Should the FDA approve Vertex’s new cystic fibrosis drug?

The scene at the FDA hearing was familiar. All the  advisory committees have seen it play out again and again. Yesterday, it was the Pulmonary-Allergy Drug committee: The pharma doc with the convincing statement. The weeping patients. The drug likely to cost a couple thousand a month offering a slim  benefit over an existing drugs. But it works, and it’s safe.

From the Globe:VertexLogoSOP

“I think this is a much-needed advance for patients with cystic fibrosis,” said committee member Dr. Michelle S. Harkins, associate professor of medicine at the University of New Mexico Albuquerque, one of the majority voting to recommend approval.

The lone dissenter in the 12-1 vote recommending approval of the drug, Dr. Yanling Yu, the president and cofounder of Washington Advocates for Patient Safety in Seattle, said she was not convinced the data generated by the Vertex testing supported the approval of Orkambi.

“I really understand the patients critically need a new drug, but sometimes a new drug does not provide [the needed effectiveness],” she said.

From The New York Times story:

An issue for the advisory committee was that Orkambi had what the F.D.A. said was only modest effectiveness, improving lung function by only about 3 percentage points relative to placebo.

Some family members or advocates, some of them crying, pleaded with the committee to endorse the drug.

Some patients who took the drug in clinical trials said it had made a huge difference in their lives, reducing their coughing, allowing them to exercise better, helping them gain weight or reducing how often they ended up in the hospital…

Michael Yee of RBC Capital Markets, for instance, expects the price will be $225,000 to $250,000 a year.

The vote is advisory. The FDA staff will make the final call.

More here:

The financial stakes for Vertex.

FDA briefing for meeting.

Vertex briefing for the meeting

Yale’s Dr. Ross and the promotion of me-too meds

From ProPublica on me-too meds. Yale’s Dr. Joseph Ross gets a quote in this story. , Vox offers a summary of his NEJM piece on digital marketing to docs. More here on his work into the accuracy of clinical trial registries.

Here’s the NEJM abstract: Pharmaceutical marketing can lead to overdiagnosis, overtreatment, and overuse of medications. Digital advertising creates new pathways for reaching physicians, allowing delivery of marketing messages at the point of care, when clinical decisions are being made.

From ProPublica

Vying for Market Share, Companies Heavily Promote 2018 Me Too’ Drugs

by Charles Ornstein and Ryann Grochowski Jones ProPublica, Jan. 7, 2015, 2 p.m.

propub logoThis story was co-published with the New York Times’ The Upshot.

For more than five decades, the blood thinner Coumadin was the only option for millions of patients at risk for life-threatening blood clots. But now, a furious battle is underway among the makers of three newer competitors for the prescription pads of doctors across the country.

The manufacturers of these drugs 2014 Pradaxa, Xarelto and Eliquis 2014 have been wooing physicians in part by paying for meals, promotional speeches, consulting gigs and educational gifts. In the last five months of 2013, the companies spent nearly $19.4 million on doctors and teaching hospitals, according to ProPublica’s analysis of federal data released last fall.

The information, from a database known as Open Payments, gives the first comprehensive look at how much money drug and device companies have spent working with doctors. What it shows is that the drugs most aggressively promoted to doctors typically aren’t cures or even big medical breakthroughs. Some are top sellers, but most are not.

Instead, they are newer drugs that manufacturers hope will gain a foothold, sometimes after failing to meet Wall Street’s early expectations.

“They may have some unique niche in the market, but they are fairly redundant with other therapies that are already available,” said Dr. Joseph Ross, an associate professor of medicine and public health at Yale University School of Medicine. “Many of these, you could call me-too drugs.”

In almost all cases, older, cheaper products are available to treat the same conditions. Companies typically try to differentiate the new drugs by claiming they are easier to use; carry fewer side effects; work faster than competitors; or have medical advantages.

The makers of Pradaxa, Xarelto and Eliquis, for example, say their drugs are at least as effective as Coumadin for certain conditions but do not require routine blood tests or limitations on what patients can eat. (Patients taking Coumadin, also known as warfarin, shouldn’t eat grapefruit or cranberries and have to limit green leafy vegetables in their diet.)

Officials at the Centers for Medicare and Medicaid Services, which administers Open Payments, and the Pharmaceutical Research and Manufacturers of America, the drug industry trade group, said they had not analyzed the data in order to rank spending by drug.

When told of ProPublica’s analysis, John Murphy, PhRMA’s assistant general counsel, said drug makers’ spending should be seen not only as a marketing strategy, but also as a way of ensuring the best treatment options for patients. “On paper, a drug may not look like it is monumentally better than another drug, but to an individual patient, it might be,” Mr. Murphy said.

* Note: General Payment figures do not include royalties. Source: Centers for Medicare and Medicaid Services, Food and Drug Administration, ProPublica reporting

According to ProPublica’s analysis, Victoza, a diabetes medication made by Novo Nordisk, was the drug associated with the most payments to doctors, by dollar amount. The company spent more than $9 million on physician interactions related to Victoza in the last five months of 2013, excluding research payments and royalties, which relate more to drug development than marketing. (ProPublica created a tool that lets you look up any drug, device or company and compare it with any other.)

Victoza, through a once-a-day injection, helps lower blood sugar among diabetics, but researchers and advocacy groups have said drugs of its class carry an increased risk of thyroid cancer and pancreatitis. Dr. Todd Hobbs, chief medical officer of Novo Nordisk in North America, said the company’s spending reflected Victoza’s newness and the need to address such safety concerns.

“We just received a huge amount of interest and questions and need for education,” Hobbs said, referring to inquiries by health care professionals, particularly primary care doctors. “You see the fruits of that in this report.”

Eliquis, the anticoagulant jointly marketed by Bristol-Myers Squibb and Pfizer, ranked second in its link to spending on physicians, with nearly $8 million, our analysis showed. In a statement, the companies said their spending helps ensure physicians understand the appropriate use of Eliquis. Because the drug is prescribed by physicians in different specialties, the statement said, “it is critical to have a speaker program that adequately provides robust education to these physicians.”

The drug associated with the third-most payments to doctors was Brilinta, a different type of blood thinner made by AstraZeneca that vies for sales with Plavix, which is now available generically. In an email, AstraZeneca said it had identified Brilinta as one of its “key platforms for growth” and increased speaker and research spending on it. “Physicians are also indispensable partners in our efforts to bring new medicines to patients,” the company said.

ProPublica has tracked drug companies’ payments to doctors since 2009 through a searchable database called Dollars for Docs. But this covers only 17 companies, most of which have been compelled to release this information under legal settlements with the government. It has no information from medical device makers.

The list of most promoted drugs featured many recent arrivals: 14 of the top 20 were approved by the Food and Drug Administration since 2010. Some treat similar conditions, including diabetes, schizophrenia and chronic obstructive pulmonary disease, so the competition among them is fierce. “They’re fighting over the same doctors, I guarantee you,” said Rhonda Greenapple Simoff, founder of a consulting firm that advises pharmaceutical companies in Bernardsville, N.J.

Largely absent from the top of the list were drugs that cure disease, such as a new class of hepatitis C treatments, or those that significantly extend life, particularly for cancer patients. If a drug is either the first to treat a disease or is much better than existing drugs, said Dr. Sidney Wolfe, the founder and now senior adviser to Public Citizen’s Health Research Group, “they ‘sell themselves’ on the merits of their unique benefits.”

According to ProPublica’s analysis, a few of the most heavily promoted drugs, including Samsca, which treats low sodium levels in the blood, have serious side effects that came to light after their approval by the federal government. The manufacturers of several others, including Copaxone, Latuda, Xarelto, Daliresp and Humira, have been faulted by the F.D.A. for improper promotion.

Subsys, approved in 2012 to treat cancer pain, ranked 23rd in spending on doctors. It’s often prescribed for off-label, or unapproved, uses; in November, The New York Times reported that some of the doctors paid the most to promote the drug had disciplinary or legal troubles. In a statement to The Times, Insys Therapeutics, the drug’s maker, said its marketing of Subsys was appropriate.

The medical device associated with the most payments to doctors was Intuitive Surgical’s da Vinci surgical robot system, which the company has marketed as an effective, less invasive option for an array of procedures. Critics have complained that the device is needlessly expensive and overused, and say it has been linked to patient complications and deaths.

Intuitive spent nearly $12.8 million on physician interactions to promote the robot in the last five months of 2013, not including royalties and research. The spokeswoman Paige Bischoff said in an email that about half of the company’s outlays for education and training were “pass through” spending: Surgeons or hospitals paid the company for services, and the company, in turn, paid doctors to provide them.

Dr. Robert Takla, an emergency room physician in the Detroit area, earned about $75,000 in the last five months of 2013 by delivering promotional talks about several of the most heavily marketed anticoagulants and blood thinners, particularly Brilinta, according to Open Payments.

He said he enjoys speaking on behalf of companies and thinks he offers a different perspective than cardiologists and internists 2014 the usual prescribers of the drugs 2014 because he treats complications of blood clots in the emergency room.

Dr. Takla said he reviews clinical studies before deciding to speak for a drug and turns companies down when he isn’t impressed. He said he no longer spoke on behalf of Pradaxa because of what he characterized as public backlash against it, driven by a spate of lawsuits against its manufacturer, Boehringer-Ingelheim. (The company agreed to pay $650 million last year to settle the suits.) He accepts fees to speak about Xarelto, a drug he has taken himself for a deep vein thrombosis.

“It’s a very fertile and very robust marketplace right now,” he said of the anticoagulants.

News applications developer Mike Tigas contributed to this report.

Methodology: How we calculated company payments to doctors

ProPublica is a Pulitzer Prize-winning investigative newsroom. Sign up for their newsletter.

A closer look at the downside of the Vertex – Cystic Fibrosis Foundation deal

And some comments from the Knight Science Journalism Tracker: 

A new cystic fibrosis drug–the first to be developed based on understanding of the genetics of the illness–is an orphan drug that can help only about 4 percent of patients with cystic fibrosis. Its financial toxicity is such that it can destroy the finances of those whose lives it saves: It costs $307,000 per year. And it sits in the center of a swarm of conflicting interests involving a pharmaceutical company, a non-profit patients’ organization, and researchers who have taken their money

This comes from John Fauber of the Milwaukee Journal-Sentinel, whodescribes a disturbing case 

VertexLogoSOP

Click here for the MJS story:

What happens when a disease-fighting charity dives into venture capitalism?

In the first case of its kind, the results include one of the planet’s most expensive pills, huge sales projections for a drug company and windfalls for executives who sold stock in the glow of enthusiastic news releases abo

ut the drug.

downloadKalydeco is a breakthrough drug designed from knowledge of the genetic roots of cystic fibrosis, a lung disease that kills most victims before they reach middle age. Developed by Vertex Pharmaceuticals with a $75 million investment from the Cystic Fibrosis Foundation, it is an early example of “venture philanthropy,” where a nonprofit helps finance development of a treatment in return for a cut of sales.


Yet it costs each patient $307,000 a year to take two Kalydeco pills a day – a price borne by taxpayers through Medicaid and other government programs and by the workers and companies who finance employee health insurance plans.
Much of the initial science behind Kalydeco involved nonprofit research universities and hospitals, with funding from the Cystic Fibrosis Foundation and by taxpayers through the National Institutes of Health.

In 2012, with less than a full-year on the market, Vertex sold $172 million worth of Kalydeco and the foundation cashed in by selling future royalties from the drug to an undisclosed firm for $150 million. The foundation is investing that money with Vertex and other companies to develop more new drugs.

For more on the links between patient advocacy groups and drug companies, see TR’s  2006 Washington Post story:

Some groups’ Web sites feature the names of their corporate donors, but often these names appear only in an annual report. Exact donation amounts are rarely listed, though some groups are willing to provide numbers on request.

People who look to these groups for independent information may never learn about their financial ties to drug makers. That needs to change, say some consumer advocates, now that nonprofits are playing a larger role in drug safety debates. When disease advocacy groups weigh in on a drug up for review by the FDA’s Drug Safety and Risk Management Advisory Committee, they say, the public may be unaware of potential conflicts of interest.

 

Will competition in Hep C drug market bring down costs? #hepatitis

Lots of health news emerges from investor events like this week’s J.P. Morgan Healthcare Conference. The Globe’s Robert Weisman reports this morning on competition for Cambridge-based (soon to be Boston-based) Vertex Pharmaceuticals.

SAN FRANCISCO – It was supposed to be a victory lap for Vertex Pharmaceuticals Inc. executives: their first appearance at the life sciences industry’s most important annual conclave since the Cambridge biotechnology company won long-sought approval of its potential blockbuster drug to treat hepatitis C.

But the Vertex team, including the departing chief executive and his newly appointed successor, was partly upstaged by an announcement on the eve of the 30th annual J.P. Morgan Healthcare Conference that the giant drug maker Bristol-Myers Squibb Co. was entering the hepatitis C market.

Bristol-Myers said it will spend $2.5 billion to buy Inhibitex Inc., which is developing a next-generation hepatitis C treatment that will compete with two being developed by Vertex.

As he notes, the company’s stock price has dropped from $58.87 in May to $35.68 yesterday.

Will competition also bring the price of Telaprevir down?  The Globe reports that the drug will cost between $30,000 and $50,000 per treatment.

In the meantime, the company is building a new waterfron tower.The Globe also reports that the new headquarters will cost $2 billion. Boston will kick in a $11.8 million tax break, for the company, which is now located in Cambridge.

Radio Boston: The Influence of Pharma on Docs

WBUR’s Radio Boston takes on the ever-present issue of the influence of drug makers on docs. Listen on their web site or today at 3. Guests include:

Jerry Avorn, M.D., PChief of the Division of Pharmacoepidemiology and Pharmacoeconomics in the Department of Medicine at Brigham and Women’s Hospital and author of Powerful Medicines: The Benefits, Risks and Costs of Prescription Drugs.

Leslie Jackowski, M.B.B.S. (M.D.), Exeuctive Director for Program at the ALOSA Foundation a nonprofit organization “dedicated to the dissemination of accurate, unbiased, evidence-based and non-commercial information about medications for health care professionals and patients by providing educational services (interactive outreach to physicians, classes, presentations, seminars, conferences) about the development, regulation, risks, and benefits of prescription drugs.” In other words, they come in after the drug companies pitch their drugs and give other docs more objective information about meds.  The scientific jargon for this is “counter-detailing,’ a reference to the term “detail man” used to describe drug reps who do direct sales to docs. That term is no longer relevant, especially since many detail men are now young, attractive women.

Thomas P. Stossel, M.D.,  American Cancer Society Professor of Medicine atHarvard Medical School, Director of the Translational Medicine Division at Brigham & Women’s Hospital, Founder of the Association of Clinical Researchers and Educators. For more on ACRE, see the BHN report on the group’s inaugural meeting, as well as a comment from former NEJM editor Arnold Relman. (Brother John Stossel, a crusading TV journalist who often takes on what he describes as “junk science,” just made the leap from ABC to Fox. )

Georgia Maheras,  Esq., Private Market Policy Manager at Health Care for All

Partners limits questionable moonlighting for docs

The New York Times reports:

Senior officials at the two hospitals, Massachusetts General and Brigham and Women’s Hospitals in Boston, must limit their pay for serving as outside directors to what the policy calls “a level befitting an academic role” — no more than $5,000 a day for actual work for the board. Some had been receiving more than $200,000 a year. Also, they may no longer accept stock.

Criticism has been mounting in recent years as the conflicting roles of some medical leaders have been disclosed through Congressional investigations, lawsuits and reports in the news media. Those disclosures have raised questions about bias and the cost and quality of patient care at the nation’s medical institutions.

enior faculty in leading teaching hospitals are in high demand on medical product boards, but corporate filings show that Partners and Harvard Medical have a disproportionate share.

The story points out that Dr. Samuel O. Thier was president of Partners when he was named to the Merck board in 1994. He is now retired from Partners.

Dr. Joseph B. Martin, dean of the Harvard Medical School from 1997 to 2007, was named to the board for Baxter International in 2002. Dr. Thier and Dr. Martin each receive over $200,000 a year from the corporate boards.

Dr. Martin, a professor of neurobiology, declined to comment. Dr. Thier did not return calls seeking comment.

For more on medical conflict of interest and research integrity, click here.

Lilly funds 60 Boston docs

From The Boston Globe:

lilly logoAt least 60 Massachusetts doctors collectively have earned more than a half-million dollars this year as speakers paid by pharmaceutical giant Eli Lilly & Co. – including two Boston Medical Center physicians whose participation is being reviewed for possible violation of a hospital policy against marketing activities by its doctors.

After learning of the doctors’ company-sponsored talks from the Globe, Boston Medical Center said it would investigate the matter and directed the physicians not to make any further presentations on behalf of Lilly in the meantime.

So much for internal disclosure. More from the Globe:

bmcLogo1[1]The use of physicians in speakers programs or “bureaus’’ like Lilly’s, in which doctors generally use company-prepared materials to explain a drug’s uses and dosing to their colleagues, is widespread in the drug industry. But the practice is under growing scrutiny and some academic medical centers are barring their doctors from participating, believing that physicians essentially become hired advertising guns, with weakened credibility.

10/1 update  The Wall Street Journal reports on new industry authorship guidelines from PHRMA.

 Here are a few interesting details, followed by a big grain of salt:… The guidelines are purely voluntary.

Also see today’s Globe editorial and letters both for and against industry support.

 WCVB Channel 5 in Boston also reports.

 Health reformers at Boston’s Health Care for All commented on their blog. So did Gooz News. 

 Here’s the Lilly list of company funded docs.

 Note that Lilly was not a big supporter of the state’s ban on pharma junkets for docs. From the Boston Business Journal.

 The head of pharmaceutical giant Eli Lilly & Co. on Friday blasted a new law governing how drug companies market to physicians, calling it a bad move that will hamper innovation and force companies to reconsider expanding in Massachusetts.

Still, it didn’t stop the biotech industry from planning their annual meeting here — for the second time in three years — as announced yesterday.

For more on critics of pharma sponsorship of doctors, see the Integrity in Science Project.   

For more on docs who want to be free to work closely with industry, see the webcasts and slides from July’s inaugural meeting of The Association of Clinical Researchers and Educators (ACRE) an “organization of medical professionals who recognize that appropriate physician-industry collaborations and relationships benefit patients and advance science.”  Also note BHN’s reporting from the meeting, which took place at Brigham and Women’s Hospital.   

 At that meeting, Dr. Carey D. Kimmelstiel,head of clinical cardiology at the Tufts University School of Medicine, talked about the benefits of having clinicians give industry-sponsored talks. Preparing the talk educates the speaker. He or she gets feedback and an audience of busy docs gets a quick update. He did say some of the transparency are good because it discourages clinicians from showing company-produced slides without really understanding them.

 But he believes the law has constrained continuing medical education by limiting the “the only reliable source of such funding, which is industry.”  

 Click here for his full statement and all of the presentations at the ACRE meeting.

9/30 The Wall Street Journal reports on new industry authorship guidlelines from PHRMA.

 Here are a few interesting details, followed by a big grain of salt:… The guidelines are purely voluntary.

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