Congressman Leonard Lance

Representing the 7th District of New Jersey

ALTERNATIVE PRESS: Lance Pushes For System-Wide Changes to Treat Patients Faster, Better

Jan 30, 2012

Jackie Lieberman

Friday, January 27, 2012 • 5:16pm

WESTFIELD, NJ— Rare disease champion John Crowley, patients and advocates joined Congressman Leonard Lance (NJ-07) yesterday at a news conference at his headquarters in Westfield to discuss the MODDERN Cures Act. The act was recently introduced by Lance who, in the House of Representatives, serves as a co-chair of the bipartisan Rare Disease Congressional Caucus.

“I intend to be an advocate for better health care in this country,” said Lance, who told those present of how this act would update the nation’s drug and diagnostics evaluation and regulatory network, encouraging the discovery and development of new treatments for the many diseases that have few or no options. He said the legislation would also create a system that rewards efficiency and effectiveness to the benefit of all people with chronic diseases.  

Health care “Is not only a bipartisan issue. It is a nonpartisan issue,” said Lance. Although fighting between Republicans and Democrats is often in the news, “I’m here to accentuate the positive, ladies and gentlemen,” said Lance. “I am confident that this bill will receive wide bipartisan support.”

In addition to getting more treatments to patients and getting treatments to market faster, Lance said he hopes the day will come soon when we will see “customized, individualized therapies” for those who need them.

Lance was joined by advocates and patients representing several chronic and rare diseases, including ALS, Alzheimer’s, mesothelioma and osteoporosis, to discuss their support for the legislation. 

John Crowley, the chairman and chief executive officer of Amicus Therapeutics, a nationally recognized rare-disease advocate and father of two children with a rare genetic disorder, spoke. Crowley’s story served as the inspiration for the 2010 motion picture Extraordinary Measures.  

“There are seven thousand rare diseases just in the United States,” said Crowley. But though medical science has advanced and “we are really in the dawn of the new golden age of medicine,” he said, “We still develop these drugs in the same way as 40, 50, 60 years ago.”

This bill, he said, would change the regulatory system in a way that could speed treatments from the lab to the patients who need them. “It’s an incredibly important piece of legislation and one that I’m happy to support,” said Crowley.

Although the act could make it possible to change the way studies are done—especially on treatments for rare diseases, for which large populations often can’t be studied—“It will not affect the way the FDA approves something,” explained Lance. It will affect patents, however, making it more likely for a company to attain patents and make money from a rare-disease treatment it develops.

“We’re extremely grateful to Congressman Lance for introducing this,” said Debbie Schlossberg, patient service coordinator, ALS Association, Greater New York Chapter, serving Greater New York and Central New Jersey.

Bonnie Anderson of Berkeley Heights, a 10-year survivor of mesothelioma and advocate for the Mesothelioma Applied Research Foundation, noted that the legislation would enable trials to move faster so that patients in urgent need could get treatments sooner.

Sally Fullman of New Providence, a coordinator with the Healthy Bones Program for New Jersey Seniors with osteoporosis, thanked Lance for supporting not only New Jersey’s pharmaceutical companies but patients and consumers, as well.

“Obviously, we need to work together,” responded Lance, “With the pharmaceutical industry, medical device industry, advocates, patients and consumers.”

“One of the biggest challenges facing patients today is a lack of cures and treatments,” noted Eric Gascho, associate director, government affairs, National Health Council. Gascho hopes this legislation will help eliminate the barriers that keep treatments from reaching patients and give companies more incentive to bring products for patients with rare diseases to market.

Gascho said he hopes the act will improve not only the amount of drugs that go to market but also help doctors better predict which drugs will help which patient.

“It is, in my opinion, a civil right to be able to get the medicines that can help you,” said Lance. “This is the type of legislation that can help the federal government do a better job.”