Deregulatory Mood Spices PDUFA Reauthorization

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Includes: BBC, BBH, BBP, FBT, HQL, IBB, LABD, LABS, LABU, PBE, UBIO, XBI, ZBIO
by: EP Vantage

The US government’s authority to collect and spend FDA new drug user fees runs out on September 30, and this year’s bid to renew the programme comes against a backdrop of a White House pledge to trim requirements on drugmakers massively.

After consulting patient and industry representatives the agency outlined a set of performance objectives it would try to meet should Congress allow it to continue charging fees and using the revenue to pay drug review staff; among the proposals are greater use of adaptive trial designs and patient-directed endpoints. However, with an FDA commissioner yet to be named and President Donald Trump pledging to slash agency regulations, plans to move forward with the Prescription Drug User Fee Act (PDUFA) reauthorisation remain uncertain.

Revenge of the sixth

If it goes ahead, this would be the sixth passage of PDUFA authority by Congress since the legislation first passed in 1992 – hence the shorthand PDUFA VI. The funding stream has allowed the FDA to hire more dedicated drug review staff, which has led to a dramatic decrease in approval times; the average time now sits at 10 months (Faster FDA adds a positive spin to 2016's disappointing approval record, January 18, 2017).

The last iteration of PDUFA – effective from 2012 to September 30, the end of the 2017 fiscal year – created the breakthrough therapy designation and a mechanism for patient input into drug development.

FDA staff began meetings with patient and biopharma sector representatives in mid-2015, and in August 2016 outlined a proposal for its commitments under PDUFA VI.

Among these are: development of staff capacity for evaluating adaptive trial designs and Bayesian statistical analysis; intensified discussion with drug developers about trial designs that use biomarkers as surrogate endpoints; beefed up staffing to accommodate requests for breakthrough therapy designation; and use of patient-centred measures in drug development and review (Vantage point – Patient-reported data gain traction in FDA decisions, June 8, 2016).

As a carefully negotiated package, this will likely serve as the core of reauthorisation legislation – although it should be noted that the breakthrough designation was incorporated into the last PDUFA renewal only on Capitol Hill, so similar additions could be made.

Episode VI

That is, when it moves. There do not appear to be any congressional hearings scheduled for PDUFA VI. In any case, Mr. Trump has yet to nominate an FDA commissioner to help guide the legislation, and it is not necessarily clear how PDUFA VI and his anti-regulatory initiative can co-exist.

If Mr. Trump is to be taken at his word his administration would cut FDA regulations by 99%, as he told the pharmaceutical executives he met on January 31 (Speedy US drug reviews would make payers the new gatekeepers, February 1, 2016).

The new commissioner will probably want to put Mr. Trump’s imprimatur on FDA regulation, although any such initiative could be independent of PDUFA VI. Eric Gascho, vice-president of policy and government affairs with the National Health Council, told EP Vantage that he believed the industry wanted the current PDUFA VI commitments agreed with the FDA and patient groups to remain intact.

The idea of FDA deregulation and PDUFA reauthorisation being considered simultaneously would have been unthinkable five years ago, the last time the law was renewed. But as with all things in American government, it is necessary to readjust views on what is possible.