The FDA has issued emergency use authorization for Gilead Science Inc's (GILD) antiviral drug Remdesivir for the treatment of COVID-19 in adults and children hospitalized with severe disease.

Today's Daily Dose brings you news about the revised FDA decision date for Bristol Myers' CAR T cell therapy for refractory large B-cell lymphoma; Enanta Pharma's primary biliary cholangitis trial results; MacroGenics' anticipated clinical data read-outs and regulatory event for this year and Trovagene's name change.

Today's Daily Dose brings you news about FDA approval of Immunomedics' breast cancer drug; promising results from Kiniksa Pharma's prurigo nodularis trial; Mallinckrodt's regulatory catalyst; Myovant Sciences' phase III SPIRIT 2 study results and another disappointment in Parkinson's disease drug development space.

The U.S. Food and Drug Administration granted marketing authorization to Medtronic Inc.'s CARPEDIEM System, a new device for continuous dialysis therapy for use in pediatric patients with certain kidney conditions. The system is indicated to provide continuous hemodialysis or hemofiltration therapy to critically ill pediatric patients weighing between 2.5 and 10 kilograms.

Life sciences company LabCorp (LH) announced Tuesday that it has received an Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its COVID-19 at-home test kit.

Today's Daily Dose brings you news about the acquisition of Portola by Alexion; encouraging results from TG Therapeutics' chronic lymphocytic leukemia trial; Akebia's INNO2VATE trial results; and Vapotherm's Q1 financial results.

These are exciting times for Vapotherm Inc. (VAPO) whose share price has almost doubled in value since the beginning of this year.

Today's Daily Dose brings you news about Blueprint Medicines' disappointing results from gastrointestinal stromal tumor trial; revised timeline of Clearside Biomedical's resubmission of XIPERE NDA; encouraging trial results of INOVIO's MERS coronavirus vaccine and initial data from Syndax Pharma's acute leukemia trial.

More than 2 million Americans suffer from opioid use disorder, but only about 25% of people receive any sort of care. For many, inpatient treatment often means leaving a job and loved ones behind to seek recovery.

BASF recently announced the launch of Accelon™ absorption accelerating technology, developed as a breakthrough solution to the bioavailability challenge of today’s omega-3 supplements.

Probi's largest clinical trial ever, focused on probiotic immune health, has recently been completed.

Nutrition 21, LLC has announced its Nitrosigine® is now featured in EVERTRAIN PRE, a recently launched pre-workout supplement designed to provide athletes with sustained energy, superior focus and increased endurance.

Another leading athlete has joined Arla Foods Ingredients’ growing number of brand ambassadors for Lacprodan® HYDRO.365.

Valensa International announced Non-GMO Project has been awarded to Valensa’s Organic Spirulina, Chlorella and Microalgae products.

New Legal Motion Challenges Tony Huge’s Supposed Unpaid Role in Enhanced Athlete

Bergstrom Nutrition, manufacturer of OptiMSM®, a branded form of methylsulfonylmethane (MSM), recently published an article detailing how the small intestine absorbs MSM, particularly in relation to sulfur.

It is "inconceivable" that immunity will not remain high on the list of health priorities when this pandemic ends and now is the time to create more "convincing experiences" to ensure trial turns into adoption, according to food and drink research and branding experts.

Older adults who consumed large amounts of flavonoid-rich foods were two to four times less likely to develop Alzheimer's disease and related dementias over 20 years compared with people whose intake was low, in a study of 2,800 people.

Rousselot, the collagen-based ingredients producer, has revealed a new study which it says provides important answers surrounding the bioavailability of collagen peptides and the modifications they undergo during digestion.

As a sponsor or CRO, you understand the importance of a thorough site selection process. A site needs to be able to meet enrollment targets and time frames, protect the rights and safety of study participants, execute the protocol, deliver quality data, and maintain GCP compliance. That’s what your site feasibility surveys and pre-study visits are designed to evaluate. And as you’re assessing a site’s abilities, the site is conducting its own feasibility process. They’re mining their patient database and assessing inclusion/exclusion criteria. They’re reviewing staff credentials and ensuring they have adequate resources to manage the number of subject visits and collect the data the protocol requires.

But when we conduct GCP audits, we find there’s one perspective that is sometimes overlooked by both sides: the needs of the study drug itself.




Study Drug Attributes Affecting Site Selection Process

IP Environment.  Aside from needing sufficient storage space, many drugs have special storage requirements. Does the site have the equipment and resources needed to maintain and adequately monitor and record environmental conditions such as temperature or humidity? Do they have agreements with their vendors that guarantee a specific response time for repairing or replacing faulty equipment? If they lose electricity, do they have back up power, or at least provisions to move the IP off-site? (This is a common auditor question in hurricane-prone areas.)


Preparation of Study Drug.  Does your investigational product need to be reconstituted in a liquid? Do doses need to be compounded in different concentrations? Does the protocol require that an IV solution be prepared, filtered, and sterilized? These activities take time, specially trained personnel, and sometimes specialized equipment such as ventilation hoods. If your protocol demands an involved IP prep, your feasibility survey must include questions that allow you to assess these site capabilities and your pre-study visit should definitely include some time in the pharmacy. 

Drug Administration. Handing over a bottle of capsules to a study participant is one thing; inserting a butterfly catheter into an antecubital vein is something else again. If drug administration is very invasive, you’ll want to verify that the site has taken this into account when providing you enrollment projections. During subject visits, staff members may have to calculate doses, give intramuscular injections, perform infusions, or conduct sterilization procedures. You’ll want to verify that site staff has this expertise if required. Some clinical trials require a blinded dispenser who cannot be involved in any other study procedure or activities. If so, does the site have the resources for this?

Site Selection: it’s not just the PI, it’s the IP too
The study success and patient safety are jeopardized when a site can’t meet its enrollment target or doesn’t have the resources to execute the protocol. IP requirements can affect a site’s ability to do both. It’s critical that your site selection process – both your feasibility questionnaire and your pre-study visit – evaluate how well the site can meet the storage, preparation, and administration requirements of the study drug.

__________________________________________________________________________
A version of this article originally appeared in InSite, the Journal of the Society for Clinical Research Sites.

Photo Credit: By Harmid (Own work) [Public domain], via Wikimedia Commons

Sites frequently want to know how they can stand out to Sponsors and CROs to win more studies.
Our advice: Implement internal QC procedures.

Sponsors and CROs we work with consider a tight quality control program to be evidence that a site can be counted on to produce reliable data. It shows that managing quality at your site is a continual process, and doesn’t wait for monitors to arrive. In a risk-based monitoring environment, this is an increasingly compelling attribute.

Where to Start: The Usual Suspects
It makes sense for you to focus your QC efforts on those areas where you’ve historically had the most problems. If the phrase “trend analysis” makes you want to jump through a window -- it's okay -- you can climb back inside. You don't have to do a trend analysis. We've identified 3 areas in which audit findings are common and how you can avoid them.



Adverse Events (AEs) and Concomitant Medications (ConMeds). Often two sides of the same coin, AE and ConMed documentation needs to tell a consistent story. If source documents indicate a study participant had a sinus infection, it must be documented on an AE page, and any associated medications documented on the ConMeds page. A medication noted on the AE page must have a corresponding notation on the ConMed page. And all start and end dates must match across the source, AE, and ConMeds pages.

Drug Accountability Records. Calculating compliance percentages and counting pills are positively uninteresting tasks, easy to mess up, and involve math (which for some people triggers terrifying flashbacks of word problems about trains leaving stations). Is it any wonder that drug accountability records are frequent sources of error? Do some spot-checking: verify that the number of returned tablets matches the tallies recorded for them and recheck compliance calculations.

Essential Documents. Maintaining a complete, organized, uniform set of essential documents is an important, yet decidedly unsexy task. That’s why it’s a good indicator of your commitment to quality; a site that is disciplined enough to keep tight control over its essential documents is likely to carry that control into all aspects of trial execution. Make sure to file all documents associated with protocol amendments, such as IRB approvals and revised informed consent forms -- our auditors find these are the items most frequently missing from the essential document set. 

Write It All Down
Document your QC procedures in an SOP. It will serve as training material for site staff and a repository for worksheets and checklists.

There’s no magic organization for this QC SOP. A general set of instructions could outline how reviewers can verify that all documents follow ALCOA principles. For example, on (paper) source documents, are all pages and required signatures present? Are entries legible? Are corrections initialed, dated, and explained? Does the data make sense and lie within expected ranges? Have all data elements been populated? (Tip: turn the paper upside down to catch missing data.)

Checklists that are focused on particular types of documents should be as specific as possible. For example, QC reviews of source documents for screening visits would verify that the correct informed consent form was used, administration of consent was documented, medical release forms were sent if required, demographics were correct, all labs were received, reviewed and signed, all protocol assessments were completed, and all inclusion/exclusion criteria were met and documented.

A Virtuous Cycle
While designed to control quality, performing QC over time may actually improve quality. Results of QC reviews often suggest revisions you should make to your tools and operations to reduce error in the future.

Okay, you can climb back through the window again -- no one said CAPA. (But wouldn't that be impressive?)


Showcasing Site QC Processes
Does implementing a QC program require resources and time? Yes, and that’s the point. It’s evidence to Sponsors and CROs of your commitment to running a quality study. Not only that, but it demonstrates a proper respect for your study participants by ensuring their data can be used.

Oh, and make sure you highlight your QC program on feasibility questionnaires. It’s something to brag about.

________________________________________________________________________
A version of this article originally appeared in InSite, the Journal of the Society for Clinical Research Sites

Trying to comply with the GDPR got you down?
Maybe our parody will cheer you up.

(Sung to the tune of Lennon-McCartney's "Back in the U.S.S.R.")

Vertex Pharmaceuticals announced a plan late Tuesday to begin trials before the end of the year of the third in its so-called “triple combo” of pills designed to treat as much as 90 percent of the 75,000 patients worldwide who suffer from cystic fibrosis. That news, announced in conjunction with the Boston-based drugmaker’s third-quarter financial results last night, spurred a 6 percent stock increase after hours, implying the company’s market cap could increase by about half a billion dollars…

• Independent Data Monitoring Committee recommended continuing ongoing Phase 3 trial only in the more advanced “critical” group with Kevzara higher-dose versus placebo and discontinuing less advanced “severe” group

- Libtayo decreased the risk of death by 32.4% compared to chemotherapy

Pfizer partners with Valneva to progress Phase II-stage vaccine candidate for Lyme disease.

With the COVID-19 impact making itself apparent on a daily basis as the numbers climb and organizations respond, the effects on the pharma and biotech sector also shifts. Yesterday FDA announced a new concentration of agency assets to be focused … Continue reading →

Posted by Roger Bate My op-ed with Dinesh Thakur in The Hill today highlights the recent recommendation by Europe's chief drug regulator to suspend 700 generic drugs whose approvals were based on flawed – or forged – clinical studies conducted by GVK Bio, an Indian contract research organization. We urge U.S. Federal regulators to follow Europe’s lead and move to rescind market approval for these drugs while conducting their own investigation. You can read the op-ed here [...]

by J. David McSwane

The Transportation Security Administration ignored guidance from the Department of Homeland Security and internal pushback from two agency officials when it stockpiled more than 1.3 million N95 respirator masks instead of donating them to hospitals, internal records and interviews show.

Internal concerns were raised in early April, when COVID-19 cases were growing by the thousands and hospitals in some parts of the country were overrun and desperate for supplies. The agency held on to the cache of life-saving masks even as the number of people coming through U.S. airports dropped by 95% and the TSA instructed many employees to stay home to avoid being infected. Meanwhile, other federal agencies, including the Department of Veterans Affairs’ vast network of hospitals, scrounged for the personal protective equipment that doctors and nurses are dying without.

“We don’t need them. People who are in an infectious environment need them. Nobody is flying,” Charles Kielkopf, a TSA attorney based in Columbus, Ohio, told ProPublica. “You don’t take things for yourself. It’s the wrong thing to do.”

Kielkopf shared a copy of an official whistleblower complaint he filed Monday. In it, he alleges the agency had engaged in gross mismanagement that represented a “substantial and specific danger to public health.”

TSA has not required its screeners to wear N95s, which require fitting and training to use properly, and internal memos show most are using surgical masks, which are more widely available but are less effective and lack the same filtering ability.

Kielkopf raised a red flag last month about the TSA’s plan to store N95 respirators it had been given by Customs and Border Protection, which found more than a million old but usable masks in an Indiana warehouse. Both agencies are overseen by DHS. That shipment added to 116,000 N95s the TSA had left over from the swine flu pandemic of 2009, a TSA memo shows. While both stockpiles were older than the manufacturer’s recommended shelf life, the Centers for Disease Control and Prevention said that expired masks remain effective against spreading the virus.

Kielkopf and another TSA official in Minnesota suggested that the agency send its N95 masks to hospitals in early April, records show. Instead, TSA quietly stored many of them in its warehouse near the Dallas-Fort Worth airport and dispersed the rest to empty airports across the nation.

“We need to reserve medical masks for health care workers,” Kielkopf said, “not TSA workers who are behind an X-ray machine.”

The Number of Travelers Passing TSA Checkpoints Has Dropped to Historic Lows

The TSA didn’t provide answers to several detailed questions sent by ProPublica, but spokesman Mark Howell said in an email that the agency’s “highest priority is to ensure the health, safety and security of our workforce and the American people.”

“With the support of CBP and DHS, in April, TSA was able to ensure a sufficient supply of N95 masks would be available for any officer who chose to wear one and completed the requisite training,” the statement read.

“We are continuing to acquire additional personal protective equipment for our employees to ensure both their and the traveling public’s health and safety based on our current staffing needs, and as supplies become available,” TSA said.

A review of federal contracting data shows the agency has mostly made modest purchases such as a $231,000 purchase for gallons of disinfectant, but has not reported any new purchases of N95s.

An internal TSA memo last month said the surplus of N95s was expected to last the agency about 30 days, but the same memo noted that estimate did not account for the drastic decline in security officers working at airports. ProPublica asked how long the masks were actually going to last, accounting for the decreased staffing levels.

“While we cannot provide details on staffing, passenger throughput and corresponding operations have certainly decreased,” the TSA statement said.

The trade journal Government Executive reported this week that internal TSA records showed most employee schedules have been “sharply abbreviated,” while an additional 8,000 security screeners are on paid leave over concerns that they could be exposed to the virus.

More than 500 TSA employees have tested positive for COVID-19, the agency reported, and five have died.

The CDC has not recommended the use of N95s by TSA staff, records show, but that doesn’t mean workers who have or want to wear them can’t.

In one April 7 email, DHS Deputy Under Secretary for Management Randolph D. Alles sent guidance to TSA officials, urging them to wear homemade cloth face coverings and maintain social distancing. But the N95s, which block 95% of particles that can transmit the virus, were in notoriously short supply and should be “reserved” for health care workers.

“The CDC has given us very good information about how to make masks that are suitable, so that we can continue to reserve medical masks and PPE for healthcare workers battling the COVID-19 pandemic,” Alles wrote.

But two days later, on April 9, Cliff Van Leuven, TSA’s federal security director in Minnesota, followed up and asked why he had been sent thousands of masks despite that guidance.

“I just received 9,000 N-95 masks that I have very little to no need for,” he said in the email, which was first reported by Government Executive. “We’ve made N95s available to our staff and, of the officers who wear masks, they overwhelmingly prefer the surgical masks we just received after a couple months on back order.”

Minnesota Gov. Tim Walz had publicly asked that anyone who had PPE donate their surplus to the state’s Department of Health, Van Leuven said in the email to senior TSA staff.

“I’d like to donate the bulk of our current stock of N-95s in support of that need and keep a small supply on hand,” he wrote, adding the Minneapolis-St. Paul International Airport had screened fewer than 1,500 people the previous day, about a third of which were airport staff.

Van Leuven declined to comment, referring questions to a TSA spokesperson.

Later that day, Kielkopf forwarded the concerns to TSA attorneys in other field offices, trying to get some attention to the stockpile he felt would be better used at hospitals.

“I am sharing with you some issues we are having with n95 masks in Minnesota,” he wrote. “And the tension between our increasing supply of n95 masks at our TSA airport locations and the dire need for them in the medical community.”

Weeks went by, and finally, on May 1, Kielkopf wrote: “I have been very disappointed in our position to keep tens of thousands of n95 masks while healthcare workers who have a medical requirement for the masks — because of their contact with infected people — still go without.”

DHS did not respond to ProPublica’s questions about why it transferred N95 masks to TSA despite a top official saying they should be reserved for healthcare workers.

“So now the TSA position is that we desperately need these masks for the protection of our people,” Kielkopf said. “At the same time, most of our people aren’t even working. It’s a complete 180 that doesn’t make any sense.”

Do you have access to information about federal contracts that should be public? Email david.mcswane@propublica.org. Here’s how to send tips and documents to ProPublica securely.

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Pharmacies dispensed approximately 35% more prescriptions in March 2020, compared with the previous month, according to a survey by the National Pharmacy Association.

To read the whole article click on the headline

Supplies of the selective serotonin reuptake inhibitor, sertraline, are returning to stock after manufacturers reported “industry-wide” supply challenges, exacerbated by export bans and border closures implemented as a result of COVID-19. 

To read the whole article click on the headline

Pharmacy staff who are thought to have died as a result of the COVID-19 pandemic are to be among the healthcare workers remembered with a minute’s silence on 28 April 2020.

To read the whole article click on the headline

Community pharmacies need millions of pounds extra “to keep their heads above water” during the COVID-19 pandemic, pharmacy bodies have warned.

To read the whole article click on the headline

All the most important developments in the COVID-19 pandemic for pharmacists and their teams, as they happen.

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Wholesalers have “almost completely run out” of the personal protective equipment supplied by Public Health England for distribution to community pharmacies during the COVID-19 pandemic, the wholesaler trade body has warned.

To read the whole article click on the headline

NHS England has advised pharmacies to risk assess staff who may be particularly vulnerable to COVID-19, including those from a black, Asian or minority ethnic background.

To read the whole article click on the headline

The European Medicines Agency has begun a rolling review of data on Gilead’s remdesivir, positioning it to cut the time it takes to decide whether to approve the drug in COVID-19 patients.

Pfizer and partner BioNTech are right in the middle of one of the most important vaccine trials in the world right now, but that doesn’t mean the Big Pharma is taking its eyes off the inoculation ball elsewhere.

The successful activation of T cells is critical to the immune system's ability to clear infections. A retrospective study in China found that COVID-19 patients had remarkably low T-cell counts in their blood, while some pro-inflammatory cytokines such as IL-6—which Roche’s Actemra targets—were elevated.

Inovio Pharmaceuticals’ stock has climbed higher and higher over the past month since it said it was working on a speedy COVID-19 vaccine.

The FDA has delayed its decision on whether to approve Bristol Myers Squibb’s CAR-T cell therapy by three months. Bristol Myers attributed the delay to its submission of additional information upon the request of the FDA.

Sanofi and Regeneron’s Dupixent has become a popular treatment for atopic dermatitis and asthma. Now, a research team in Japan has discovered that IL-4 and its receptor—which Dupixent inhibits—could be promising targets for treating the eye disease age-related macular degeneration.

PTC Therapeutics is adding to its rare disease pipeline with a midstage biotech buyout with a low upfront payment tied in with stock and biobucks.

Moderna is finalizing the protocol for a phase 3 trial of its COVID-19 vaccine with a view to starting the study early in the summer. The establishment of the timeline, which follows FDA clearance to run a phase 2 trial, puts Moderna on track to win approval for its mRNA vaccine mRNA-1273 next year.

A recent study looking at the effects of caffeine on brief, high intensity exercise found the substance improved performance, regardless of genetic variations in how subjects metabolized caffeine.

The video has been viewed millions of times on YouTube via links that are replaced as quickly as the video-sharing service can remove them for violating its policy against "COVID-19 misinformation."

A major health agency fears a humanitarian crisis. Migrant workers are returning home from the hard-hit Dominican Republic. Medical equipment is in short supply. And social distancing is improbable.

President Trump wants businesses to start reopening after the coronavirus forced shutdowns. Here's what the White House task force recommends for states.

With social distancing, many people are speaking less and their voices sound raggedy. NPR's Scott Simon talks with speech pathologist Sandy Hirsch, about keeping the voice sounding as it should.