The House of Representatives has passed legislation that could pave the way for more treatment options for people who are ill, and the bill’s bipartisan support bodes well for its passage in the Senate, said Congressman Gus Bilirakis, who has taken a leadership role on this issue.
The 21st Century Cures Act passed the House by a vote of 344-77 on July 10.
“Now, we’re waiting on the Senate to pass a bill,” Bilirakis said, and he’s confident that will happen, perhaps by the end of the year.
Passage can’t come soon enough, Bilirakis said, noting the legislation offers hope to people who are desperate for new treatment options.
The Senate’s version of the bill will likely have fewer provisions, Bilirakis said.
To help protect provisions he believes are important, Bilirakis has sponsored stand-alone bills, as well.
One of the stand-alone bills he has introduced is called the Orphan Product Extensions Now Accelerating Cures & Treatments Act (OPEN ACT). That calls for giving drug makers and innovators incentives to “repurpose” major market drugs for life-threatening rare diseases and pediatric cancers.
The aim is to open the door to the development of hundreds of safe, effective and affordable treatments for rare disease patients, Bilirakis said.
Sen. Orrin Hatch, of Utah, has co-sponsored that bill in the Senate.
Bilirakis said he doesn’t care whether these provisions are adopted in the overall legislation or his stand-alone bill. He just wants action on these issues.
While legislators were drafting the 21st Century Cures Act, roundtables were held around the country to give stakeholders a chance to discuss issues being faced by patients and providers.
Bilirakis convened roundtables in Lutz, Land O’ Lakes and Carrollwood, where he listened to hours of testimony from patients, patient advocates, doctors, researchers, makers of medical devices and other stakeholders.
The settings for the sessions were comfortable meeting rooms, but the testimony delivered dealt with issues of life and death, and was often poignant and dramatic.
At a session in Lutz, patients told Bilirakis about difficulties in getting a proper diagnosis and finding effective treatments.
Ashleigh Pike was one of those patients.
Before she became ill, she was a vibrant young woman who delighted in teaching elementary school children. Now, she lives life from a wheelchair and requires full-time care.
“On the outside, I look fine,” the former teacher told Bilirakis. But, she added: “My quality of life has been greatly compromised.”
Pike suffers from a form of dysautonomia, a malfunction of the autonomic nervous system. That system controls automatic functions of the body, such as heart rate, blood pressure, digestion, kidney function, temperature control, and dilation and constriction of the pupils, according to Dysautonomia International’s website.
At another session, health care providers told Bilirakis about a variety of roadblocks that frustrate their work.
Dr. David Morgan, the chief executive of the University of South Florida’s Byrd Alzheimer’s Institute, told Bilirakis the diagnosis of Alzheimer’s patients must improve. About one in five people diagnosed with Alzheimer’s do not have the disease, Morgan said.
The disease can be accurately diagnosed with PET — positron emission tomography — scans, but those are expensive and generally not covered by insurance, Morgan said.
Proper diagnosis is important not only for treatment of patients, but also to ensure that clinical trial results are not skewed by including patients in the trials who do not have the disease.
Reforms also are needed in the way clinical trials are conducted, Morgan said, noting the current approach takes too long and costs too much.
At that same session, Dr. Richard Finkel, chief neurologist at Nemours Children’s Hospital of Orlando, told Bilirakis that the focus must be greater on patient-centered cures.
“Patients are willing to accept different levels of risk. But the FDA (Food and Drug Administration) doesn’t look at it that way,” he said. “They are very risk averse.”
Medical device makers face other obstacles, Lisa Novorska, chief financial officer for Rochester Electro Medical Inc., told Bilirakis.
Her company can know how to improve a device, but can’t pursue those improvements because of the costs to comply with FDA requirements. The FDA plays a valuable role in protecting the public, but it also creates paperwork nightmares for small businesses, she said.
Bilirakis said The 21st Century Cures Act addresses many concerns raised during the roundtable sessions, and provides an $8.5 billion increase for the National Institutes for Health.
Published July 29, 2015