FDA Approvals in 2019 and What They Mean for Healthcare Organizations

From peer-reviewed studies highlighting medical breakthroughs to updated evidence-based medical guidelines, physicians are tasked with continually keeping pace with advancements in medicine. One source for this education is the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) which approves multiple new drugs each year. In 2019, the agency approved 48 novel drugs, defined by the FDA as “often among the more innovative products in the marketplace, and help advance clinical care by providing therapies never before marketed in the United States.”

We’ve compiled a list for you of some important new drugs that came to the market last year and the research studies that advanced their approval. Each of these drugs addresses an illness prevalent in the U.S.

Aloradine: Social Anxiety Disorder

Anxiety is a prevalent mental health issue in the U.S. Social Anxiety Disorder, in specific, affects approximately 15 million adults, and an estimated 12.1 percent of adults in America experience it at some time in their lives. Defined by the Anxiety and Depression Association of America (ADAA) as “intense anxiety or fear of being judged, negatively evaluated or rejected in a social or performance situation,” it typically begins around the age of 13. However, an estimated one-third of those suffering from social anxiety disorder wait 10 years or more to discuss their symptoms with a physician.

Aloradine (PH94B), a central nervous system neuroactive nasal spray for the on-demand treatment of social anxiety disorder, received Fast Track designation from the FDA. In a phase 2, multicenter, randomized, double-blind, placebo-controlled, single-dose study of PH94B, 91 women 19–60 years of age with generalized social anxiety disorder received placebo intranasal spray (single-blind) 15 minutes before laboratory simulated public speaking and social interaction challenges. Aloradine was found to significantly reduce anxiety levels in the study’s recipients, and a phase 3 development is in preparation by pharmaceutical company VistaGen Therapeutics. According to VistaGen, the drug may have the potential to similarly treat other anxiety disorders.

XCOPRI: Partial-onset Seizures

This past November, the FDA approved XCOPRI (cenobamate tablets) to treat partial-onset seizures in adults. Developed by SK Life Science, Inc., the drug was tested in two randomized, double-blind, placebo-controlled studies of patients who experienced partial-onset seizures with or without secondary generalization for an average of 24 years. These patients also had about 8.5 seizures per day during an eight-week baseline period. After being given doses of XCOPRI from 100-400 milligrams daily, the percentage of seizures was reduced in a span of 28 days compared to patients who received a placebo.

The Mayo Clinic defines a seizure as a sudden, uncontrolled electrical disturbance in the brain that can cause changes in behavior, movements or feelings and levels of consciousness. There are numerous types of seizures, and up to 10 percent of humans will experience one at some point in their life. Most last between 30 seconds and two minutes, and almost 70 percent of nonepileptic seizures occur in women.

Piqray: Breast Cancer

Piqray (alpelisib) tablets were FDA-approved in May 2019 to be used in combination with the FDA-approved endocrine therapy fulvestrant. The aim of this drug combination is to treat postmenopausal women and men with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer (as detected by an FDA-approved test) following progression on or after an endocrine-based regimen. It’s the first new drug application for a new molecular entity approved under the Real-Time Oncology Review pilot program. Along with Piqray, the FDA approved the therascreen PIK3CA RGQ PCR Kit, a companion diagnostic test utilized to detect the PIK3CA mutation in a tissue and/or a liquid biopsy. 

In the SOLAR-1 trial, a randomized trial of 572 postmenopausal women and men with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor, Piqray added to fulvestrant showed noticeably prolonged progression-free survival in patients with tumors with a PIK3CA mutation. This survival rate increased from an average of 5.7 months to a median of 11 months.

Treatments and drugs for breast cancer are increasingly important, as a study performed by multiple cancer researchers showed that the number of women in the U.S. living with distant metastatic breast cancer (MBC) is growing. According to the Metastatic Breast Cancer Network, approximately 6-10 percent of new breast cancer cases are initially Stage IV or metastatic, and the number of metastatic recurrences are estimated to range between 20-30 percent of all existing breast cancer cases.

Zulresso: Postpartum Depression

Between 10-20 percent of new mothers report experiencing clinical postpartum depression. What might be surprising to some is that half of men who have partners with postpartum depression will go on to develop depression themselves.

Symptoms of postpartum depression include:

• Feeling sad, hopeless, empty, or overwhelmed
• Crying more often than usual or for no apparent reason
• Worrying or feeling overly anxious
• Feeling moody, irritable or restless
• Experiencing anger or rage
• Losing interest in activities that are usually enjoyable
• Withdrawing from or avoiding friends and family
• Having trouble bonding or forming an emotional attachment with your baby
• Persistently doubting your ability to care for your baby
• Thinking about harming yourself or your baby 

Zulresso (brexanolone), an injection for intravenous (IV) use, is the first treatment approved by the FDA specifically for postpartum depression in adult women. It was tested through two clinical studies in which participants received a 60-hour continuous IV infusion of the drug or a placebo. After the infusion, the participants were followed for four weeks, and Zulresso was shown to improve depressive symptoms at the end of the first infusion. There is a restriction with this drug, though – it will only be available through a program requiring it to be administered by a healthcare provider in a certified healthcare facility. 

Additional Drug Approvals

A list of the other Novel Drug Approvals for 2019 can be found on the FDA website. The FDA’s Center for Drug Evaluation and Research’s annual report provides an index of new and expanded uses of drugs that have already received FDA approval. It’s crucial to understand that the FDA ensures medical treatments are safe for human use but doesn’t conduct any clinical trials. In the FDA’s own words, the agency’s “staff meet with researchers and perform inspections of clinical trial study sites to protect the rights of patients and to verify the quality and integrity of the data.”

Experimental Use and Standard of Care

Even though a drug has received approval from the FDA, it’s important to differentiate between experimental (also referred to as investigational) and standard of care in medication prescriptions. Experimental refers to a laboratory-tested and FDA-approved drug that a health plan classifies as investigational and doesn’t cover, even if it’s deemed medically necessary. It may be approved to treat a specific illness but categorized as investigational for other diseases.

Once a drug is FDA-approved and ready for use by the public, it includes a package insert to help market and advertise it. This information is not intended to indicate a medical standard of care. As the American Medical Association notes, a medication package insert “should not be regarded as a legal standard of acceptable or accepted medical practice nor as a substitute for clinical judgment or experience nor as a limitation on usage of the drug in medical practice.”

This type of language can be confusing to interpret. That’s why independent review organizations (IROs) can be an asset to your internal peer review services. A nationwide network of physicians covering all major specialties and subspecialties ensures your drug utilization reviews are performed by physicians appropriately licensed to identify when prescriptions may be appropriate and necessary. As a comprehensive, URAC-accredited IRO, the MLS Group of Companies is adept in serving third-party administrators, insurance carriers, utilization review organizations, hospitals, state and federal agencies and other healthcare organizations. Contact us to learn more.