[Ip-health] New York Times: Making ‘Every Patient Counts’ a Business Imperative

Thirukumaran Balasubramaniam thiru at keionline.org
Thu Jan 31 01:22:21 PST 2013


Making ‘Every Patient Counts’ a Business Imperative
Published: January 30, 2013

BEDMINSTER, N.J. — Drug companies are fond of saying that every patient counts, but in the world of orphan diseases, entire business plans are built around the idea. Gattex was developed to treat fewer than 5,000 patients.

That is why Andrew E. Jablonski, a 26-year-old from Lincoln, Neb., was a guest of honor this month at NPS Pharmaceuticals, a small New Jersey company that is about to begin selling Gattex, its only approved drug. Mr. Jablonski, who was born missing most of his intestines and has a condition known as short bowel syndrome, is central to the company’s mission to win over the tiny pool of patients — fewer than 5,000 nationwide — that the drug is intended to treat.

Over the last two years, NPS has carefully tended its relationship with Mr. Jablonski, helping to finance his nonprofit, the Short Bowel Syndrome Foundation, and flying company leaders to visit him in Lincoln. On Jan. 15, he met with the chief executive and strategized with marketing employees about how to promote Gattex to his social network of 1,000 patients and caregivers.

“It’s a nice, close relationship,” Mr. Jablonski said. “I’m an asset to them, as they are an asset to me.”

NPS hopes that Gattex will ultimately bring in hundreds of millions of dollars in annual sales and help establish the company in the increasingly hot orphan drug market, where companies can charge premium prices to treat overlooked diseases with little or no competition. NPS expects to charge about $300,000 a year for Gattex, a breathtaking price tag that is nonetheless on par with other ultra-orphan drugs on the market.

Development of these drugs is increasingly attractive to pharmaceutical companies, which are searching for new sources of revenue as sales of more traditional, mass-market drugs have been lost to generic competition. The orphan drug market was worth more than $50 billion in 2011 and turns out blockbusters at the same rate as the broader industry, according to a recent Thomson Reuters analysis. Pfizer opened a rare disease unit in 2010, and in 2011, Sanofi acquired the biotech company Genzyme, considered one of the pioneers of the orphan drug industry.

As orphan drugs have become increasingly popular, companies have honed their marketing techniques, knowing that they cannot afford to lose a single patient. Following in the footsteps of drug makers like Vertex, which makes the cystic fibrosisdrug Kalydeco, NPS has formed close bonds with advocates like Mr. Jablonski and hired so-called patient care coordinators who will do everything from making sure that Gattex is delivered to patients’ doorsteps to writing appeal letters if an insurer refuses to pay for it.

Much is riding on the drug’s success: Gattex is the centerpiece of a comeback plan for NPS, which nearly folded six years ago after a previous drug, to treat osteoporosis, failed to win approval from the Food and Drug Administration. After that setback, the company closed its Utah office and laid off nearly all of its more than 400 employees. NPS has since shifted its focus to orphan drugs and now employs about 140 people, including 27 new sales representatives hired to sell Gattex.

If Gattex succeeds, NPS predicts the drug could bring in more than $350 million a year, which would significantly improve the company’s fortunes. NPS had $101.6 million in revenue in 2011, mostly from royalty payments on drugs that were licensed to other companies. It had an estimated $133 million in revenue in 2012, according to a report by the investment bank Canaccord Genuity.

To make Gattex a success, however, NPS will need to rely on people like Mr. Jablonski, who founded his group in 2011 to bring together the diverse group of patients who have short bowel syndrome.

People with the condition have lost part of their intestines for a variety of reasons, like car accidents, cancer and complications of diseases like Crohn’s. Many patients cannot absorb enough nutrients through food and require an intravenous feeding line to survive. Gattex is designed to decrease their reliance on such lines, improving their quality of life and lowering their risk for life-threatening complications like infections.

During Mr. Jablonski’s visit to NPS, employees briefed him on nearly every aspect of the drug and its rollout, from common questions that members of his foundation may ask to how they can get help paying for the drug. Mr. Jablonski, who is not on intravenous nutrition, said he had formed a close relationship with NPS because patients with short bowel syndrome were desperate for an effective treatment. He said he did not see a conflict between his role as an advocate for patients and his collaboration with NPS.

“Our mission, our goals and values, they closely align,” he said.

In clinical trials, about two-thirds of patients saw at least a 20 percent decrease in the volume of intravenous nutrition they required per week. Patients on the drug are believed to be at a higher risk for cancer and other conditions.

Many patients have heard stories like that of Devera Marshall, who participated in clinical trials and has been able to stop using intravenous nutrition altogether.

“I saw pretty instant, miraculous effects,” said Ms. Marshall, a mother of three from San Diego who said she had returned to playing tennis for the first time since losing most of her intestines after complications from surgery five years ago.

But she gasped when a reporter told her the price of Gattex, which she is receiving free because she is participating in a long-term trial. “Wow — I’m a little floored,” she said. “Does this mean I get on the horn with my insurance company and start begging and pleading?”

Despite the high cost of the drug, NPS executives say few, if any, patients will have to pay much for it. The company will cover out-of-pocket expenses for patients who are privately insured if the costs exceed $10 a month, and nonprofit foundations that receive financing from NPS will pay the expenses for people insured by Medicaid andMedicare. NPS will cover the full cost of Gattex for those who do not have any insurance.

The company said it surveyed major insurers before setting its price and was told the drug would most likely be covered. NPS executives point out that the cost of intravenous nutrition is more than $100,000 a year, and expenses like home health aides and hospitalizations can drive even higher the cost of caring for patients with the condition.

Taking a page from other companies that have recently introduced profitable orphan drugs, NPS will put every person who is prescribed Gattex in touch with a dedicatedcare coordinator who will walk the patient through the insurance reimbursement process and answer questions about the drug.

NPS said that it had identified about 1,000 eligible patients through partnerships with five home infusion companies that provide care to patients with short bowel syndrome, and that it expected 200 to 300 patients to be using the drug by the end of the year.

Karen Galambos, one of the coordinators, said she expected to get to know each of her patients on a first-name basis, exchanging photos, perhaps, or calling just to check in. “My goal is to hold the patient’s hand through the whole process,” she said. “We’re not going to let go of the ball.”


Thiru Balasubramaniam
Geneva Representative
Knowledge Ecology International (KEI)

thiru at keionline.org

Tel: +41 22 791 6727
Mobile: +41 76 508 0997

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