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Three Orphan Drug Program problems and how the FDA plans to fix them


Recently, FDA Commissioner Scott Gottlieb announced the FDA will move to close loopholes in the Orphan Drug Program. Here’s what you need to know.

Drug development has always been expensive and time consuming. In 1983, Congress passed the Orphan Drug Act (ODA) to create incentives for pharmaceutical developers to create products for conditions that occur in fewer than 200,000 people in the United States.

Rare conditions have historically been less desirable for pharmaceutical development given the challenges drug manufacturers have in recouping their investments, says Stacy Lauderdale, PharmD, BCPS, senior director, Drug Information, Vizient, Inc., a member-driven healthcare services company. ODA offers financial incentives including tax credits and financial grants as well as additional market exclusivity to encourage drug development for rare diseases.

Recently, FDA Commissioner Scott Gottlieb announced the FDA will move to close loopholes in the program. Here’s more on what those loopholes are, and how the FDA will address them.

The loophole: Companies are pursuing orphan designation as a means to avoid pediatric clinical studies

When the FDA bestows an “orphan” designation, it validates a product’s potential benefit. However, this designation also exempts a product from clinical studies in a pediatric population. When a product is approved for an adult population (not an orphan population), sponsors can pursue and receive an orphan designation for a pediatric population which waives Pediatric Research Equity Act (PREA) requirements, explains Greg Dombal, chief operating officer, Halloran Consulting Group, a management consulting firm.

But as medical understanding has expanded, the FDA has grown concerned that some companies are pursuing an orphan designation with the primary purpose of avoiding the post-approval commitment, cost, and risk of a pediatric clinical study. This apparent reduction in clinical studies is contrary to what the agency was seeking with the ODA.

In addition, ODA’s market exclusivity provision bars the FDA from approving any new or abbreviated application for the same drug for the same indication during the period of exclusivity and the manufacturer can price the drug at what the market can bear, Lauderdale explains. In 2016, the average cost per patient per year for orphan drugs was 5.5 times higher than non-orphan drugs (using median prices) and with a return on investment of 1.89 times greater than a non-orphan drug.

How the FDA plans to fix it

The FDA is proposing to stringently enforce the requirements for pediatric studies and ensure that orphan designations are granted for the purpose of (and actually encourage) studies in all groups who might benefit from a drug. “The proposed changes could eliminate the waiver of PREA requirements for some orphan products,” Dombal says.

Next: Problem #2 and #3



The problem: Backlogs and outdated requirements

Another reason why the FDA is proposing changes is because in recent years, the proliferation of diagnostic tools and better understanding of genetic markers have afforded the ability to segment many common diseases into smaller, more precise groups that qualify for orphan designation, Dombal says.

In addition, some of the FDA’s proposed changes address process improvements, such as how to clear a backlog of more than 200 requests for orphan status. The number of requests received by the FDA under the Orphan Drug Designation (ODD) Program has more than doubled from 2012 to 2016.

“Not only has the number of requests increased dramatically, but the products that are the subject of requests have become more scientifically complex and may require specialized reviews from various FDA centers,” says Nancy Pire-Smerkanich, DRSc, MS, assistant professor, Department of Regulatory and Quality Sciences, University of Southern California, School of Pharmacy, Los Angeles, California.

How these changes will affect you

 The FDA’s efforts will affect the healthcare industry in several ways. First, it will likely result in more clinical trials in pediatric populations. PREA requires that the FDA mandate clinical studies in a pediatric population for drugs approved for adult populations where they are likely to be used in a younger patient group. “Sponsors will no longer be able to gain exemption from pediatric studies via an orphan designation,” Dombal says.

The logical offshoot of increased studies in children will be significant; it could reveal new information regarding safety, efficacy, dose schedules, and other information for many drugs in the pediatric space, Dombal continues.

This could lead to a significant change (and perhaps an improvement) in drug labeling. This would increase the complexity of managing formularies, pharmacy operations, and clinical administration. On the plus side, clinical studies in the actual population will lead to significantly greater consistency in dosing, decreasing dosing errors and increasing safety for pediatric patients.

Karen Appold is a medical writer in Lehigh Valley, Pennsylvania.


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