Date: July 2007
Target Audience: Healthcare Industry, government agencies, and FDA staff
Laws and Regulations:
- Sections 502, 505, 510(k), 515, 564, and 903 of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360bbb-3)
- Project BioShield Act of 2004 (Public Law 108-276)
- Sections 319, 351 of the Public Health Service Act (PHS Act)
- Good Laboratory Practice requirements in 21 Code of Federal Regulations (CFR) part 58
- Informed consent under part 50 of FDA regulations (21 CFR part 50)
- Medtronic v. Lohr, 518 U.S. 470, 503 (1996); id. at 510
- Cipollone v. Liggett Group, Inc., 505 U.S. 504, 521 (1992) (plurality opinion); id. at 548-49
- English v. General Electric Co., 496 U.S. 72, 79 (1990)
- Florida Lime & Avocado Growers, Inc., 373 U.S. 132, 142-43 (1963)
- Hines v. Davidowitz, 312 U.S. 52, 67 (1941)
- Federal Employees’ Compensation Act (5 U.S.C. 8101 et seq.)
- Federal Tort Claims Act (28 U.S.C. 1346(b))
- Smallpox Emergency Personnel Protection Act of 2003 (42 U.S.C. 233(p))
- National Vaccine Injury Compensation Program (42 U.S.C. 300aa-10 et seq.)
- Support Anti-terrorism by Fostering Effective Technologies Act of 2002 (SAFETY Act)
- Public Readiness and Emergency Preparedness Act of 2005 (Pub. L. 109-148)
- 21 CFR 312.34(b)(3)(A)
- 21 CFR 314.610(a)(2) and (3)
FDA guidance for the administration and use of unapproved medical products in the event of a state of emergency. These emergency healthcare measures are intended to protect the public from biological attack, as a declared emergency preventative action due to heightened risk, or as a response to various acts of bio terrorism that threaten US national security.
The guidance explains the powers initiated by the Emergency Use Authorization (EUA) to be used in the event of an emergency, in order to provide diagnosis, prevention, and treatment during biological, chemical, radiological, and nuclear attacks. It states that enacting the implementation of unapproved medical products is only permitted when there are no available approved forms of prevention and treatment.
Using section 564(b)(1) of the FD&C Act to support its position, the FDA defines what constitutes declaration of an emergency. After the emergency is declared, an EUA can be issued. The guidance follows this with EUA eligibility criteria, which include but are not limited to: life threatening disease, efficacy hypotheses of potential treatment, risk vs benefit, and availability of alternative treatment.
Procedures for the drug sponsor on how to request their drug be considered as an EUA candidate are explained. This is vital to efficient emergency management due to time constraints and limitations of the process; the FDA Secretary must first officially declare an emergency, then an EUA can be issued. The guidance discusses recommended steps for overcoming these time limitations by utilizing pre-emergency activities, while the emergency is still in the process of being officially declared, and an EUA has not been issued yet.
Data included in the request for an EUA is similar to that of non-emergency drug submissions, which would include safety, efficacy, chemical, and scientific data. Where the EUA drug candidate submission process differs from Investigational New Drug (IND) and New Drug Applications (NDA) is that there will be far less information available to submit, so it may be necessary to include interim study reports to make up for this shortcoming, and speed along the process due to the expediency an emergency mandates.
The guidance explicates how an EUA is processed, including conditional criteria for emergency use of an unapproved product, and for use of an approved product for an unapproved use. This section also explains that Good Manufacturing Practice (GMP) requirements can be waived or limited as the FDA Commissioner sees fit based on the conditions of the emergency. As with clinical trials, adverse events must be reported.
Compared to clinical trials, though, there are some fundamental differences regarding the administration of an EUA treatment product vs a non-emergency test article. One of the primary differences is that informed consent is not required, “informed consent under part 50 of FDA regulations (21 CFR part 50) is not required for administration of an EUA product and the information dissemination requirements of section 564 are mandatory only to the extent conditions establishing such requirements are practicable.” However, the guidance also recommends that, “recipients be given as much appropriate information as possible given the nature of the emergency and the conditions of the authorization.” Although informed consent is not necessary, the guidance states that it is required that patients be informed that an emergency use of the product has been authorized by the FDA, and that the benefits and risks involved are unknown.
This guidance also includes recommendations on preemption, EUA termination, liability protection, and possible compensation issues. Appendices feature fact sheets for both healthcare providers and treatment recipients. As with other FDA guidances, these methods of emergency response are not legally enforced but strictly recommendations based on “the Agency’s current thinking.” However, there is extensive legislation cited that adequately supports the FDA’s recommendations for the entire EUA process.
This guidance clarifies how the EUA supports the legal distribution of unapproved medical products in the event of an emergency for the healthcare industry, FDA staff, US government agencies, and emergency management professionals. It can help said professionals to develop emergency management plans for bio terrorism and similar emergency scenarios. The numerous laws and regulations referenced can help establish the legal framework for protecting these government and industry professionals from liabilities encountered in an emergency situation.
The FDA recommends that patients be informed as much as the situational conditions of the emergency will allow, and that candidate treatment sponsors have a pre-emergency plan to ensure that EUA treatment can become available as soon as possible, for timely public safety.
This guidance should be understood and utilized by healthcare providers, local and state government agencies, and FDA regulatory reviewers. The instruction and recommendations are practical considerations to fulfill the goal of reacting to a state of emergency in a timely fashion, in order to protect the public from harm as best as possible.
For more information on this issue, contact the Kulkarni Law Firm.