Docket No. FDA-2013-N-0502
Thank you for the opportunity to speak today. I am Dr. Tracy Rupp, a pharmacist with more than 10 years of hospital and public health experience. I was previously a clinical pharmacist at Duke University Medical Center and am now a senior fellow at the National Center for Health Research. Our research center scrutinizes scientific and medical data and provides objective health information to patients, providers and policy makers. Those are the perspectives I bring with me today. We do not accept funding from pharmaceutical companies and therefore I have no conflicts of interest.
Our Center strongly supports research and programs to improve drug safety. We remember when REMS were first proposed in legislation, the reason was to enable FDA to approve drugs with serious risks by providing a mechanism to mitigate those risks. Patients are the ultimate stakeholders. Are REMS effective at making sure the benefits of these drugs outweigh the risks?
The bottom line question is whether REMS reduce the risks and, if so, by how much? The answer is that we don’t know. And, if we find out REMS aren’t effective or are too burdensome, how should the criteria for FDA approval of new drugs be tightened?
Pharmacists and providers have put in hours and hours of work to fulfill program requirements and many patients have had to go through lots of red tape to get their drugs each month. And yet, we still have too many unanswered questions. When I worked as a hospital pharmacist, some orders for REMS drugs could take an hour or more to process. Since I may have more than 300 medication orders to verify during a typical eight hour shift, this is a lot of time for one order.
I want to take the time necessary to ensure patient safety. But, because of time constraints or the complexity of some programs, it is possible that REMS requirements aren’t always followed. The big question is whether, on average, patients are getting the safeguards they need for drugs that are subject to REMS.
In 2013, an OIG report found that the FDA lacks comprehensive data to determine if REMS improve drug safety.[1] Nearly half of all sponsor assessments for the 49 REMS that were reviewed did not include all information requested by the FDA, and only 7 of the 49 REMS met all of their goals.
Only 1 of 19 REMS with Elements To Assure Safe Use (ETASUs), which are required for the riskiest drugs, was meeting all of its goals.
When it was determined that REMS were not meeting all of their goals, lack of patient and prescriber awareness of risks was the most common problem.
A drug sponsor’s REMS assessment is typically not publicly available. More transparency is needed. Making REMS data publicly available on the FDA’s website would allow stakeholders to assess the program. Drug safety depends on vigilance and communication from people like pharmacists, providers, and patients. For many drugs, adverse event reporting from health care systems and researchers has informed FDA of new safety risks and prompted regulatory actions for those drugs. Those reports could assist the agency in assessing the effectiveness and burden of REMS programs.
FIRST, we need to identify reliable and valid methods of assessing both the effectiveness and burdens of REMS programs. The Food, Drug, and Cosmetic Act requires the FDA to review sponsors’ assessments but does not require it to perform its own assessments of each REMS. Currently, we must rely on the drug sponsor to accurately assess the program. If the information were publicly available, stakeholders could help ensure the best assessment methods. For example, when a REMS assessment involves surveillance of a drug’s prescribing patterns, epidemiologists could help develop methods to analyze the collected data so that we could determine how the REMS affects access to REMS medications.
When a REMS assessment involves a survey of patients’ understanding of risks, researchers could help develop the most accurate survey. Researchers could also help find out the answers to broader policy questions. If we aren’t able to safely use REMS drugs, do we have to change how we consider risks and benefits in the FDA drug approval process?
Lastly, we need to know how REMS affect patients’ access to medications. Do people in rural areas have access to providers and pharmacies who can provide them with the drug and ensure its safe use? When REMS require the patient to get the drug from specialty pharmacies, does that increase drug prices and red tape? Are patients unable to get the medication because of those restrictions?
In conclusion, as a pharmacist and public health advocate, I want safe and effective drugs for patients. But we need more information to understand if we’re making drugs safer through REMS programs.
Thank you for the important work that you do on behalf of the public health, and thank you for consideration of our views.