UK Health Secretary Sajid Javid (Jack Taylor/Getty Images)

Prep­ping for yet more pricey rare dis­ease drugs, UK launch­es £340M 'In­no­v­a­tive Med­i­cines Fund'

The UK is ex­pand­ing its con­tro­ver­sial Can­cer Drugs Fund in­to rare dis­ease.

On Wednes­day, the coun­try’s Na­tion­al Health Ser­vice an­nounced a new £340 mil­lion ef­fort called the In­no­v­a­tive Med­i­cines Fund to cov­er the cost of new rare dis­ease drugs that don’t yet have enough da­ta to sat­is­fy the gov­ern­ment’s pric­ing watch­dog but might still of­fer some ben­e­fit for pa­tients. Like the sim­i­lar, decade-old can­cer ef­fort, reg­u­la­tors would then col­lect da­ta to “en­sure their pric­ing and long term use rep­re­sents good val­ue for the NHS over­all.”

The new fund comes as the UK, like many coun­tries, grap­ples with the flur­ry of rare dis­ease drugs phar­ma and biotech com­pa­nies are now bring­ing to mar­ket, and the high price tags they’ve con­sis­tent­ly at­tached to those med­i­cines. The coun­try spent years locked in an ac­ri­mo­nious bat­tle with Ver­tex on their cys­tic fi­bro­sis drugs and on­ly in the last few months se­cured ac­cess to No­var­tis’ SMA gene ther­a­py Zol­gens­ma, of­ten known sim­ply as “the most ex­pen­sive drug in the world.”

And that’s just in the UK, a coun­try that’s his­tor­i­cal­ly been more will­ing than its brethren across the chan­nel to pay for pricey drugs. Ear­li­er this year, blue­bird pulled its gene ther­a­py for a rare blood dis­or­der from Ger­many af­ter the coun­try re­fused to pay $900,000, a po­ten­tial har­bin­ger for how coun­tries — and com­pa­nies — will han­dle ne­go­ti­a­tions over one-and-done gene ther­a­pies.

The new £340 mil­lion adds to £340 mil­lion al­ready al­lot­ted to the Can­cer Drugs Fund. Al­though it is tech­ni­cal­ly for all pa­tients, NHS sin­gled out the po­ten­tial in rare and ge­net­ic dis­eases.

The fund, it said, could cov­er “cut­ting-edge gene ther­a­pies,” par­tic­u­lar­ly in es­pe­cial­ly rare dis­eases where there may be too few pa­tients to col­lect ef­fi­ca­cy da­ta in a time­ly man­ner. In ad­di­tion to Zol­gens­ma, gene ther­a­pies have reached or are near­ing ap­proval for sick­le cell dis­ease, in­her­it­ed forms of blind­ness, he­mo­phil­ia and a cou­ple of ul­tra-rare im­muno­log­i­cal and neu­ro­log­i­cal dis­or­ders, among oth­ers.

The fund “will sig­nif­i­cant­ly re­duce the time it takes for the most promis­ing new med­i­cines to reach pa­tients,” health sec­re­tary Sajid Javid said in a state­ment, “in­clud­ing chil­dren and those with rare dis­eases, sav­ing lives and giv­ing many peo­ple hope for a health­i­er fu­ture.”

The ap­proach an­swers a long-run­ning crit­i­cism that has dogged the Can­cer Drugs Fund since it was con­ceived in 2010 as a way to cov­er drugs that reg­u­la­tors deemed not cost-ef­fec­tive: Why have a fund for can­cer pa­tients, but not for pa­tients with oth­er se­ri­ous dis­or­ders?

Now, Javid said, the UK will be “bring­ing equal ac­cess to the best treat­ments for all, re­gard­less of the con­di­tion.”

Oth­er crit­i­cisms, though, still linger. In 2017, amid a tor­rent of push­back that the fund had grown in­to a more than £1 bil­lion boon­dog­gle for phar­ma com­pa­nies with in­ef­fec­tive or mar­gin­al­ly ef­fec­tive drugs, the gov­ern­ment re­struc­tured the process to more re­sem­ble the ac­cel­er­at­ed ap­proval path­way in the US, where drugs are ap­proved on lim­it­ed da­ta and then in the­o­ry can be ap­proved or pulled af­ter more da­ta on sur­vival or oth­er clin­i­cal out­comes emerge.

That path­way, though, re­mains con­tro­ver­sial in the US, par­tic­u­lar­ly af­ter it was used to ap­prove an un­proven Alzheimer’s treat­ment last month. And it has re­ceived push­back in the UK, al­beit far milder than when the fund was un­der the orig­i­nal mod­el.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.