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Shortage of a drug needed to treat Axel Zirbes’ leukemia sent his mother, Justine, on a far-and-wide search for a supply.

Richard Tsong-Taatarii, Star Tribune

Congress acts to ease shortages of vital drugs in FDA overhaul

  • Article by: KEVIN DIAZ and JIM SPENCER
  • Star Tribune staff writers
  • May 24, 2012 - 11:53 PM

WASHINGTON - Axel Zirbes' doctor said his leukemia required cytarabine, a standard chemotherapy drug that attacks cancers of the white blood cells. But when the 5-year-old Crystal boy was ready for treatment, doctors told his parents that supplies were running out.

That set the family on a nationwide scramble to find a dose -- a quest repeated by thousands of other families in Minnesota and around the country. It also touched off pleas for help to lawmakers like U.S. Sen. Amy Klobuchar, D-Minn., one of several who took up their case in legislation approved Thursday in the Senate.

The provision, which requires drugmakers to issue warnings of looming drug shortages, also has been written into a bill expected to clear the House next week.

The legislation is part of a sweeping overhaul of the Food and Drug Administration (FDA) that aims to speed approval of new drugs and medical devices in an industry with a deep corporate imprint in Minnesota.

The Senate vote was 96 to 1. At the same time, senators rejected a measure championed by Sen. Al Franken, D-Minn., that would have allowed imports of low-cost Canadian prescriptions.

Pressure for Klobuchar's provision has been building for months, as spot drug shortages kept cropping up around the country.

"It's mind boggling," said Axel's mom, Justine Zirbes, who found herself working a network of cancer moms from Oklahoma to Canada last year in a search for her son's cancer drug. "It's enough to have to deal with the fear and unknowns of cancer in your child. But then to be told the drugs are unavailable is just unacceptable."

Doctors and industry experts say economics drives many of the shortages. Manufacturers have been reported to cut back on drugs that don't earn a sufficient profit, especially generics. State and federal prosecutors also have accused drugmakers of "pay for delay" deals that pay manufacturers of generic drugs to keep cheap alternatives off the market.

Manufacturers point to regulatory burdens, the rapid growth in demand for new drugs and occasional production bottlenecks.

The Pharmaceutical Research and Manufacturers of America (PhRMA), the industry's main lobby, issued a statement Thursday saying the causes are "complex and multidimensional."

It also said manufacturers have stepped up the voluntary reporting of anticipated shortages, helping prevent 195 shortages last year.

But critics point out that the vast majority of the shortages involve low-profit generic drugs, suggesting financial forces are at play. "Have you ever heard of a shortage of Lipitor or Viagra?" said Zirbes, referring to the popular cholesterol and erectile dysfunction brands.

A rare bipartisan majority including Klobuchar, Franken and Minnesota Republican U.S. Rep. Erik Paulsen has lent solid support to a provision requiring prescription drug manufacturers to give early notification to the FDA of any event that would likely result in a drug shortage.

The Senate bill approved Thursday also includes Klobuchar's provision banning dangerous synthetic drugs such as 2C-E, which has led to the death of at least one Minnesota teenager and hospitalized several others.

'This is crazy!'

Obama administration officials say voluntary compliance under an executive order issued six months ago has helped prevent 128 drug shortages; they say the number of drugs in shortage has fallen to 42 so far this year, compared to 90 at this time last year.

But critical drug shortages continue to plague hospitals, clinics and pharmacies. By some estimates, the number of drugs in short supply has tripled since 2005.

Mary McHugh Morrison, an Edina woman who takes Doxil to combat peritoneal cancer, also was among those forced to go out on her own to find limited supplies around the nation. "I was shocked," she said. "I'd never heard about a shortage."

A physician told her the growth of one of her tumors was very likely the result of a delayed dosage during her hunt for a new supply. And she's hardly the only one, according to her doctor.

"For the first time in my career, we've had to not give patients a drug that was working for them and watch their cancer grow," said Dr. Paul Haluska, a Mayo Clinic oncologist in Rochester. "I don't know why people aren't running out in the streets and screaming 'This is crazy!'"

That view is seconded by Franken. "A shortage of any drug keeping people alive is unacceptable, especially when that shortage could be prevented," he said.

'Not a silver bullet'

If lawmakers think they've found a solution, some industry experts say early reporting requirements for potential shortages could actually make the situation worse, not better.

"Profit margins on generic drugs are tiny," said Shaye Mandle, a vice president for government relations at Minnesota's medical technology advocacy group LifeScience Alley. "It's very different from brand name drugs."

That is because they operate on a business principle called "just in time supply," or making what's needed when it's needed and not maintaining big inventories.

"Generic drug manufacturers may not know six months in advance if they're going to have a shortage," Mandle said. "One thing is for sure: When you announce that you're going to have a drug shortage, there's going to be a run on that drug."

Shortages also result from regulatory barriers and scarce resources, Mandle said. They can also occur in intervals when one manufacturer stops production of a drug and another starts making it.

"There is not," he said, "a silver bullet solution."

While the pharmaceutical industry wrestles with drug shortages, medical device makers hailed the new FDA regulations in the Senate bill as a better way to get more innovative products to patients.

Device makers agreed to double the user fees they pay the government in order to hire more device reviewers and speed the FDA approval process, said Steve Ubl, president and CEO of AdvaMed, the device industry's major trade association.

But cancer moms like Justine Zirbes are most grateful for the part of the bill addressing drug shortages. "I felt like my child's life was in the hands of these drug companies," she said, "and there was nothing I could do about it."

Kevin Diaz is a correspondent in the Star Tribune Washington Bureau.

DRUG SHORTAGE BILL OVERVIEW

• Speeds federal approval of lifesaving medicines, including lower-cost generics.

• Requires makers of drugs and medical devices to pay $6.4 billion to finance product evaluation by the FDA.

• Requires drugmakers to notify the government of potential drug shortages.

• Federal officials would help find other sources of supply.

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