Rare pe­di­atric PRV pro­gram not linked to uptick in new drugs start­ing tri­als, study finds

The rare pe­di­atric pri­or­i­ty re­view vouch­er (PRV) pro­gram was not found to be as­so­ci­at­ed with an in­crease in the num­ber or rate of new rare pe­di­atric dis­ease drugs that be­gan clin­i­cal tri­als, a Health Af­fairs study found.

But the re­searchers from Har­vard Med­ical School, work­ing on what they said is the first study on the im­pact of the rare dis­ease PRV pro­gram on drug de­vel­op­ment, al­so found that their da­ta do pro­vide some en­cour­ag­ing news about rare pe­di­atric dis­ease drug de­vel­op­ment.

“Such [rare pe­di­atric] drugs were more like­ly to ad­vance from Phase I to Phase II tri­als, com­pared to drugs for rare adult dis­eases, af­ter the PRV pro­gram’s cre­ation, al­though the same dif­fer­ence was not ob­served for progress in lat­er stages of de­vel­op­ment,” they write.

The au­thors al­so found that com­pared to drugs for rare adult dis­eases, drugs for rare pe­di­atric dis­eases pro­gressed more quick­ly through all phas­es of clin­i­cal test­ing and were more like­ly to be first-in-class.

The study’s find­ings seem to con­tra­dict the ap­pear­ance that the rare pe­di­atric dis­ease PRV pro­gram is a suc­cess. From 2012 to to­day, the US Food and Drug Ad­min­is­tra­tion (FDA) has award­ed more than a dozen rare pe­di­atric dis­ease PRVs, which have con­sis­tent­ly sold for around $100 mil­lion each.

​But the au­thors re­main cau­tious about both the rare pe­di­atric and trop­i­cal dis­ease PRV pro­grams in gen­er­al, not­ing, “The rapid pro­lif­er­a­tion of these pro­grams must be con­sid­ered care­ful­ly,” par­tic­u­lar­ly as in 2016, the 21st Cen­tu­ry Cures Act cre­at­ed an­oth­er PRV pro­gram for med­ical coun­ter­mea­sures.

And giv­en the large num­ber of rare pe­di­atric dis­eases still with­out treat­ment op­tions (on­ly an es­ti­mat­ed 5% of rare pe­di­atric dis­eases have a treat­ment), the au­thors make the case for ad­di­tion­al poli­cies out­side the PRV pro­gram to bol­ster the de­vel­op­ment of new ther­a­pies.

“For ex­am­ple, new fund­ing could be di­rect­ed to the Na­tion­al In­sti­tutes of Health to ex­pand a col­lab­o­ra­tive pub­lic-pri­vate de­vel­op­ment part­ner­ship for rare pe­di­atric dis­eases,” they write.

The study’s find­ings come as Pres­i­dent Don­ald Trump dur­ing the State of the Union sig­naled his in­ten­tions to re­new ef­forts to de­vel­op new treat­ments for pe­di­atric can­cers, pledg­ing $500 mil­lion over 10 years.


First pub­lished here and syn­di­cat­ed in part­ner­ship with RAPS. Email news@raps.org for more in­for­ma­tion.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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José Baselga, AstraZeneca cancer chief (Brent N. Clarke/FilmMagic via Getty Images)

As­traZeneca's Calquence nabs an­oth­er win against Im­bru­vi­ca, but Eli Lil­ly is on its heels

Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

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Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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Jean-Christophe-Hyvert, Lonza

Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.