COURIER NEWS: Lance introduces bill to streamline regulatory framework of treatments for rare, chronic diseases

January 27, 2012

WESTFIELD— Behind U.S. Rep. Leonard Lance, R-7th District, stood Bonnie Anderson of Berkeley Heights, who a decade ago was diagnosed with mesothelioma and told she had about three months to live.

Across a table from Lance sat Janet Palkewick of North Haledon, who 22 years ago while pregnant with her son Philip was diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, and told she had about two to four years to live.

Many others, Lance noted, aren’t as lucky.

It’s why the congressman conducted a Thursday morning news conference to kick off a campaign to promote his “modernize our drug and diagnostics evaluation and regulatory network” (MODDERN) Cures Act, a piece of legislation Lance and others said could dramatically streamline what they described as a complicated, time-consuming process through which treatments for rare and chronic illnesses are vetted and approved by the federal government.

“It is, in my opinion, a civil right to be able to get the medicines that can help you,” Lance said. “I’m not saying the federal government isn’t doing this; I’m saying the federal government can do a better job.”

On hand Thursday to help Lance hail the bill, which recently was introduced in the U.S. House of Representatives and now is before the Health Subcommittee, was John Crowley, chairman and chief executive officer of the Cranbury-based Amicus Therapeutics. Crowley was the inspiration for the 2010 film Extraordinary Measures”  — starring Harrison Ford and Brendan Fraser, it was based on Crowley’s real-life decade-long search to find a cure for Pompe disease, a rare degenerative disorder affecting two of his children.

More than 30 million Americans are affected by one of about 7,000 disorders classified by the medical community as “rare,” Crowley noted, adding that the regulatory framework under which new drugs and treatments are approved by the government has remained unchanged for decades despite dramatic advances in medicine occurring during that time.

“We are really at the dawn of a golden age in medicine now,” Crowley said. “We’ll beat nature ... what we have to beat is time.”

No one seemed to appreciate that notion more than Anderson and Palkewick.

“It’s wonderful,” Palkewick said softly from her wheelchair, a necessity for many patients suffering from the same chronic, fatal neurodegenerative disease. “We’re moving in the right direction.”

Anderson recalled being misdiagnosed with irritable bowel syndrome, or IBS, before doctors finally realized that washing her husband’s clothes after he came home from work with Exxon for years was exposing her to massive amounts of asbestos.

“When they opened her up,” Anderson’s husband John recalled, “they found 175 bits of asbestos in one square inch (of tissue).”

“I want everybody to know about this illness,” said Anderson, who during recent years has been active as head of the local chapter of the Mesothelioma Applied Research Foundation. “What’s hard,” she added, “is seeing and meeting these patients, then seeing them die every month.”

That, Lance said, was why he hopes his bill can “get the right medicines to the right people,” especially for those with “few or no options” on treatment.

Eric Gascho, director of government affairs for the Washington, D.C.-based National Health Council, praised the legislation as not reinventing the wheel – it doesn’t call for changes to the Food and Drug Administration’s approval process, for example. But he said the bill’s passage would implement a number of simple, common-sense changes to the rules.

The end result, officials said, would be part of what they envision as a general shift from more wholesale delivery of healthcare to personalized forms of treatment.

“Fifty years from now,” Lance said, “I’m sure people will be saying ‘My goodness! There was no customized care back then?”

Lance added that while his bill might not generate many headlines this year — he expects the national media to devote much of its time to more contentious matters and the presidential election cycle — he considers it one of the most important pieces of legislation he’ll vote on. Passage, he noted, could come as early as this summer if it receives the broad bipartisan support he expects.

“The national media accentuates the negative,” Lance said, his voice rising in volume dramatically. “I’m here to accentuate the positive.”

What you can do: The National Health Council has created an informational link on the “Modernize our drug and diagnostics evaluation and regulatory network” (MODDERN) Cures Act. For details, visit www.puttingpatientsfirst.net/moddern.