Alexion’s Soliris REMS Gets FDA Panel Review In Wider Evaluation Effort
This article was originally published in The Pink Sheet Daily
Executive Summary
FDA advisory committee will revisit REMS for ultra-rare disease drug as part of larger agency effort to evaluate the effectiveness of the safety programs.
FDA’s latest REMS under a microscope is the program for Alexion Pharmaceuticals Inc.’s Soliris. But the rare disease blockbuster’s Risk Evaluation and Mitigation Strategy isn’t coming in for an overhaul, but rather a check up as part of the agency’s broader efforts to ensure that REMS are not an undue burden on the healthcare system.
FDA announced Oct. 2 that it will host a meeting of the Drug Safety and Risk Management advisory committee (DSaRM) on Nov. 18 that will evaluate whether the REMS program for Soliris (eculizumab) “assures safe use of the drug, is not unduly burdensome on patient access to the drug, and to the extent practicable, minimizes the burden on the health care delivery system,” said the announcement.
The advisory committee is part of a movement by the agency to determine how well the REMS programs are working and if they are allowing patients access to the drugs they need, while also keeping them safe.
The agency has been evaluating these programs for several years with the promise to make the process more transparent and provide guidance on the risk-benefit assessment FDA uses to determine REMS for drugs. It also intends to provide a framework for REMS evaluation that will include an evidence-based approach (Also see "REMS Decision, Evaluation Guidances In Process, FDA Says" - Pink Sheet, 3 Mar, 2014.).
Finding the proper metrics for assessment has been a problem with determining the usefulness of REMS. Sponsors have sometimes had trouble showing whether the programs are effective and how changes to REMS tools might improve the programs (Also see "REMS Need Metrics For Assessing Success, Burden On Health Care System" - Pink Sheet, 30 Jan, 2012.).
In September, FDA identified four priority projects in a draft report outlining feedback and ideas to help better standardize and integrate the drug risk management plans into the health care system. Comments are due Nov. 24 (Also see "REMS Enhancements Could Include Provider Education Credits" - Pink Sheet, 23 Sep, 2014.).
Patient Safety Card Required
In the case of Soliris, the ultra-rare disease drug has a REMS with elements to assure safe use (ETASU) because of a boxed warning in the label that warns that life-threatening and fatal cases of meningococcal infections have occurred with patients on Soliris.
Under the OneSource Safety Support program, prescribers are required to enroll in the program and patients are required to carry a Patient Safety Card.
Soliris, a first-in-class terminal complement inhibitor, was first approved in 2007 to treat paroxysmal nocturnal hemoglobinuria (PNH), which is characterized by excessive destruction of red blood cells (Also see "Alexion Biologic Soliris Is First To Get FDA Nod For Rare Blood Disease" - Pink Sheet, 19 Mar, 2007.). The drug gained approval from FDA for a second indication in 2011 – atypical hemolytic uremic syndrome (aHUS), a condition in which blood clots in small blood vessels throughout the body and can lead to kidney failure, stroke, heart attack and death (Also see "Alexion's Soliris Gains New Orphan Indication But Limited Sales Expectations" - Pink Sheet, 26 Sep, 2011.).
The drug is priced at approximately $400,000 a year for treatment in PNH, and has performed well for the company – bringing in $512.5 million in the second quarter of 2014 – but has had pushback from British reimbursement watchdog NICE due to its high price tag (Also see "Alexion’s Costly Soliris For AHUS Raises “Cost Of Life” Issues At NICE" - Pink Sheet, 4 Mar, 2014.).