By John Miller
ZURICH (Reuters) - Swiss drugmaker Roche (ROG.S) is breaking into hemophilia A treatment, a $10 billion (£7.70 billion) global market dominated by rivals who have cultivated close ties to sufferers of the genetic bleeding disorder.
Roche's Hemlibra, on sale since late 2017 for patients with resistance to existing treatments, is widely expected to win the U.S. Food and Drug Administration's blessing for use in nearly all patients this week. The FDA declined to comment.
First-half sales were just 57 million Swiss francs ($58 million).
To hit $5 billion global annual sales that some analysts forecast, Hemlibra must wrest business away from hemophilia giants like Shire (SHP.L), Bayer (BAYGn.DE), Novo Nordisk (NOVOb.CO) and Octapharma, whose "clotting factors" are now standard for people who lack proteins that stop bleeding.
These companies have long sponsored patient groups, summer camps, even kayak trips on the Pacific Ocean, as part of efforts to build loyalty they hope will slow an exodus from their products once new drugs like Roche's arrive.
Roche is convinced word of mouth in the hemophilia community, backed up by its studies showing that Hemlibra shots may be more effective and must be administered less frequently than infusions of clotting factors, will overcome any current allegiances.
"I'm not familiar with what the other companies are telling the patients," said Dr. Gallia Levy, who heads Roche's Hemlibra program.
"What I do know is that I've seen a lot of patients telling other patients what is happening in their lives. Those sorts of stories mean the most to the patients."
As part of Roche's initial marketing campaign for Hemlibra, it has produced videos of hemophilia patients participating in daily activities, including riding bikes, in spite of their serious illness.
One video shows the consequences of failing to get appropriate treatment, featuring a Serbian man who suffered joint bleeds as a child and now must use crutches.
Hemophilia A has become one of medicine's most lucrative diseases, despite just 20,000 patients in the United States and several hundred thousand worldwide. It almost always affects men, passed down by a mutation on the X chromosome from their mother.
In 2016, the U.S. government alone spent some $2 billion on hemophilia A therapies, data from its Medicaid and Medicare insurance programs show.
Shire's Adynovate clotting factor runs at $537,000 annually, although costs for severe patients can hit several million dollars.
Roche has priced Hemlibra at $482,000, hefty but a level the independent U.S. pricing group ICER has said could cut overall hemophilia treatment costs significantly.
Traditionally a cancer drugs maker, Roche is turning to rare diseases like hemophilia to replace revenue from its older medicines whose expiring patents have exposed the company to unprecedented generic competition.
Hemophilia represents the largest market for rare diseases and is expected to grow above 7 percent per year through 2022, Evaluate Pharma has forecast.
'RELUCTANT TO SWITCH'
In August, Roche defeated a challenge from Shire aimed at delaying Hemlibra's broad introduction.
Shire fears losing 30 percent of its $2.4 billion annual clotting factors sales to Hemlibra in the short term, court documents show. However, it also indicated it expected patient allegiances to at least mitigate losses.
"Patients have loyalty to the brand and to manufacturers, and physicians are reluctant to switch due to risks," according to Shire testimony cited in the U.S. federal court judge's ruling.
Clotting-factor makers have fostered this loyalty, in part, by sponsoring summer camps for patients and their families. Camp Red Sunrise in Idaho's Rocky Mountains, sponsored by Shire and Bayer, among others, is free. Another Shire-sponsored retreat, Missouri's Camp Notaclotamongus, costs $25.
In September, Switzerland-based Octapharma sponsored a free sea kayaking expedition for 10 hemophilia patients on Washington State's Puget Sound, offering a chance to see pods of orca killer whales.
Shire, Bayer and Octapharma did not respond to requests for comment on their efforts to boost ties to patients.
Adriane Fugh-Berman, a professor at Georgetown University Medical Center in Washington, runs PharmedOut, a project that scrutinizes the influence the drug industry has on the practice of medicine.
A 2016 paper she co-authored described hemophilia's corporate-patient links and how companies use gifts, financial opportunities and one-on-one marketing to advance their commercial interests.
Hemophilia is unique, Fugh-Berman said, since patients play a bigger role in their treatment decisions than elsewhere in medicine where doctors more often call the shots.
"Industry's relationship with individual patients or health care providers is always about selling specific targeted products," Fugh-Berman told Reuters, adding tight corporate links to patients could hamper discussion including over drug prices, even among patient groups with the best of intentions.
"Taking money from industry prevents them from asking hard questions," she said.
The National Hemophilia Foundation (NHF), like nearly all hemophilia patient groups, gets money from companies including Roche to pay for education, research and programs.
But a spokesman said the group had strict rules that forbid advocating for a given product. "We are focused on ensuring patients receive access to the treatment that their physician determines to be the most appropriate," he said by email.
Michelle Rice, the NHF's head of external affairs and the mother of a hemophilia patient, said convincing everyone in the hemophilia community to quickly switch therapies will be difficult, despite benefits promised by new drugs.
That is in part because of the disease's tragic history. Many hemophilia patients died after acquiring HIV and hepatitis through tainted blood products in the 1980s.
"We lost about 10,000 people in our community," Rice said. "They're not inclined to just jump to the next bright shiny thing, unless they see a real potential advantage."
When her son was born 28 years ago, Rice said, there were just a handful of therapies available.
Now, there are some two dozen approved products, with more in the pipeline as hemophilia's potentially rich returns lure companies to pursue new approaches.
France's Sanofi (SASY.PA), which this year bought Biogen's (BIIB.O) hemophilia business, is working with U.S.-based Alnylam (ALNY.O). Novo Nordisk has an alternative to clotting factors in the works, too.
And experimental one-time gene therapies now being tested by BioMarin (BMRN.O), Spark Therapeutics (ONCE.O) and Sangamo (SGMO.O) would be very expensive, but could end indefinite infusions or injections.
This could threaten reliable cash flows for clotting factor makers as well as pose a challenge to Hemlibra.
Despite existing relationships between patients and companies, Spark's Chief Operating Officer John Furey said scientific and financial arguments should win the day.
If gene therapy helps patients and reduces overall costs for insurers, Furey said, "we'll be able to persuade the community this is a very good option for them."
(Reporting by John Miller; Editing by Mike Collett-White)