HHS reg­u­la­to­ry agen­da for 2021 is light on new pro­posed and fi­nal FDA rules

The im­pact of the coro­n­avirus on the FDA’s work plans is ev­i­dent in the lat­est se­mi-an­nu­al reg­u­la­to­ry agen­da for HHS, which in­cludes on­ly two new bio­phar­ma-re­lat­ed pro­posed rules and one fi­nal rule.

The two pro­posed rules cen­ter on one that seeks to change FDA med­ica­tion guide reg­u­la­tions and an­oth­er to clar­i­fy FDA’s study con­duct ex­pec­ta­tions for an­a­lyt­i­cal and clin­i­cal phar­ma­col­o­gy, bioavail­abil­i­ty (BA) and bioe­quiv­a­lence (BE) stud­ies.

In the case of up­dat­ing FDA med­ica­tion guide reg­u­la­tions, the agency is seek­ing to re­quire drug spon­sors of out­pa­tient drugs to sub­mit a new form of pa­tient la­bel­ing to be re­viewed by the FDA.

“The pro­posed rule would in­clude re­quire­ments for Pa­tient Med­ica­tion In­for­ma­tion de­vel­op­ment and dis­tri­b­u­tion,” HHS says. “The pro­posed rule would re­quire clear and con­cise­ly writ­ten pre­scrip­tion drug prod­uct in­for­ma­tion pre­sent­ed in a con­sis­tent and eas­i­ly un­der­stood for­mat to help pa­tients use their pre­scrip­tion drug prod­ucts safe­ly and ef­fec­tive­ly.”

For the oth­er pro­posed rule that HHS ex­pects to un­der­take in the fore­see­able fu­ture, FDA is propos­ing to amend 21 CFR 320, in cer­tain parts, and es­tab­lish a new 21 CFR 321 to clar­i­fy FDA’s study con­duct ex­pec­ta­tions for an­a­lyt­i­cal and clin­i­cal phar­ma­col­o­gy, BA and BE stud­ies that sup­port hu­man re­search and mar­ket­ing ap­pli­ca­tions.

“The pro­posed rule would spec­i­fy need­ed ba­sic study con­duct re­quire­ments to en­able FDA to en­sure those stud­ies are con­duct­ed ap­pro­pri­ate­ly and to ver­i­fy the re­li­a­bil­i­ty of study da­ta from those stud­ies,” HHS said. “This reg­u­la­tion would align with FDA’s oth­er good prac­tice reg­u­la­tions, would al­so be con­sis­tent with cur­rent in­dus­try best prac­tices, and would har­mo­nize the reg­u­la­tions more close­ly with re­lat­ed in­ter­na­tion­al reg­u­la­to­ry ex­pec­ta­tions.”

Oth­er pre­vi­ous­ly pro­posed FDA rule­mak­ings, such as re­vi­sions around when the FDA can refuse a gener­ic drug ap­pli­ca­tion or the mod­ern­iza­tion of FDA’s bi­o­log­ics reg­u­la­tions, were not in­clud­ed in the lat­est HHS agen­da.

The one fi­nal FDA rule on the HHS’ agen­da this year deals with a pro­posed rule from 2019 that will amend the agency’s 503A Bulks List, which is a list of bulk drug sub­stances that can be used in com­pound­ing.

The fi­nal rule will add five ad­di­tion­al bulk drug sub­stances to the list, in­clud­ing Glu­taralde­hyde, Gly­col­ic acid, L-cit­rulline, Pyru­vic acid and Trichloroacetic acid. The rule will al­so iden­ti­fy 26 bulk drug sub­stances that FDA has de­cid­ed not to in­clude on the 503A Bulks List. Ad­di­tion­al sub­stances nom­i­nat­ed by the pub­lic for in­clu­sion on this list are cur­rent­ly un­der con­sid­er­a­tion and will be the sub­ject of a fu­ture rule­mak­ing, HHS said.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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