The new PDUFA review model might be changed mid-stream based on data compiled by the industry trade groups who helped create it.

The showcase provision of the 2012 prescription drug user fee act reauthorization extends new molecular entity NDA and BLA application reviews two months and creates mid- and late-cycle communications between the agency and sponsors.

The “Program” as it is known at FDA, is nearing its ninth month in use, and the Pharmaceutical Research and Manufacturers of America and Biotechnology Industry Organization have started tracking participating applications to ensure the model is working as intended.

They created a secure database, which went live April 1, where both groups’ members can enter data describing how their applications move through the system.

As trends emerge, both groups intend to use the data to urge participants to make changes if the model is not functioning as intended.

“We cannot wait for five years to make course corrections,” Sascha Haverfield, PhRMA VP of scientific and regulatory affairs, said during a June 25 conference call about the database. “We do not want to wait for two and a half or five years if we see major trends emerging to discuss those trends with the agency. It’s in no one’s interest to delay discussions on how we can improve the review process.”

PDUFA V, which launched Oct. 1, 2012, made the review time for participating sponsors eight months for priority review and 12 months for standard applications. The review clock does not start until 60 days after submission so the agency can conduct a filing review and ensure all portions of the package are as expected.

In return for the extension, sponsors can talk with FDA at mid- and late-cycle review meetings about problems that have arisen and potentially correct them. The program is intended to increase the rate of first-cycle approvals Also see "Buying Time: Industry Sacrifices Early To Gain Later With PDUFA V Review Model" - Pink Sheet, 1 October, 2012..

The first application approved using the new review model was Bayer AG/Algeta ASA’s Xofigo (radium Ra223 dichloride) for the treatment of castration-resistant prostate cancer Also see "Bayer/Algeta’s Xofigo Brings New Mechanism To Prostate Cancer Field" - Pink Sheet, 15 May, 2013..

Some parts of the PDUFA agreement cannot be changed because they are enshrined in law, but others, such as FDA or sponsor behavior within the model, can be adjusted, BIO Executive VP of Health Sara Radcliffe said during the conference call.

That could include asking for changes to the mid- and late-cycle communications if they are not having the intended effect.

“An example would be situations where either FDA or the companies has not introduced the right information earlier on in the process,” Radcliffe said. “So perhaps FDA has a concern that could have been raised earlier, instead it was raised later, and so the interactions with the company got off track. ... Those kinds of behaviors, I think if we see those trends, are things that we would certainly want to try and address.”

Participants Could See Aggregated Data

PhRMA and BIO said they received a commitment from all their members to voluntarily self-report information into the database about applications using the new model.

Haverfield said it will include items such as review outcome and timeliness of action, as well as measures of FDA-sponsor interactions, including meetings and letters. Sponsors should need one to three hours to complete the online module, he said.

Information in the database is secure and confidential. PhRMA and BIO staff can view it to research trends and make conclusions about program operations.

Sponsors also will be able to view aggregated information. Haverfield would not say how much data already has been collected.

“In order to preserve confidentiality and follow anti-trust guidelines, you have to have a sufficient number of applications in any one category,” he said. “We will be able to follow information down to the … review division level at FDA.”

No regulatory approval was needed to create the database because all information remains confidential and is blinded or aggregated based on the PhRMA Antitrust Compliance Guide, the group said.

When the Generic Pharmaceutical Association proposed the Accelerated Recovery Initiative, a program that would empower a third party to collect production data in an effort to predict and prevent drug shortages, it required Federal Trade Commission approval Also see "GPhA’s Drug Shortage Initiative Clears FTC Hurdle; Still Working With FDA" - Pink Sheet, 13 August, 2012..

Creating A Stronger Bargaining Position

It also appears the database will help the trade groups improve the industry bargaining position when formal talks begin for PDUFA VI.

In the past, PhRMA and BIO had asked FDA for application data to analyze and requested members’ experiences, but it was difficult to compile.

Radcliffe said the groups wanted a better way to compile their own data. She said BIO has collected data on specific issues previously, but not like what the tracking database will provide.

“By adopting this different approach, we’ll be able to work much more efficiently in terms of coming to the FDA with our thoughts,” she said. “It certainly isn’t a matter of not trusting the FDA. I think both sides will be collecting data in their own way and hope to identify trends mutually.”

During PDUFA V negotiations, industry argued for changes to make the review system more predictable. But FDA said its internal study found that application quality problems also decreased the likelihood of on-time action Also see "Want A Predicable Review For Your NDA? Use eCTD And Don't Amend It" - Pink Sheet, 1 June, 2011..

FDA also argued that it is the most predictable of all parts of the drug development process with a nearly 80% success rate Also see "FDA Still Most Predictable Part of Drug Development, Despite Bumps, Woodcock Says" - Pink Sheet, 1 June, 2011..

PhRMA and BIO likely want the ability to conduct similar studies to develop their negotiating positions and need more in-depth data to do it.

Radcliffe said ongoing data collection ideally could allow the reauthorization process to begin earlier.

“We’re talking about sustained engagement here and so we’re trying to think in a lifecycle way about PDUFA reauthorization rather than approaching it on an intermittent every-five-year basis,” she said.

PDUFA IV began in October 2007. The formal PDUFA V reauthorization process began with a public meeting in April 2010, ending in July 2012 when President Obama signed the FDA Safety and Innovation Act Also see "FDASIA Is Signed, Not That White House Wanted Anyone To Notice" - Pink Sheet, 16 July, 2012..

PhRMA, BIO’s PDUFA V Application Tracker Could Inform “Course Corrections”

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