Each month, we round up the latest FDA approvals and regulations to give you an expert take on what you need to know.
NASH Pipeline Update
A form of fatty liver disease called non-alcoholic steatohepatitis (NASH) currently has no treatment options, but several drug manufacturers are working to change that. NASH is most commonly found in people that are obese, diabetic, have high blood pressure or high triglycerides. NASH may not produce any symptoms in early stages and goes undiagnosed because it requires liver biopsy to identify. Once the disease progresses it leads to cirrhosis, liver cancer and ultimately liver transplant. The NIH estimates that up to 12% of the US population have NASH.
Currently there are several manufacturers testing new drugs to treat NASH in various phases of clinical trials. Gilead recently announced their investigational product, selonsertib, did not meet their trial’s primary goal of improving liver fibrosis without worsening NASH. While Gilead is disappointed in this outcome, they will continue to further test this product in combination with their other 2 investigational NASH drugs: cilofexor and firsocostat. The results of these trials are expected later this year.
As manufacturers race to be first to market, Intercept Pharmaceuticals announced positive results for their investigational NASH drug, obeticholic acid. After 18-weeks, patients in the study showed liver fibrosis improvement without worsening of NASH. Based on these results, Intercept intends to file for FDA approval in the U.S. in the second half of 2019.
Gout Drug Gets Black Box Warning
The FDA has completed an extensive review of a safety clinical trial that found an increased risk of death associated with Uloric (febuxostat). The FDA has issued a black box warning for Uloric, which is the strictest safety alert the FDA can issue to call attention to serious or life-threatening risks.
Uloric was approved in 2009 for the treatment of gout, which is a type of arthritis that effects over 8 million people in the US. The FDA has recommended that health care professionals reserve Uloric for use only in patients who have failed or do not tolerate allopurinol. Based on this safety update, pharmacy benefit managers (PBMs) may update their formularies and utilization management policies for Uloric prior authorizations or step therapy programs.
HHS to Block Drug Rebates
The Health and Human Services (HHS) secretary Alex Azar announced plans to eliminate drug rebates for government sponsored plans. The proposal intends to end rebates on prescription drugs paid by manufacturers to pharmacy benefit managers (PBMs), Medicare Part D plans, and Medicaid managed care organizations. Additionally, this proposal seeks to replace these rebates with manufacturer discounts provided to beneficiaries at the point of sale. A new safe harbor protection will also be created to allow fixed fee services arrangements between manufacturers and PBMs.
While this proposed policy change hopes to reduce the cost of prescription drugs for beneficiaries, HHS acknowledges that eliminating rebates may increase member premiums. So while the biggest benefits would be evident for seniors taking high cost prescription medications, other Medicare Part D beneficiaries may likely see their overall costs increase due to the rise in premiums.
HHS is accepting public comments regarding the proposed rule until April 8, 2019 and once finalized the proposed effective date for policy changes is January 1, 2020.
Truveris will continue to monitor this proposal and provide updates as they are announced.