Risk Management Plans May Restrict Patient Access, Gottlieb Says
The FDA and Congress may focus on regulating medical practice issues as lawmakers work on legislation that would “fundamentally change” the agency, American Enterprise Institute Fellow Scott Gottlieb said at the Third Annual FDA Regulatory and Compliance Symposium at Harvard University.
The Food and Drug Administration Revitalization Act (FDARA), S. 1082 and H.R. 2900, would “create a new vision” for the agency’s drug regulation activities, said Gottlieb, former FDA commissioner for medical and scientific affairs. The bills, which would reauthorize the Prescription Drug User Fee Act, are currently pending before a congressional committee.
The bills will pass soon after Congress returns from its recess Sept. 4, and the FDA will quickly announce several new activities as a result of the legislation, according to Gottlieb.
The FDA faces a difficult challenge implementing the new requirements included in the legislation, Gottlieb said. The bill would require specialized personnel, a more active postmarketing surveillance system and a larger adverse event reporting database, he added. The challenge of finding resources concerns the agency, and leading members at the Center for Drug Evaluation and Research are discussing how they can create the needed architecture to support the requirements, he said.
One of the provisions in FDARA could burden the agency as well as restrict patient access to drugs, Gottlieb said. A provision to give the FDA legal authority to require risk management plans as part of the drug approval process is “the most significant part” of the bill, Gottlieb said.
Risk management plans can create obstacles blocking patients from receiving medication, according to Gottlieb. For example, the risk management plan for Amylin’s diabetes drug Symlin (pramlintide acetate) meant that only a diabetologist could prescribe the drug, due to the agency’s concerns about potential dangerous side effects. However, this meant that many patients in inner city or rural areas without access to a diabetologist could not get a prescription for the drug, he said.
In addition, risk management plans do not always prevent or decrease serious adverse events. For example, the plan for Accutane (isotretinoin) was put in place due to the risk of serious birth defects in children if women taking the drug became pregnant. But the plan “had almost no effect,” Gottlieb said. The number of exposed pregnancies remained virtually the same — approximately 200 — both before and after the risk management plan.
The FDA will have to audit how companies comply with risk management plans and how they review physicians who prescribe their drugs, creating another responsibility for the agency, Gottlieb said. “If you have a requirement, you have got to audit it. And if you don’t, you are going to get burned one day as a regulator,” he said.
However, he predicted most companies will comply with the plan requirement and the agency will “tread cautiously” when calling for a risk management plan. In addition, the plan could help the FDA introduce drugs to the market earlier or keep drugs on the market if the plan makes the agency feel like it has more control over the drug, he said. — Emily Ethridge