Ever since his uncle died at age 57 from Parkinson's disease, Congressman Gus Bilirakis (R-FL) has been passionate about health care issues in general and neurology in particular. His memories of seeing his once-vibrant uncle waste away after being diagnosed in his late 40s are still sharp.

"Neurologic disease is very personal to me," says the Congressman, who was elected to the House of Representatives in 2007, after his father Michael vacated the seat. "Besides my uncle, several other members of my family have had conditions like Parkinson's and dementia." Bilirakis also worked for a Tampa Bay area legal practice that focused on elder law. "I've seen so many of my clients, and now my constituents, come in with debilitating diseases, such as Parkinson's, Alzheimer's, and ALS [amyotrophic lateral sclerosis]. I ran for office because I wanted to help people and do the right thing on many issues, but I've always wanted to be on health care-related committees."

That's why he joined the House Energy and Commerce Committee and its Subcommittee on Health, which is now driving the bipartisan 21st Century Cures Initiative, announced in May 2014. The initiative represents Congress's first-ever comprehensive look at accelerating the pace of discovery and development of medical cures and treatments.

Promoting Public Hearings

Between May and November of 2014, 21st Century Cures held at least a dozen national roundtables and forums on how to modernize the drug and device approval process. These public hearings explored everything from overhauling clinical trials and incorporating the patient perspective to ensuring personalized medicine and improving digital health care by developing electronic tools such as e-communications with doctors and wearable sensors and smartphone health apps.

Committee members also held their own roundtables back in their districts, a practice Bilirakis endorsed and adopted enthusiastically. "We get lobbied by advocacy groups in Washington, but you know what it's like up there: They call a vote, we have a committee hearing, and we don't have the time to really listen. In the district we have plenty of time," he says.

Bilirakis says the first two hearings in his Florida district—one for patients and patient advocates, the other for health care providers—lasted three hours each.

"My staff wasn't too happy when I opened up the conversation to the whole room," he says with a laugh, "but it was very productive, because we heard a lot of stories. People in the audience were able to get involved, give each other advice, and help each other."

Changing the System

It's easy to sense Bilirakis's frustration with the current system. "The drugs we have for Alzheimer's disease just treat the symptoms, not the disease," he says.

What can Congress do? Bilirakis says the Energy and Commerce Committee's Subcommittee on Health is working to move forward with legislation that supports medical research and development. One example is the Orphan Product Extensions Now Accelerating Cures & Treatments Act, which extends patents on drugs for an additional six months as long as they treat rare diseases and pediatric cancers in a safe and effective manner.

Another proposed bill, which Congressman Bilirakis has co-sponsored, is known as the MODDERN (Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network) Cures Act. Under this legislation, if a drug manufacturer can show that a drug whose patent has expired can be used in another way to treat "unmet medical needs," the patent will be extended.

Bilirakis predicts that a bill will make it to the President's desk during the current session of Congress. "The goal is to encourage the development of treatments and cures for rare diseases, speed up the process at the FDA, and get treatments in the hands of patients as soon as possible."

"What I see people go through on a daily basis makes me want to work even harder," says Bilirakis. "I don't have a health care degree or background, but I'm learning a lot, and I have the heart for it."

What the Public Wants

At hearings in Congressman Bilirakis's district, the public made a few suggestions for where the 21st Century Cures Initiative should direct its efforts.

Faster, More Flexible Access to Drugs

Colleen Labbadia, whose 3-year-old son has Duchenne muscular dystrophy, talked about the muscle disorder, which primarily affects boys and is usually fatal by around age 30 (although advances in cardiac and respiratory care are improving life expectancy). She asked that the FDA be more flexible when considering new treatments for these degenerative diseases, facilitate a faster approval process, and increase access to experimental and off-label drugs that are approved for other conditions. "The current system is not working for our boys," she said. "The full benefit of many of these drugs cannot be seen in the standard 48-week period of a trial."

Bilirakis is sympathetic to these concerns. "We have been actively working with people in the rare disease community, like Pat Furlong, president of Parent Project Muscular Dystrophy, to brainstorm potential legislative solutions to these situations," he says.

Improved Long-Distance Health Care

Gretchen and Michael Church, a married couple with Parkinson's disease, spoke about telemedicine, the practice of having long-distance medical consultations using video conferencing, Skype, or FaceTime.

One obstacle to telemedicine is the burden of state licensing requirements: Doctors must be licensed in the states where their patients live. So, if someone with Parkinson's lives in a state that has no doctors who specialize in movement disorders, that patient cannot be treated by out-of-state doctors, leaving him or her with few options.

"I know from my own experience that a close relationship with your doctor really matters," says Bilirakis. "In rural areas where there isn't much access to specialists, you can't expect people with Parkinson's to get in the car and drive for six hours. We have to find ways for people to have access to neurologists and other specialists on a regular basis."

That's why he's urging support for the TELE-MED Act, which he has co-sponsored. It would allow certain Medicare providers licensed in one state to provide telemedicine services to Medicare beneficiaries in another state. This bill would allow individuals who have Parkinson's or other conditions that make it difficult to travel to use telemedicine to access a specialist.