NASHVILLE —  More orphan drugs approved in recent years are filling the need for treating rare disease and medical conditions, according to a presenter at the the American Association of Nurse Practitioners 2014 meeting.

Alan Agins, PhD, who gave a pharmacologic overview of drug approvals since 2012, noted a growing trend among pharmaceutical companies.

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“I think what we’re going to see for the next [two decades] is more specialty drugs,” Agins told Clinical Advisor. “Overall for the last 15 years, the number of drugs approved has gone down, partly because we’ve pretty much filled out our dance cards on chronic diseases.”

There were 27 first-of-a-kind drugs approved by the FDA in 2013 compared to 39 approvals just the year before. About a third of those approvals were indicated for the treatment of rare conditions and diseases.

Of note, afflicted patients with several conditions would benefit the most from recent approvals, Agins said.

Patients with chronic obstructive pulmonary disease (COPD) can now benefit from once-daily inhalers such as fluticasone and vilanterol (Breo Ellipta). Ease of use and convenience allow for better adherence in the patient population.

Patients with type 2 diabetes can take once-weekly GLP-1 agonists, enabling patients to avoid the hassle of maintaining the pharmacokinetics of the drug with once- or twice-daily dosing. Drugs such as exenatide, although expensive, have better efficacy and fewer side effects.

Newer oral anticoagulants such as dabigitran give patients more flexibility when it comes to diet and lifestyle modifications compared to warfarin. In studies, these medications were associated with lower rates of hemorrhage compared to warfarin, showed no interactions with diet/lifestyle, and did not need to be monitored.

Agins also warned about several unmet needs in drug development, most damagingly from growing drug resistance in antibiotics and the lack of new antibiotic classes.

“The newest class of antibiotics for primary care providers is now 32 years old,” he said about fluoroquinolones. “It’s so much so [a problem] that the government actually spent about $200 million last year to hire one of the pharmaceutical companies to develop new antibiotics.”

Alzheimer disease is another condition with an increasing lack of drug treatment options. “Since 1998, over 104 drugs have entered clinical trials and have all failed in terms of AD,” Agins said. “We have an idea of what causes the disease but we’re still not definitive, and we certainly don’t have any drugs out there on the near horizon.”

Agins is hopeful that in the next 20 years, we will be making the switch from simply managing diseases with drugs to curing them outright.

“But it’s going to take some biotechnology and microbiology,” he said. “We’re treating symptoms of hypertension and cholesterol and all those other things now, eventually we’re going to cure those diseases or prevent them totally.”


  1. Agins AP. #14.3.039. “New Drugs in the Arsenal in Primary Care.” Presented at: AANP 2014. June 17-22; Nashville, Tenn.