The procurement process isn't simple for the health care industry. While technology and scientific advancements are helping people live longer and overcome serious diseases, certain cancers, rare disorders, Alzheimer's and other conditions remain incurable.
However, federal officials are aiming to provide patients with more treatment options, in consultation with their primary care physicians, through an updated expanded access program.
Installed by the U.S. Food and Drug Administration, Expanded Access is an initiative optimized by the Right To Try Law (RTT), which Congress passed and President Donald Trump signed into law earlier this year. As its title implies, EA is designed to give consumers more control over their health care by enabling them to take advantage of investigational drugs and biologics for life-threatening conditions despite no clinical trials being done on them to determine their safety or efficacy.
"EA has helped bring about 9,000 health service applications in the last five years."
"FDA staff is deeply committed to this program and ensuring that it works quickly and effectively for patients and their physicians," Gottlieb said in a statement recently issued by the agency. "Emergency requests for individual patients are usually granted immediately by phone. Non-emergency requests are generally processed within a few days."
RTT, EA aren't one and the same
Although RTT and EA have a number of similarities, they're different in function and practice. As noted in the journal Health Affairs by Alison Bateman-House, an assistant professor at the Division of Medical Ethics at New York University Langone Health, EA is slightly more expansive. For instance, while RTT provides individuals with access to investigational drugs, EA applies to these and other medical services, such as technologies, devices and products. Additionally, while the Right To Try program provides certain legal protections for pharmaceutical firms and medical facilities when treatments don't work, EA has no such defenses.
It's in light of this that the FDA has sought to more assiduously structure the EA program to optimize health care facilities supply chain and simplify it so patients aren't hamstrung by red tape.
"One improvement FDA made was streamlining the required supporting documentation for expanded access requests submitted by a physician for access to a drug or biological for the treatment of an individual patient," FDA Commissioner Gottlieb said. " These changes reduced the administrative burden for these physicians. Following these changes, we estimate that it takes about 45 minutes, on average, to complete a patient application form. That form typically requires just one attachment."
More EA oversight now in place
The FDA has also tightened up the program to assuage the concerns of organizations opposed to the RTT. The American Lung Association, American Society of Clinical Oncology and the Cystic Fibrosis Foundation were a few of the groups that signed a petition in March, which urged lawmakers to modify RTT so it provided patients with more assurances as to their well-being. The missive warned that because the agency approves the overwhelming majority of investigational therapies - 99 percent, by some estimates - expanded access could wind up doing more harm than good.
"We welcome the opportunity to continue constructive dialogue on ways to improve the ability of patients to genuinely and safely access both approved and unapproved lifesaving therapies," the letter read.
"The FDA itself has implemented some internal restructuring."
"The patient affairs staff is already in place and charged with serving as the 'home base' and primary point of entry for patients and physicians starting the EA process and navigating them through the steps," Gottlieb noted.
He went on to mention that the agency has installed an EA coordinating committee. Gottlieb said he's both hopeful and optimistic that opening the doors to investigational medicines will lead to greater understanding of life-threatening diseases, which can inform the manner in which health care manage their supply chain of treatments.