher prices,” said Dr. Steven Pearson, president of the Institute for Clinical and Economic Review, a nonprofit that analyzes new drugs. “I think we need to think about ways insurers can cover them and not break the bank, but in a way that doesn’t push all the costs back onto the individual patient.”

A year’s supply of Esbriet costs $94,000. A year of Ofev: $96,000. For Wanda, that broke down about $2,000 per month for each drug after Medicare paid its share — far more than the McTees can afford. (The McTees could not provide a receipt proving the $2,000 price tag because they said they could not afford to purchase the drugs, but some research has estimated a 30 percent coinsurance rate on orphan drugs.)

Chester said he understands producing drugs is expensive. He said he wouldn’t expect free pills, but thinks people should be able to buy them at a “reasonable” price.

“Even if she could get enough pills just to try them out to see what it could do,” he said. “Hell, I’d probably sell my house, my cars, myself and whatever. Get ten cents for me.”

Expensive orphans

Orphan drugs are expensive because they’re designed to treat small populations. They also tend to be unique products with no competitors, Joshua Cohen, a research associate professor at the Tufts Center for the Study of Drug Development, wrote in an email.

Of particular concern among policymakers, insurers and patients is the growing number of orphan drugs that cost more than $100,000 per patient per year, Cohen said.

“Ten years ago, there were very few,” he said. “Now, there are at least a dozen.”

In a 2013 article, Cohen listed 11 orphan drugs with annual costs exceeding $225,000. They included the short bowel syndrome drug Gattex at $295,000, Kalydeco, for cycistic fibrosis, at $325,000 and Myozyme, for Pompe disease, at $575,000.

The orphan program offers other benefits, too: tax credits, reduced fees, clinical trial grants and seven-year protection from competition, M. Ian Phillips, director of the Center for Rare Disease Therapies at the Keck Graduate Institute wrote in a 2013 article. And while the FDA requires orphan drugs meet the same approval standards as non-orphans, clinical trials can be smaller and faster, he wrote.

At the same time, Phillips wrote the pipeline for non-orphan drugs is slowing. In 2012, many non-orphans fell off the “patent cliff,” meaning patents were expiring faster than they were being replaced and generic and biosimilar drugs were increasing competition, he wrote.

Despite their comparatively small reach, orphan drugs have been shown to create significant profits for manufacturers. The rare disease industry has reported gross profit margins of more than 80 percent, compared with 16 percent average in the pharmaceutical industry, according to Phillips’ study.

Dr. Louis D’Avignon, a pulmonary specialist with Bend Memorial Clinic, said he wasn’t surprised by the prices on Esbriet or Ofev, given their orphan status. He now has conversations with patients who, even after their insurance covers a portion, still can’t afford the new drugs.

Most patients have insurance and thus don’t see the original $94,000 to $96,000 price tag, D’Avignon said. But the costs are felt at a societal level, because insurance carriers and governments end up footing much of the bills, he said.

“Eventually, the cost gets out to everybody,” D’Avignon said. “If the insurance is having to pay for this or if it’s an (Oregon Health Plan) patient and the state is having to pick up the cost of this medication, that eventually makes it back to everybody in some form.”

‘It was a big deal’

Still, advocates say the drugs’ approval represented a major advancement in the treatment of idiopathic pulmonary fibrosis.

“It was a big deal,” said Dr. Gordon Carr, the director of the University of Arizona’s Interstitial Lung Disease Program at the University of Arizona Medical Center.

Before the approval, there were no treatments for the disease, whose cause is still unknown. Wanda has none of the typical risk factors for lung disease: She never smoked and didn’t have prolonged exposure to secondhand smoke or asbestos.

Physicians can treat some types of pulmonary fibrosis by linking them to an underlying cause, such as an autoimmune disorder like rheumatoid arthritis or lupus, and attacking that, Carr said.

“But in cases of idiopathic pulmonary fibrosis, which means pulmonary fibrosis where we can’t find a cause, we really didn’t have any satisfactory therapies,” he said.

The past 15 years have seen a lot of momentum and enthusiasm around getting drugs to treat scarring lung diseases through the pipeline, said Dr. Gregory Cosgrove, chief medical officer for the Pulmonary Fibrosis Foundation.

“That led to, in all honesty, some frustration because of the intense need to identify a treatment for this devastating disease,” he said.

Ordering drugs online

In the meantime, other countries approved versions of pirfenidone that weren’t yet available in the U.S., prompting some patients, like the McTees, to order them online. Friends of the couple recommended a pharmacy in Canada, Chester said.

Buying prescriptions from other countries is technically illegal in the U.S., though it’s not likely to be prosecuted. Doctors are sympathetic to the plight of patients who have no other options, although they warn it’s difficult to verify the medications’ safety or authenticity.

D’Avignon, Wanda’s physician, said he knew she was ordering the pirfenidone; many patients did before the new drugs were available in the U.S. The harder conversation is whether patients can even afford the drugs now that they’re here, he said.

“Is it something you can get even if it is available?” D’Avignon said.

For his part, Cosgrove said he does not advocate ordering medications online because he believes the FDA’s process is necessary to ensure they’re safe and effective.

Wanda recently stopped taking the generic pirfenidone because she believes it caused stomach pain.

Both of the new drugs’ manufacturers, InterMune and Boehringer Ingelheim, offer free or reduced price medications to patients who meet certain criteria, although neither would provide specifics on who would qualify. Such programs typically aren’t available to Medicare patients like Wanda because of a federal anti-kickback statute that prohibits manufacturers from providing products to patients in an effort to influence them to choose those particular products.

D’Avignon said he’s had some luck getting patients enrolled in such programs, but it requires a significant amount of paperwork on the provider end.

As for Wanda, D’Avignon said on one hand, she’s beating the odds with respect to how long she’s lived with the disease, but she’s also one of his more severe cases. Unfortunately for her, the new drugs have the biggest impact if taken earlier in the disease’s progression, while they can still preserve lung function, he said.

After less than two minutes of being interviewed for this article, Wanda’s breathing intensified as though she were running, and a slight wheezing could be heard between her words. She’s on oxygen 24 hours, seven days a week, and doesn’t leave her bedroom except to go to the doctor.

That’s left Chester, 83, as his wife’s caretaker. He’s taken over the cooking, cleaning and even the plumbing around the couple’s well-manicured home neighboring Pilot Butte to the south.

Wanda said she can get dressed and use the restroom on her own — but after that, it’s straight back to bed.

“That’s where I’m at the rest of the day,” she said.

— Reporter: 541-383-0304,

tbannow@bendbulletin.com

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