Viewpoints: Health Costs At Home And Abroad; Candidates' Health Policy Details; VA And Suicides

A selection of opinions on health care from around the country.

Viewpoints: 'Public Option' Doesn't Work; Developing A Zika Vaccine; Pockets Of Innovation

A selection of opinions on health care from around the country.

A Golden Ticket That Fast-Tracks A Drug Through The FDA

Drugmaker Sarepta Therapeutics won a big victory when its $300,000 muscular dystrophy drug was recently approved, but the company had other reasons to celebrate, too.

They were also awarded the drug world's equivalent of a Willy Wonka golden ticket.

The ticket, known as a rare pediatric disease priority review voucher, is part of a program created by Congress in 2007 to encourage the development of drugs for tropical diseases and later expanded to rare pediatric disorders. Any company awarded a voucher can use it for a fast-track government review of one of its future drugs - or it can sell the voucher to another company.

“The only people who would buy a priority review voucher would be someone who had something that wouldn't merit its own priority review but they want the priority review,” says Dr. Tim Coté, a former FDA official who now runs a consulting firm for rare disease drugs.

In other words, the companies willing to pay for a voucher are most likely trying to get a medicine that treats a common disease on the market quickly for competitive reasons, he said.

“It might be a blockbuster - say a new statin,” Coté said, referring to a drug that lowers cholesterol. “A priority review would make all the difference in the world.”

A voucher guarantees a company that its drug will be reviewed within six months, as compared to the standard 10. But approval isn't assured.

This KHN story also ran on NPR. It can be republished for free (details).

And the sale of one voucher can earn hundreds of millions of dollars for a small company like Sarepta. Last year, a voucher sold for a record $350 million when AbbVie Inc. bought one from United Therapeutics. The company has not announced plans for the voucher.

It's those price tags - as well as admonishments from the Food and Drug Administration - that have some industry watchers debating whether the program should continue. The House voted Tuesday to extend the program until the end of the year, after the Senate had already done so. It was set to expire this month.

Members of Congress, the pharmaceutical industry, and rare disease advocates have passionately supported the voucher program as a way to incentivize rare disease drug development. The goal, they say, is to steer more money toward rare disease drug developers by allowing them to sell the voucher to the highest bidder. But others, such as FDA officials and academics, have questioned whether the program is leading to any new drugs for rare diseases.

Nancy Goodman, executive director of Kids v Cancer, is a champion of the bill. She created her foundation after her son died seven years ago of medulloblastoma, a rare pediatric brain cancer.

There were no drugs available for her 10-year-old son, she said.

“I believe and hope we will see a whole crop of pediatric disease drugs because of the program,” Goodman said.

The legislation extending the program passed the House and Senate by unanimous consent and awaits action by President Barack Obama, who is expected to sign it. Advocates are fighting to keep the program authorized until Dec. 21, 2018 as part of the 21st Century Cures Act, which has passed the House and has stalled in the Senate, where backers hope it will be taken up after the elections.

But officials at the FDA, which must implement the vouchers, are at odds with industry and advocates.

Dr. John Jenkins, director of the office of new drugs in the Center for Drug Evaluation and Research at the FDA, has said he supports the overall goal of providing incentives to promote drug development but that the voucher program is wrong.

In December, Jenkins told a reporter at the industry publication Pharmaceutical Executive that vouchers raise safety concerns because the program requires the FDA to give any drug accelerated review - even when reviewers have to address complex issues.

“We're not making pizza here,” he said.

Jenkins declined interview requests for this story. FDA spokeswoman Sandy Walsh said, the “FDA has not seen evidence that the program is effective.”

Walsh also said the agency is concerned that the voucher program “adversely affects the agency's ability to set its public health priorities” and “the additional workload from the program strains the agency's resources.”

The voucher program does require companies to pay a user fee of nearly $3 million to help the agency pay for the program. But the additional money may not be sufficient.

In March, a Government Accountability Office report said FDA officials complain there is too little time to use the money to hire and train additional employees to do accelerated reviews and that the one-time fee does not enable hiring of long-term staff.

The GAO's report said it was too early to say whether the pediatric priority review voucher program, approved in 2012, is stimulating development of drugs.

Of the seven pediatric review vouchers that have been awarded, four have been sold to other drug companies.

Drugmaker Sanofi has redeemed two of the vouchers. For the first, they paid $67.5 million and used it to expedite review of their cholesterol drug Praluent. Later, Sanofi spent $245 million for a voucher and said in February it would be used to speed up the FDA's decision on a new type 2 diabetes treatment.

If a voucher is not used, it can be sold an unlimited number of times before being used.

Sarepta plans to sell its voucher, Chief Financial Officer Sandy Mahatme told Wall Street analysts in announcing approval of its drug to treat Duchenne muscular dystrophy. He said the money would help finance other drugs in its pipeline, pay for the scaling up of its manufacturing and support the company's entry into European markets.

Mahatme said they have already reached out to “a bunch of potential buyers.”

KHN's coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Viewpoints: Recognizing The Superbug Danger; Confronting Mental Health Stigma

A selection of opinions on health care from around the country.

When Drug Reviewers Leave The FDA, They Often Work For Pharma

More than a quarter of the Food and Drug Administration employees who approved cancer and hematology drugs from 2001 through 2010 left the agency and now work or consult for pharmaceutical companies, according to research published by a prominent medical journal Tuesday.

Dr. Vinay Prasad, a hematologist-oncologist and assistant professor at Oregon Health and Science University, sought to understand the so-called “revolving door” between the FDA and the pharmaceutical industry, which he said is often discussed but hadn't been quantified.

“We all know about these anecdotal cases” of a person who was  “often a major player at the FDA, someone in an important role - and then they leave the FDA and go and work for industry,” Prasad said, but he couldn't find anyone who knew whether this happened “5 percent or 60 percent” of the time.

Use Our Content

This KHN story can be republished for free (details).

Prasad and his colleague Dr. Jeffrey Bien, an internal medicine resident also at Oregon Health, tracked 55 FDA reviewers in the hematology-oncology field from 2001 through 2010, using LinkedIn, PubMed and other publicly available job data. They found that of the 26 reviewers who left the FDA during this period, 15 of them, or 57 percent, later worked or consulted for the biopharmaceutical industry. Put another way, about 27 percent of the total number of reviewers left their federal oversight posts to work for the industry they previously regulated. They published their findings in The BMJ as a research letter.

Going to work for industry after leaving the FDA is not inherently bad, but it does raise some questions.

“If you know in the back of your mind that your career goal may be to someday work on the other side of the table, I wonder whether that changes the way you regulate,” Prasad said. “Are you more likely to give [companies] the benefit of the doubt? Are you less likely to beat them up hard over [using bad comparisons in drug studies]?”

Prasad focused his research on his own field - hematology-oncology - because it spawns a large number of new drugs and reviewers have a lot of autonomy, he said.

“There's a lot of room for interpretation in deciding whether or not a cancer drug should be approved,” he said, because so many studies of cancer drugs rely on what's called a “surrogate endpoint,” meaning that something other than survival or quality of life was measured to determine whether a drug worked. For example, shrinking a tumor may be a stand-in for survival. But according to one of Prasad's previous studies, there isn't always evidence that surrogate endpoints are linked to better health outcomes, suggesting that some approved drugs aren't as beneficial as they appear.

“Sometimes, the public needs [the FDA reviewers] to be firm. If they're not, no one else in the health care sector is going to be,” Prasad said, adding that once the agency approves a drug, the Centers for Medicare & Medicaid Services has to cover it and can't negotiate prices under current laws. “The FDA is often the only real wall between ineffective, harmful drugs and patients.”

He and Brien tracked reviewers instead of higher ranking officials because reviewers provided a larger sample size, Prasad said. They would have liked to include reviewers named on denials as well, but the denial documents are secret, making it impossible to identify the reviewers.

Although Prasad said FDA reviewers have a lot of power over approvals, Dr. Joshua Sharfstein, the FDA's principal deputy commissioner until 2011, disagreed.

“There are just so many checks and balances within the review process that it's really not up to one person by and large,” said Sharfstein, now an associate dean at the Johns Hopkins Bloomberg School of Public Health in Baltimore. “Key regulatory decisions are looked at from many different angles. I think it would be very difficult for an individual to do something inappropriate and not have that caught.”

Sharfstein said he has met with companies to offer advice, but was not compensated for it.

The revolving door is a fair topic to study, he said, but having former FDA officials on the pharmaceutical industry payroll can have a public health benefit. Former FDA employees with deep knowledge of the approval process help make it go smoother by ensuring all the relevant research is complete and the latest pathways to approval are understood.

“Companies are very nervous about the FDA,” he said. “So they need to have an understanding of how the FDA works.”

FDA spokesman Jason Young said employees leave the government to work for industry at various agencies, not just the FDA.

“The FDA has a strong set of rules in place to ensure that our employees are working in the public interest, not to advantage any company, organization or individual,” he said, adding that these include protecting confidential information they learned at the FDA and a “cooling-off” requirement for senior officials before they can work for industry.

Former FDA Chief Dr. Margaret Hamburg, who left the administration last year, told Stat News in March that she would wait before jumping into an industry job, adding that “this perception of the revolving door is damaging to everyone” and that she would not consider “any boards of any company big or small that was regulated by the FDA for a couple of years.” She also is quoted as saying it is “unfortunate” that people think “a complete division” is necessary between the agency and the industry.

The FDA Alumni Association, whose slogan is “serving those who have served,” currently lists four job openings for former FDA employees. Three are at consulting firms and one is at a law firm seeking an “FDA compliance paralegal.” Of the consulting jobs posted, one is for a regulatory scientist and the other is for a regulatory toxicologist. The third is a food and cosmetic consulting job.

KHN's coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Viewpoints: House Calls - A Better Idea Than Ever; Is The FDA Tightening Hold On Generics?

A selection of opinions on health care from around the country.

Viewpoints: Obama Needs To Lead On Cures Act; Pricing Drugs To Value; Preventing Alzheimer's

A selection of opinions on health care from around the country.