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House bill that would require new clinical studies from sponsor for tropical disease priority review vouchers met with industry concerns about limiting the US agency's flexibility.


Efforts to prevent another Martin Shkreli-like abuse of FDA incentives are proving difficult, in part because of the potential effects on the US agency.


The Biotechnology Innovation Organization warned a proposed tweak to the tropical disease priority review voucher program intended to ensure a sponsor cannot simply purchase a drug already approved elsewhere and gain the US incentive could take away some of the agency's flexibility in its approval process.



The less-publicized provision in the Lower Drug Costs Through Competition Act also could wind up lengthening development and increasing the cost to develop treatments in the sector. It would mandate that the tropical disease voucher-eligible application include "reports of new clinical investigations (other than bioavailability studies) essential to the approval of the application and conducted or sponsored by the applicant," according to bill text.


A priority review voucher allows the holder to obtain a priority review for any application it chooses. Sponsors of treatments for certain neglected tropical diseases like Zika can be eligible for such a voucher upon approval. (Also see"Voucher Does Not Guarantee A Priority Review, FDA Says" - Pink Sheet, 5 Oct, 2016.) Applications already must be for the prevention or treatment of a tropical disease and be deemed eligible for priority review, as well as have an active ingredient that has not been approved in any other application.


Mandating an application include new clinical data could be "taking away from FDA the ability to decide on an application-by-application basis, what data are needed to provide an approval for a drug," said BIO's Kay Holcombe.

The bill's co-sponsor, Rep. Kurt Schrader, D-Ore., said during a recent House Energy and Commerce Health Subcommittee hearing that the provision is meant to close a loophole in the priority review voucher program.


"Some bad actors have announced plans to access priority review vouchers by buying the rights to manufacture a drug from overseas and then bring it back to the US for approval without having to do any additional research or development," Schrader said during the hearing, which covered the bill and FDA's generic drug and biosimilar user fee programs.


Schrader argued that the tropical disease priority review voucher (PRV) program is intended to incentivize new research and new drugs for the US market, not simply the transfer of applications already approved overseas.


But Kay Holcombe, BIO senior VP of science policy, told Schrader that mandating an application include new clinical data could be "taking away from FDA the ability to decide on an application-by-application basis, what data are needed to provide an approval for a drug."


Holcombe argued that a sponsor could have obtained approval outside the US first in a country where the tropical disease is prevalent and seeks US approval later to make the drug available to Americans traveling overseas.


"If there have been legitimate good, solid clinical studies that already have been done that are applicable to the US patients who are affected by this condition, FDA will decide that maybe we don't need additional clinical studies," she said. "If FDA has a different view, then of course they should be able to say to the company, you need to do new studies."


Indeed, forcing a sponsor to conduct more clinical studies could send costs high enough that development may be tougher to justify, even with the voucher.


Sponsors have chased vouchers because they have been selling for hundreds of millions of dollars, which can serve as an unrestricted stimulus for a company. But voucher prices have been dropping in recent months as more become available. (Also see "Gilead Buys Its Third Priority Review Voucher, But Is The Mania Over?" - Pink Sheet, 21 Feb, 2017.)


Will House Pass A Shkreli Bill?


Schrader's intent with the provision is most likely another attempt at dealing with recent high-profile drug price increases. Martin Shkreli, the infamous "pharma bro," had said after KaloBios Pharmaceuticals Inc. acquired benznidazole, an older Chagas disease treatment, that he planned to use it to seek a tropical disease voucher. (Also see "Shkreli's Empire Collapses: KaloBios Seeks Bankruptcy Protection" - Scrip, 31 Dec, 2015.)


Shkreli became the face of anger over the drug pricing issuewhen he engineered Turing Pharmaceuticals AG's price increase for Daraprim, and lawmakers raised the voucher program as potentially helping the so-called bad actors. (Also see "PRVs Revisited During Drug Pricing Hearing: FDA Still Doesn’t Like Them" - Pink Sheet, 5 Feb, 2016.)


Holcombe did not bring up cost in the exchange, and Schrader said he wants FDA to have the final say "using whatever appropriate studies are out there."


Schrader's bill aims to addresses drug pricing concerns buy creating a priority review pathway and voucher program for some generics, but those proposals have been meet with skepticism by FDA and ANDA sponsors. (Also see "Bill To Speed ANDA Approvals Gets Cool Reception From Industry, US FDA" - Pink Sheet, 2 Mar, 2017.)


Bill Among Many Candidates For User Fee Package


Nevertheless, Schrader's bill, co-sponsored by Rep. Gus Bilirakis, R-Fla., and others, appears to be a candidate for the upcoming user fee legislative package, which will include reauthorizations of the prescription drug and medical device fee programs, as well as those for generics and biosimilars.


The package remains in the early stages of its development and could wind up encompassing a number of other FDA-related issues, but it seems that drug pricing will be one of them.


Rare disease advocates also are pushing a bill that would grant additional exclusivity for drugs repurposed for orphan indications (Also see "Rare Disease OPEN-ing In PDUFA Possible For Drug Repurposing Advocates" - Pink Sheet, 2 Mar, 2017.), but that legislation may be open to the same criticism over pricing and incentive abuse that is being leveled against Shkreli and others.


Indeed, orphan drugs are increasingly being seen as a big pricing problem. Lawmakers want answers from Marathon Pharmaceuticals LLC about their pricing of Emflaza (deflazacort), a corticosteroid previously available for years in Europe that was recently approved by FDA for use in Duchenne muscular dystrophy.


The company received orphan exclusivity as well as a rare pediatric disease priority review voucher, and priced the product much higher than it cost US patients to import (Also see "Marathon's High-Priced Window May Not Stay Open Long" - Scrip, 13 Feb, 2017.). Amid the anger and pressure, Marathon chose to hold its product launch. (Also see "Amid Pricing Criticism, Marathon 'Pausing' Emflaza Launch" - Scrip, 13 Feb, 2017.)

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