Decades of di­ver­si­ty ini­tia­tives lat­er, cer­tain groups re­main 'con­sis­tent­ly un­der­rep­re­sent­ed' in can­cer tri­als, GAO says

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,” the Gov­ern­ment Ac­count­abil­i­ty Of­fice said in a new re­port re­leased this week.

The GAO re­port ex­plains many of the fed­er­al ef­forts around in­creas­ing di­ver­si­ty, par­tic­u­lar­ly in fed­er­al­ly-fund­ed can­cer tri­als, with DOD, HHS, and VA de­vel­op­ing re­search col­lab­o­ra­tions, mod­i­fy­ing re­search prac­tices, re­duc­ing bar­ri­ers to pa­tient par­tic­i­pa­tion with pay­ments and via oth­er means, and col­lect­ing and shar­ing more da­ta.

But there are still ma­jor gaps.

For ex­am­ple, Con­gress man­dat­ed that the NIH in­clude women and per­sons from racial and eth­nic groups oth­er than non-His­pan­ic white pop­u­la­tions in clin­i­cal tri­als, but an analy­sis that searched NIH’s clin­i­cal tri­als data­base in Jan­u­ary 2013 found that NIH’s Na­tion­al Can­cer In­sti­tute spon­sored or co-spon­sored 10,000 clin­i­cal tri­als, and that less than 2% of these tri­als fo­cused on racial or eth­nic groups oth­er than non-His­pan­ic white pop­u­la­tions.

The re­port al­so high­lights one study of Black men — whose in­ci­dence rate of prostate can­cer is 70% greater than that of white men and whose mor­tal­i­ty rate is 2.5 times high­er than white men —”looked at avail­abil­i­ty of prostate can­cer clin­i­cal tri­als by coun­ty and pop­u­la­tion. It found that the coun­ties with a high­er pro­por­tion of Blacks were less like­ly to have ac­cess to both can­cer fa­cil­i­ties and prostate can­cer tri­als,” GAO notes.

The cost bur­den as­so­ci­at­ed with can­cer tri­als can al­so push some of these un­der-served pop­u­la­tions away. An­oth­er study cit­ed by GAO found that over­all, 48% of the pa­tients in can­cer clin­i­cal tri­als had month­ly out-of-pock­et costs of at least $1,000, forc­ing many pa­tients to turn to sav­ings, re­tire­ment ac­counts, bor­row­ing mon­ey from friends and fam­i­ly, hold­ing a fundrais­er, and work­ing ex­tra hours or an ad­di­tion­al job.

But fed­er­al agen­cies have sought to in­crease the en­roll­ment of di­verse groups of pa­tients with rec­om­men­da­tions for re­searchers on set­ting min­i­mum re­quire­ments for tri­al en­roll­ment, broad­en­ing pa­tient el­i­gi­bil­i­ty cri­te­ria, and in­ten­tion­al en­roll­ment of un­der­rep­re­sent­ed pop­u­la­tions.

The GAO re­port comes as Con­gress re­cent­ly at­tached sev­er­al pro­vi­sions to the year-end spend­ing bill that aim to in­crease tri­al di­ver­si­ty. Mov­ing for­ward, spon­sors will be re­quired to sub­mit 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 the 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.

Out­side of the fed­er­al gov­ern­ment, the GAO al­so found that 17 non-fed­er­al can­cer cen­ters put to­geth­er a range of prac­tices de­signed to fa­cil­i­tate en­roll­ment of pa­tients from di­verse back­grounds in can­cer clin­i­cal tri­als. GAO re­viewed prac­tices falling in­to four cat­e­gories: (1) or­ga­ni­za­tion-lev­el prac­tices, (2) com­mu­ni­ty-lev­el prac­tices, (3) work­force-lev­el prac­tices, and (4) pa­tient-lev­el prac­tices, and said that of the 17 cen­ters re­viewed, 15 not­ed im­ple­ment­ing prac­tices in at least three of the four cat­e­gories.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Mass­a­chu­setts judge dis­miss­es law­suit against Bio­gen over failed launch of Alzheimer's drug Aduhelm

A Massachusetts federal judge on Wednesday dismissed a class action lawsuit filed by investors against Biogen and several of its current and former executives over the company’s failed Alzheimer’s drug, Aduhelm (aducanumab).

The investors argued that Biogen’s contact with the FDA was unlawful and that the company made 25 false and misleading statements, including statements about the rollout and price of the drug.

Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”