Bill Looks to Establish Provisional Approval Framework for Some Products

Posted 09 August 2012 | By Alexander Gaffney, RAC 

A new piece of legislation proposed in the House of Representatives would amend the Federal Food, Drug and Cosmetic Act (FDCA) to allow for the US Food and Drug Administration (FDA) to issue tentative approvals for products given fast track designation.

The bill, H.R. 6288 - The Patient Choice Act of 2012, is sponsored by Rep. Brian Bilbray (R-CA) and currently is co-sponsored by Reps. Dan Boren (R-OK), Morgan Griffith (R-VA), Duncan Hunter (R-CA) and Jean Schmidt (R-OH).

"It is inexcusable that patients have to travel overseas to receive the treatment they need," stated Bilbray in a statement.  "While we continue to streamline the approval process at home, we should recognize the advances made in patient care in other countries that have rigorous approval standards similar to our FDA."

Bilbray said in a separate statement emailed to congressmen that he had seen, "A surge in companies leaving the US and heading overseas to address this issue," and that long review times and regulatory uncertainty were key drivers in this commercial exodus.

Provisional Approvals under the Act

Under the act, the FDA Commissioner would be required within 90 days-if asked by the product's sponsor-to grant "provisional approval" to drug products designated as a "fast track" product under the law so long as it has been determined to be "adequately safe."

The Act defines a "safe" product as one with a risk of death less than that presented by the disease and its secondary effects. Alternatively, a product is "safe" if it is less risky than a product approved for the same condition. Similarly, if a drug has been marketed in an approved foreign country for more than four years, that data may also be used to support the safety of a drug.

The provisional approval would allow the product to enter interstate commerce-currently only allowed for fully approved products or ones undergoing clinical testing under an approved investigational new drug application.

The bill notes the treatment of the drug is effectively the same as it would be under the sections 505 and 351 of the Public Health Service Act, both of which set the requirements for new drug and biologic products.

Informed Consent and Removal from the Market

Unlike normal drug products given full approval by the agency, marketers of provisionally approved products would be required to obtain written, informed consent from all patients receiving the drug and would also need to continue to obtain safety and efficacy data for the drugs. The bill notes the requirements for this consent would be similar to those in place under Title 21, Part 50 of the Code of Federal Regulations.

The products would also be subject to a five-year period of conditional approval, after which it would be required to pull the product off the market unless FDA determines the sponsor is, "Diligently engaging in actions for the purpose of seeking approval," or requires additional time to obtain approval. The latter could be particularly important for companies given additional requests for long-term Phase III clinical trial requests, some of which can be difficult to find the necessary number of patients to achieve statistical significance.

An additional caveat may be granted by FDA if the product is necessary for the protection of-or if its removal would harm-public health. Any drug found on the market after the time of removal would be classified as misbranded and subject to appropriate action by both FDA and the US Department of Justice.

Continued Momentum

The bill notes, "A sponsor of a drug that receives a provisional approval … shall continue to diligently conduct appropriate studies, after such provisional approval is granted."

All marketing materials to be used by the company will be required to be submitted to FDA 30 days in advance of their use, though prior approval will not be required from FDA for the materials.

Depending on their risk profile, some drugs will also be required to implement a Risk Evaluation and Mitigation Strategies (REMS) plan, which can include black box warnings, patient education plans, restricted prescribing authority, physician education programs and other factors.

The drug's approval would not have any effect on marketing exclusivity provisions contained within federal law, with any additional time on the market being construed as being, "An addition to the applicable period of marketing exclusivity for such drug."


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