Are pri­or­i­ty re­view vouch­er pro­grams stim­u­lat­ing drug de­vel­op­ment? The da­ta sug­gest not re­al­ly, finds GAO re­port

What is the im­pact of the three pri­or­i­ty re­view vouch­er pro­grams (PRV) on drug de­vel­op­ment? Not much, ac­cord­ing to da­ta an­a­lyzed by Con­gres­sion­al watch­dog GAO.

PRV pro­grams of­fer com­pa­nies a perk in the form of a vouch­er when a new treat­ment — in the field of rare pe­di­atric dis­ease, trop­i­cal dis­ease or med­ical coun­ter­mea­sure — is ap­proved. These vouch­ers can be used to has­ten the re­view of an­oth­er drug or be sold. Al­to­geth­er, the three pro­grams: trop­i­cal dis­ease PRV (au­tho­rized in 2007), the rare pe­di­atric dis­ease PRV (sanc­tioned in 2012) and the med­ical coun­ter­mea­sure PRV (backed in 2016) have led to the de­ploy­ment of a to­tal of 31 PRVs — of which 17 have been sold for vary­ing sums, rang­ing from $67.5 mil­lion to $350 mil­lion.

Af­ter con­duct­ing a lit­er­a­ture re­view, the GAO found some large­ly dis­heart­en­ing da­ta on their im­pact on drug de­vel­op­ment. A 2017 study found that not on­ly did the trop­i­cal dis­ease PRV pro­gram not re­ju­ve­nate the pipeline for trop­i­cal dis­ease drugs — but that the pro­por­tion of trop­i­cal dis­ease drugs among all drugs-in-de­vel­op­ment ac­tu­al­ly fell a smidge fol­low­ing the cre­ation of the PRV pro­gram. A 2018 study found that con­sid­er­ing the ma­jor­i­ty of med­ical coun­ter­mea­sures un­der clin­i­cal de­vel­op­ment al­ready get di­rect or in­di­rect fed­er­al fund­ing, al­ter­na­tives oth­er than the PRV pro­gram could bet­ter stim­u­late drug de­vel­op­ment in the field.

And fi­nal­ly, a 2019 study found that al­though the rare pe­di­atric dis­ease PRV pro­gram did not en­hance the num­ber or rate of new pe­di­atric dis­ease drugs that start­ed or com­plet­ed clin­i­cal tri­als, drugs that could be el­i­gi­ble for a rare pe­di­atric dis­ease PRV were more like­ly to ad­vance from Phase I to Phase II clin­i­cal tri­als when com­pared to rare adult dis­ease drugs.

But when GAO spoke to a slew of drug­mak­ers, the re­ac­tions to in­cen­tives of­fered by PRV pro­grams were large­ly pos­i­tive, sug­gest­ing that the PRV pro­grams were a fac­tor in drug de­vel­op­ment de­ci­sions. Re­searchers, on the oth­er hand, had mixed re­views. Over­all, drug spon­sors, re­searchers and oth­er stake­hold­ers sug­gest­ed that the sheer num­ber of PRVs award­ed, and the de­clin­ing rev­enue from their sales, could im­pact the pro­grams’ abil­i­ty to in­cen­tivize. There was no con­sen­sus on whether the rare pe­di­atric dis­ease and med­ical coun­ter­mea­sure PRV pro­grams — set to ex­pire by 2022 and 2023 — should be reau­tho­rized.

Pre­scrip­tions for im­prove­ment were al­so of­fered. Some stake­hold­ers took is­sue with the way the trop­i­cal dis­ease PRV is award­ed — be­cause it can be giv­en to com­pounds that have been pre­vi­ous­ly ap­proved out­side the Unit­ed States once they are sanc­tioned for use in the Unit­ed States. For ex­am­ple, No­var­tis won a PRV af­ter se­cur­ing FDA ap­proval for Egat­en, a treat­ment for fas­ci­o­lia­sis in 2019 — even though the drug has been on the WHO’s es­sen­tial med­i­cines list since 2002 and has been ap­proved well be­fore 2019 in oth­er re­gions.

An­oth­er loop­hole crit­i­cized was that drug­mak­ers award­ed a PRV have no oblig­a­tion to make the ap­proved drug avail­able at an af­ford­able price — and some sug­gest­ed that a man­dat­ed ac­cess plan that ac­com­pa­nies the PRV could en­hance ac­cess for pop­u­la­tions that need it. Some stake­hold­ers al­so sug­gest­ed award­ing PRVs to fi­nan­cial­ly strapped or­ga­ni­za­tions, like non-prof­its, to off­set de­vel­op­ment costs.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the presidential election, the FDA has issued a final regulation fulfilling President Trump’s promise to let states import certain prescription drugs from Canada.

On Thursday, Trump told a crowd in North Carolina that the new rule goes into effect “today.” But the published regulation states that it won’t take effect for 60 days. And even then, it could be a while before cheaper drugs make it across the border.

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.