On­ly 1 of 25 can­cer drug de­vel­op­ers fair­ly in­clud­ed mi­nor­i­ty pa­tients over five-year win­dow, BMJ analy­sis finds

New clin­i­cal tri­al di­ver­si­ty plans, which FDA will soon ex­plain fur­ther, were fea­tured in the gov­ern­ment spend­ing pack­age that Pres­i­dent Joe Biden signed late last week and are com­ing at a time when they’re very much need­ed, ac­cord­ing to a new analy­sis pub­lished in the BMJ to­day.

Of the 25 com­pa­nies re­viewed for the new di­ver­si­ty rank­ings, Unit­ed Ther­a­peu­tics was the on­ly one with a per­fect fair in­clu­sion score of 100, but sev­en oth­ers — Puma Biotech­nol­o­gy, Sanofi, Take­da, Am­gen, Bris­tol My­ers Squibb, Eli Lil­ly, and Mer­ck KGaA — re­ceived a score in the top quar­tile on clin­i­cal tri­al di­ver­si­ty per­for­mance, ac­cord­ing to the analy­sis from Stan­ford, Yale and Bioethics In­ter­na­tion­al re­searchers, which was sup­port­ed by a grant from the Su­san G. Komen Foun­da­tion.

More than half of the 25 spon­sors par­tic­i­pat­ing (56%) fair­ly in­clud­ed women, the re­searchers said, but just five spon­sors (20%) fair­ly in­clud­ed old­er adults and on­ly one spon­sor (4%) fair­ly in­clud­ed racial­ly and eth­ni­cal­ly mi­nori­tized pa­tients in tri­als. For in­di­vid­ual prod­ucts’ piv­otal tri­als, the analy­sis found that 80% fair­ly in­clud­ed women, 24% fair­ly in­clud­ed old­er adults, and 5% fair­ly in­clud­ed racial­ly and eth­ni­cal­ly mi­nori­tized pa­tients.

At the prod­uct lev­el, 42% (25/59) of the can­cer ther­a­peu­tics re­viewed in­clud­ed trans­par­ent da­ta on the pro­por­tion of racial and eth­nic mi­nor­i­ty par­tic­i­pants in their tri­als, the re­search showed.

The so-called fair in­clu­sion score, which is in­formed by da­ta from 64 piv­otal clin­i­cal tri­als of nov­el drugs and bi­o­log­ics ap­proved be­tween 2012 and 2017, con­sid­ers trans­paren­cy of re­port­ing and the rep­re­sen­ta­tion of three de­mo­graph­ic groups that are of­ten un­der­rep­re­sent­ed in clin­i­cal tri­als in com­par­i­son to the dis­ease-spe­cif­ic pop­u­la­tion: sex (per­cent­age of par­tic­i­pants that were fe­male), age (per­cent­age of adults old­er than 64) and race/eth­nic­i­ty (per­cent­age of par­tic­i­pants iden­ti­fy­ing as Black, Asian and/or Lat­inx).

Jen­nifer Miller

“The lack of di­ver­si­ty in clin­i­cal re­search is a so­cial jus­tice and a pub­lic health prob­lem,” co-au­thor Jen­nifer Miller, founder of Bioethics In­ter­na­tion­al and an as­so­ciate pro­fes­sor at Yale School of Med­i­cine, said in a state­ment.

While on­ly a few spon­sors have done well in the rank­ings, the au­thors not­ed that “most have sub­stan­tial room for im­prove­ment on their in­clu­sion of old­er adults and racial­ly and eth­ni­cal­ly mi­nori­tized pa­tients, and to a less­er ex­tent women.”

The rank­ings fol­low a Gov­ern­ment Ac­count­abil­i­ty Of­fice re­port from last month that found that af­ter more than three decades of in­sti­tut­ing gov­ern­ment poli­cies to im­prove clin­i­cal tri­al di­ver­si­ty, cer­tain racial and eth­nic groups, as well as ado­les­cents, old­er adults, women, low-in­come in­di­vid­u­als, and in­di­vid­u­als from rur­al com­mu­ni­ties “re­main con­sis­tent­ly un­der­rep­re­sent­ed in can­cer clin­i­cal tri­als.”

Mov­ing for­ward, spon­sors will soon be re­quired to sub­mit to FDA a “di­ver­si­ty ac­tion plan” for Phase III tri­als, in­clud­ing spec­i­fy­ing en­roll­ment goals up­front.

But the FDA has the abil­i­ty to waive such a re­quire­ment if an in­di­ca­tion is too small or for oth­er dis­cre­tionary rea­sons. Con­gress is man­dat­ing that FDA pub­lish guid­ance on such ac­tion plans with more de­tails, in­clud­ing on whether to grant a spon­sor’s re­quest to waive the re­quire­ment.

The agency will have two years to is­sue a re­port on what the di­ver­si­ty ac­tion plans have done up un­til then, in terms of boost­ing the en­roll­ment of mi­nori­ties and typ­i­cal­ly un­der­rep­re­sent­ed pop­u­la­tions.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.