On March 22 and 23, 2012, the U.S. Food and Drug Administration (FDA) will hold a public hearing on a potential new paradigm that would enable the agency to approve drugs that would otherwise require a prescription for nonprescription use under conditions of safe use.  In this newsletter, we provide a summary of the proposed paradigm and address some of its implications.

In a February 28, 2012, Federal Register notice, the U.S. Food and Drug Administration (FDA) announced it will hold a public hearing on March 22 and 23, 2012, on a potential new paradigm that would enable the agency to approve drugs that would otherwise require a prescription for nonprescription use under conditions of safe use.  The conditions of safe use would be specific to the drug product and might require sale in circumscribed health care settings, such as a pharmacy.  FDA has invited the public to provide input on the feasibility of this paradigm and its potential benefits and costs.

FDA currently classifies approved drug products into one of two categories: prescription drugs or nonprescription drugs.  Under the Federal Food, Drug, and Cosmetic Act (FDCA), prescription drugs are deemed not safe for use except under the supervision of a practitioner licensed by law to administer the drug and thus, are only dispensed pursuant to a prescription from an authorized prescriber (e.g., a physician, nurse practitioner or physician assistant).  Nonprescription drugs are drugs that do not meet the statutory criteria for prescription products, are safe and effective for use in self-diagnosis or self-medication as directed in the product’s labeling, and thus, may be purchased without a prescription and without a practitioner’s input.

Under the potential new paradigm, FDA would create a third category of approved drugs: drugs that would generally require a prescription, but that the agency will allow to be distributed on a nonprescription basis when used consistent with product-specific conditions of safe use.  Potential conditions of use identified by the agency include pharmacist intervention to ensure appropriate nonprescription use, use of innovative technologies (e.g., diagnostics) approved or cleared by FDA for use in the pharmacy setting to identify appropriate patients, the consumer’s use of an available algorithm that enables consumers to properly self-diagnose certain medical conditions, confirmation of a diagnosis or routine monitoring using a diagnostic test available in a pharmacy, and limiting sales to certain settings.

FDA signifies that it is considering a number of alternatives for the potential new paradigm, to provide consumers with a broader array of options.  For example, FDA is considering whether the same drug product could be simultaneously marketed as a prescription and nonprescription product under conditions of safe use.  By allowing such circumscribed flexibility, a consumer could potentially choose to continue seeing their medical practitioner to obtain prescriptions or, if the consumer’s local pharmacy is equipped to offer the nonprescription product, to obtain the nonprescription product with conditions of safe use at that pharmacy.

The agency also discusses the type of data that might be required to demonstrate the proposed product could be used safely and effectively in the nonprescription setting with conditions of use.  In this regard, FDA anticipates an application may need patient studies, such as self-selection studies, label comprehension studies and actual use studies, depending on the situation, for drug products.  When a device, such as a diagnostic test or computer algorithm is needed as a condition of safe use, the application may need to demonstrate the device will work as intended and can be appropriately administered in the particular setting.  Further, while FDA states certain classes of drugs may be appropriate candidates for the new paradigm, FDA will need to evaluate each New Drug Application, and when applicable, each device, on a case-by-case basis.

In explaining why FDA is considering the new paradigm, the agency states the requirement for a prescription, including the need to make one or more trips to a medical practitioner, leads to the undertreatment of common medical conditions such as high cholesterol, hypertension, migraines and asthma.  FDA notes that in instances where an initial visit to a practitioner may be needed, the paradigm could permit, for example, a certain number of refills beyond that currently required without a return visit under specialized conditions of safe use.  FDA believes this paradigm, if implemented, may improve patient access to pharmaceuticals, improve health outcomes, free up prescribers to spend time with more seriously ill patients, reduce health care costs and reduce burdens on an overburdened health care system.

Scope of the Public Hearing

In the Federal Register, FDA states it will be seeking the public’s response to a series of 23 questions related to the potential new paradigm at the public hearing.  Questions posed by the agency include:

• Can you suggest specific medical conditions or diseases for which consumers may benefit if the treatment drug were available as a nonprescription product with conditions of safe use?
• What types of technologies (e.g., kiosks, computer algorithms) are currently in development that could assist in allowing drugs to be used safely and effectively in the nonprescription setting?
• What other types of conditions of safe use (e.g., pharmacy monitoring or counseling) could be used to help ensure the safe and effective use of certain drug products as nonprescription products?
• What types of studies could be conducted to evaluate the effects of conditions of safe use on the safety and efficacy of particular drugs and on behavior and health outcomes?
• What types of studies could be conducted to evaluate the safety and efficacy of any technologies that might be relied upon as conditions of safe use?
• Would this new paradigm increase consumer access to necessary medical care?
• Would a lack of oversight from a practitioner, including involvement in diagnosing the condition or monitoring for drug interactions or other drug effects, be a concern?  If so, how could these concerns be addressed?
• How might the new paradigm be expected to affect consumers financially or otherwise affect access to and delivery of health care generally?
• Would expanding what would be considered nonprescription drugs under the new paradigm, and thus creating greater consumer access to needed drug products, reduce the burden on emergency rooms and on individual health care providers, or otherwise increase the availability of these resources for other consumers?  Are there other ways in which the new paradigm might reduce the burden on the health care system?
• Would additional specialized training be needed for pharmacists if this paradigm were adopted?
• What are the public health and regulatory implications of the use of in vitro diagnostic tests as conditions of safe use for nonprescription drug products in a pharmacy setting (e.g., as a laboratory under the Clinical Laboratory Improvement Amendments (CLIA))?
• How would insurance coverage of pharmaceuticals be affected by approving nonprescription products with conditions of safe use for widely prescribed prescription drugs under this paradigm?
• How would out-of-pocket costs for the insured be affected by making prescription drugs available as nonprescription products with conditions of safe use?
• What proprietary, technological, economic or competitive barriers might impede widespread implementation of this paradigm?  To the extent such impediments exist, are there suggestions for mitigating or avoiding the impediments specific to this paradigm?
• Would overall health care costs decrease if this paradigm were instituted?

Parties interested in presenting oral comments at the public meeting should register with the agency by March 9, 2012. 

FDA will accept written comments regarding the potential new paradigm until May 7, 2012.

Implications

For the second time in five years, FDA will be holding a public meeting to discuss the creation of a hybrid prescription/nonprescription class of drug products.  The potential new paradigm touches on and proposes to take advantage of a number of medical advances to increase access to medical products, such as the development of diagnostic devices for use in novel settings, as stand-alone or as combination products, and personalized medicine.  The new paradigm would also potentially utilize pharmacists and pharmacy settings in new and innovative ways.

The primary immediate beneficiaries of the new paradigm, if adopted, would appear to be sponsors of prescription drugs that have sought to switch to nonprescription status, but for which FDA has been reluctant to approve the switch.  Other candidates specifically mentioned by FDA for the potential new paradigm are products whose effectiveness is dependent on a patient’s ability to obtain access to a drug soon after the onset of symptoms, e.g., rescue inhalers for the treatment of asthma or epinephrine for allergic reactions. 

The FDA is to be commended for actively exploring new ways to expand the availability of medications to consumers and to potentially reduce health care costs.  The questions posed by FDA suggest the agency is in the preliminary stages of considering this potential new paradigm. 

Among the important questions that will need to be considered and addressed in assessing the viability of the new paradigm are the type and extent of data that would be necessary to support an application to market products under the new approval mechanism, and the type and extent of equipment, resources and training that would be needed at the pharmacy or other retail establishment level.  As such, drug and device manufacturers, health care practitioners, pharmacies and other interested parties should consider presenting at the public meeting and/or submitting written comments regarding this proposal.