Second Opinion:
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August 5, 2010:
Improving the FDA's REMS Program

The FDA recently concluded meetings reviewing industry, patient, and provider concerns surrounding the implementation of its Risk Evaluation & Mitigation Strategy (REMS) authorities. The driving concept behind REMS was to find a way of improving the use of drugs with known safety risks through a variety of tools. Patients and pharmacists complain that they have not been given enough input into REMS creation, while companies argue that the FDA has been slow to respond to requests to modify or improve REMS when it seems appropriate to do so. Given that the Food and Drug Administration Act of 2007 requires REMS not be "unduly burdensome" to providers or patients, how do you think that the FDA can incorporate these concerns into an improved REMS process that balances timely patient access with mitigating severe drugs risks?

This edition of our expert panel includes:


By Marc Boutin

The National Health Council (NHC) is an umbrella organization providing a united voice for the millions of people in this country with chronic diseases and disabilities. These individuals rely on medications to live more normal and healthy lives, and several of their drugs include risk evaluation mitigation strategies (REMS).

The FDA faced many difficulties creating a program from scratch. That said, the patient community believes there must be a better way to balance the risks of medications relative to their benefits.

For example, when deciding whether to implement REMS, the FDA should consider both the size of the population affected and the range of existing treatment alternatives. Any data collection required as part of REMS must report both the benefits and risks of the treatment as compared to the risk of non-treatment or reatment with existing therapies.

The FDA should also consider the impact of REMS on patient access. We recommend establishing criteria and metrics that would allow for an evaluation of the impact of REMS on patient care.

The reality is we are still not getting new treatments to market as quickly as the patient community would like or need. By working together with the FDA and all stakeholders we can do better in getting new treatments through the regulatory process.


Marc M. Boutin, J .D., is the executive vice president and chief operating officer of the National Health Council. Before joining the Council, he most recently served as the vice president of government relations and advocacy at the American Cancer Society for New England.

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By Scott Gottlieb, M.D.

REMS ultimately obligate FDA to regulate the practice of medicine, but give the agency few legal tools to regulate doctors. As a result, this new authority – which the agency actively sought – will leave FDA vulnerable to future criticism.

The fact remains FDA has few direct means for influencing physicians. It is dependent upon drug companies to implement the elements contained in a REMS. The drug companies, in turn, lack legal authority over physician prescribing. All they can do is try and cut off supply of a drug from doctors who might not be compliant with a particular risk mitigation scheme. As evidence mounts that the REMS are not having their intended effect, or that doctors are non-compliant with provisions, the tendency will be to exert increasingly tighter control over prescribing behavior. Eventually, Congressional oversight will expose some of these weaknesses. In turn, the political scrutiny will force still-tighter strategies.

FDA can mitigate some of these potential pitfalls by making sure that its mitigation strategies are tightly mapped to evidence that demonstrates an impact on risk. Right now, many of the measures FDA imposes as part of REMS aren’t grounded in the science of risk mitigation or social marketing. There isn’t any science to support one REMS approach over another, and no guidance mapping particular drug risks with certain mitigation steps. FDA needs to ground its activities more closely in the social sciences of risk management to ensure that its activities are effective, efficient, and cosseted from political criticism.


Scott Gottlieb is a resident fellow at the American Enterprise Institute and a practicing physician. He has served in various capacities at the Food and Drug Administration, including senior adviser for medical technology; director of medical policy development; and, most recently, deputy commissioner for medical and scientific affairs.

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By Steven Grossman

Our current system of drug information generates far too many preventable errors, resulting in mortality, morbidity and therapeutic failures. REMS is a possible answer and FDA is working to make it a success. It will take about five years of debate, reaction, and conflicting demands for FDA to work out some fundamental kinks. FDA is about on schedule.

Neither medical product companies nor FDA benefit from unsuccessful REMS, but they often have different ideas about what is needed. Patients, pharmacists, physicians and insurers also have a stake in the outcome. If an increasing number of REMS require extended consultation with physicians and pharmacists, then access to these products will diminish. For multi-morbid patients, there is certain to be information overload about their many medicines with REMS requirements.

The FDA needs to open a public docket for comment on potential REMS issues for every product it considers. FDA won’t be happy with the additional regulatory burden and for many products it will be a waste because they won’t get approved. However, the comment period needs to start soon after the NDA/BLA is filed and accepted. It can’t wait until approval is more certain…without delaying patient access to needed therapies. FDA is starting to develop REMS by drug class, but is learning that this brings its own difficulties. Further, some of the most needed drugs are likely to raise novel issues or fall outside of homogeneous drug classes.

One can wish for patients and prescribers to hear and heed information and advice on drug usage. Until that ideal is achieved, FDA and medical product companies are going to struggle with REMS and they need to accept a less-than-perfect system for stakeholder input.


Steven Grossman is a consultant specializing in FDA policy and regulatory issues. In the early 1980’s, he was health staff director and counsel to what is now the Senate HELP Committee. He served as a deputy assistant secretary for health at HHS from 1985 to 1989. He is the author of the blog, FDA Matters: www.fdamatters.com.

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By Henry Miller

The Food and Drug Administration Amendments Act, which took effect on March 25, 2008, gave the FDA sweeping new powers, among which was the ability to require a Risk Evaluation and Mitigation Strategy (REMS) for every newly approved drug. According to the FDA, a REMS is "a strategy to manage a known or potential serious risk associated with a drug or biological product," which could be said to encompass virtually every medicine. The agency's explanation continues, "A REMS can include a Medication Guide, Patient Package Insert, communication plan, elements to assure safe use, and an implementation system, and must include a timetable for assessment of the REMS." The term "elements to assure safe use" sounds benign enough, but regulators' demands can be so drastically restrictive as to constitute a new, distinct and limited, or conditional, class of approvals. The elements to assure safe use as defined in the legislation may include:

  • "Health care providers who prescribe the drug have particular training or experience, or are specially certified."
  • "Pharmacies, practitioners, or health care settings that dispense the drug are specially certified."
  • "The drug is dispensed to patients only in certain health care settings, such as hospitals."
  • "The drug is dispensed [only] to patients with evidence or other documentation of safe use conditions, such as laboratory test results."
  • "Each patient using the drug is subject to certain monitoring."
  • "Each patient using the drug is enrolled in a registry."

These limitations taken together imply that FDA has invented a new second-class approval--a limited, or conditional, approval that has the potential to reduce substantially the potential market for a drug. That is obvious for the first three elements above, but it also applies to the last two because anything that makes a drug more difficult or complicated to prescribe discourages physicians from doing so, even when it is needed.

As to the FDA avoiding "unduly burdensome" REMS, the current agency leadership, culture and trends suggest that that won't be a high priority.


Henry I. Miller, M.S., M.D., is a research fellow at the Hoover Institution. Miller joined the Food and Drug Administration in 1979 and from 1989 to 1993 was the founding director of the FDA's Office of Biotechnology.

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