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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Some can­cer pa­tients now have to find oth­er op­tions as Bris­tol My­er­s' Abrax­ane falls in­to short­age from man­u­fac­tur­ing woes

When Beth Hogan, a metastatic pancreatic cancer patient, showed up for her infusion at Yale’s Smilow Cancer Hospital in New Haven, CT on Oct. 11, she said she was informed that day that she would not be receiving Bristol Myers Squibb’s Abraxane, part of her combo treatment, because of a shortage.

“I was told we don’t know when you can have it,” she told Endpoints News via email, adding that she doesn’t expect to receive any Abraxane this coming Monday at her treatment appointment either, and she doesn’t know when things will change.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Pascal Soriot, AstraZeneca CEO (via Getty images)

As­traZeneca's MCL-1 can­cer drug put on hold af­ter flag­ging a safe­ty is­sue — 2 years af­ter Am­gen axed ri­val drug

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca is now flagging safety issues for a rival therapy of the same class.

The pharma giant noted on that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.” There was no further explanation and a spokesperson for AstraZeneca hadn’t followed up with us ahead of our publishing deadline.

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FDA shoots down a biotech’s last-stand pa­tient pitch af­ter fault­ing da­ta

Stealth BioTherapeutics appeared unsure its pitch for an ultra-rare disease would be accepted when submitting an NDA in August, and on Wednesday its fears came to pass.

The FDA issued Stealth a refusal to file letter for a candidate looking to treat Barth syndrome, the biotech announced Wednesday morning. It’s a move not entirely unexpected, given Stealth’s own admission earlier this year that the agency didn’t find existing data to support NDA review.

Kelly Martin, Radius Health CEO

Ra­dius rock­ets high­er as Kel­ly Mar­tin boasts of a big PhI­II suc­cess — but the spoils be­long to Menar­i­ni

Radius Health stuck with some fuzzy top-line Phase III results for its oral SERD therapy elacestrant, but investors saw enough to push a rally that sparked a big surge in its share price.

According to researchers, the drug hit both primary endpoints among ER+/HER2- breast cancer patients: beating standard of care on progression-free survival in the overall population and PFS with tumors harboring estrogen receptor 1 mutations — a key factor in developing resistance. And while some analysts were left wondering about specific data, most of the crowd seemed happy to hear that the drug is now being steered to the FDA.

Raymond Stevens, ShouTi Pharma CEO

A new Schrödinger-backed start­up emerges from the sci­en­tist who mapped the first hu­man GPCR

One of the most popular targets in drug development, representing about a third of existing drugs, are G-protein coupled receptors — the tiny but integral membrane proteins responsible for recognizing things like light, taste, smell, hormones and pain.

But due to challenges in mapping their structure, the protein family remains largely unexplored.

A slate of companies has emerged over the last few years to change that. If one can figure out the structure of these elusive membrane receptors, it might be possible to create small molecule drugs that overcome the limitations of, say, biologic and peptide therapies. That promise is what gets serial entrepreneur Raymond Stevens out of bed in the morning.

Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.