Catching up on recent specialty approvals————

The FDA recently approved a new ORAL, kinase inhibitor therapy, Vanflyta (quizartinib) from Daiichi Sankyo Company, Ltd., in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated.

Vanflyta was approved with a Black Box warning and REMS. 

CLICK HERE to access prescribing information

AML is one of the most common forms of leukemia in adults and an estimated 20,380 new cases diagnosed in the U.S. in 2023.  Up to 37% of newly diagnosed patients with AML have a FLT3 gene mutation and approximately 80% of these are FLT3-ITD mutations, which drive cancer growth and contribute to increased risk of relapse and shorter overall survival.  The five-year survival rate for patients with FLT3-ITD AML has been reported at approximately 20%.

VANFLYTA is the first and only FLT3 inhibitor to be approved specifically for FLT3-ITD positive AML and across the three phases of treatment – induction, consolidation and maintenance in patients without transplant – for newly diagnosed AML.

Daiichi Sankyo will launch Vanflyta at $546 per tablet. Total cost per patient will vary based on number of cycles (induction, consolidation, maintenance). By comparison, Xospata, another FLT3 therapy, comes with the highest price tag at $750 a day.

Distribution logistics were not announced at the time of approval. However, it is expected that Vanflyta will launch through specialty pharmacy limited distribution.

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Vanflyta First FLT3 Inhibitor Approved in the U.S. Specifically for Patients with Newly Diagnosed FLT3-ITD Positive AML

First and only FLT3 inhibitor approved across three phases of AML treatment, when added to chemotherapy, improved overall survival

Jul 20, 2023 TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)– Daiichi Sankyo (TSE: 4568) today announced that Vanflyta (quizartinib) has been approved by the U.S. Food and Drug Administration (FDA) in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated.

Earlier this week we sent a Report on biosimilar utilization and trends. Today we follow up with some stats from another credible source that add a few new insights.

AmerisourceBergen Corp (ABC) recently published results from a survey of payers related to treatment of biosimilars within their coverage policies. The findings are worth considering for future planning.

Top line highlights—

Organization types: 51 payors / advisors representing 300 million covered lives

(about 90% of the U.S. population)

• 55% health plans
• 24% integrated delivery networks, and
• 22% pharmacy benefit managers

• 45% said their organization would allow multiple biosimilars
• 35% said they would select a single preferred biosimilar and
• 4% said they would prefer the reference product.
• 16% selected ‘Other’ (hmmmmm??)

• 96% and 82% of respondents, respectively, said that lower price and contracting arrangements (legal speak for rebates) continue to drive preference for a reference product.
• 88% of respondents said that interchangeability will inevitably force the shift to greater biosimilar adoption.

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What’s Driving Biosimilar Decision Making? AmerisourceBergen Payor Survey Has Some Answers

Biosimilar competition is heating up, with eight biosimilars to adalimumab (Humira, AbbVie) already launched in 2023 and more on the horizon. Other reference products also have anywhere from two to five biosimilar competitors. With this intense level of competition, payors are more likely to select multiple biosimilars for the same product, rather than a single biosimilar or the reference product only, according to a survey from AmerisourceBergen…..

CLICK HERE to read the full article

by Gina Shaw, Pharmacy Continuum

A recently released study on payor practices related to biosimilars was detailed in an article published by the Center for Biosimilars….. and some of the findings are thought provoking.

Here are the highlights from the study:

• The study identified 1181 coverage decisions for 19 commercially available biosimilars, representing 7 reference products and 28 indications
• Health plans dictated coverage exclusions or step therapy limitations on biosimilars in 229 (19.4%) of the 1181 coverage decisions
• Payers were more likely to restrict biosimilar coverage for pediatric populations whereas the reverse was observed for all other categories, including Oncology, for specialty biosimilars.
• Biosimilar utilization was higher for diseases with US prevalence higher than 1,000,000 cases
• Biosimilar utilization was higher if the health plan did not contract with 1 of the 3 major pharmacy benefit managers
• Payers were less likely to enforce restrictions on the biosimilar if:

— the biosimilar was indicated for cancer treatments 

— if the product was the first biosimilar to market

— there were two available biosimilar competitors, and

— could generate annual list price savings of more than $15,000 per patient. 

• However, payers were most restrictive if the biosimilar did not have a cost-effectiveness measure available.

While many of the findings are confirmatory, the report doesn’t address the underlying dynamics of how rebates affect payer policies over preferred utilization of reference products.

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Payers Reveal Most Significant Factors Impacting Biosimilar Coverage Decisions

Jun 29, 2023

Justina Petrullo    AJMC Center for Biosimilars

It was found that cancer treatment, pediatric population, and coverage restriction of reference products were revealed as some of the strongest factors associated with biosimilar coverage decisions by US commercial health plans relative to reference products.

Cancer treatment, pediatric population, and coverage restriction of reference products were discovered as some of the most significant factors associated with biosimilar coverage decisions by commercial health plans in the US relative to their reference products, according to BioDrugs.

CLICK HERE to access the full article

We’ve written recently on legislation grinding its way through Congress targeting the PBM industry and its various business practices that industry stakeholders say are unfair and must be remedied. The article below adds yet another practice to the list we’ve already shared…..Step Therapy.

Step therapy has been around for many decades in one form or another. In its earliest iteration it was simply – ‘Let’s try this first to see if it works…. and then we can try these ten other drugs to see what happens!’ Things have advanced significantly since then. What we have now is ‘Let’s try this less costly, legend therapy before we try the newest, uber-expensive therapy.’ 

While there is an argument for efficiency in managing overall healthcare spend, step therapy can be problematic for many patients who expend time that they may not have experimenting with a therapy that may only manage symptoms vs. a new therapy option that may be disease modifying. 

It appears that language restricting step therapy has been included in the Pharmacy Benefit Reform Act slowly making tis way through the US Senate. The bill has been bouncing around the Senate since 2017 even with bipartisan support. Given the momentum that other bills are gaining in Congress it is hoped that the step therapy bill will be incorporated in a final version.

It is noteworthy that thirty states have already enacted step therapy legislation similar to the provisions in the Safe Step Act. In those instances, the state laws apply only to health plans subject to those states’ laws but do not impact ERISA qualified health plans.

CLICK HERE to read the full article to learn about provisions in the bill that specify timing requirements for step therapy appeals as well as five exceptions where a patient may qualify for an exemption from step therapy.

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Step Therapy

Jun 19, 2023 — Legislation designed to smooth the path to exemptions from step therapy has been incorporated into the Pharmacy Benefit Reform Act, one of several bills targeting pharmacy benefit managers that is making legislative headway in the Senate. Legislation that aims to make it easier for patients to get exemptions from the step therapy requirements has been folded into one of the several U.S. Senate bills targeting pharmacy benefit managers (PBMs), and patient and provider organizations are optimistic about PBM legislation — in one form or another — becoming law.

“PBM reform is going to pass in this Congress — (although) I wouldn’t say by the end of this year — and it will include step therapy reform,” Erin McKeon, associate director, federal advocacy, for the Crohn’s & Colitis Foundation.

The current Congress, the 118th, is scheduled to adjourn at the…………

CLICK ABOVE to access the full article

If you’ve been waiting anxiously (must ask why?) for yet another human growth hormone to be approved….. then wait no longer.

The FDA recently approved a new therapy, Ngenla (somatrogon-ghla) from Pfizer, a once-weekly, human growth hormone (hGH) analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone.

Human growth hormone works by replacing insufficient growth hormone in the body. Ngenla is administered subcutaneously and must be dosed based on patient weight. Ngenla is available in two single-patient-use, disposable, prefilled pens: a 24 mg/1.2 mL (20 mg/mL) with 0.2 mg increments and also a 60 mg/1.2 mL (50 mg/mL) pen with 0.5 mg increments.

Various clinical sources cite prevalence levels of ~1/ 4000-10000 children aged 3+-18 years old with inadequate secretion of endogenous growth hormone. There are an estimated 65 million children between 3 and 18 years old in the US. That translates into a potential target patient population of only about 5,000-10,000 patients who may require therapy.

Pfizer confirmed that Ngenla will launch at a monthly list price of about $8,300 for a child weighing 35 kg. It is expected to become available starting in August. Distribution details were not disclosed.

Here is a recap of the now 14 various hGH products approved by the FDA:

• Geref Diagnostic sermorelin 1990
• Norditropin (Pro) somatropin 1995
• Norditropin FlexPro somatropin 1995
• Serostim (Pro) somatropin 1996
• Saizen (Pro) somatropin 1996
• Geref  sermorelin 1997
• Nutropin AQ (Pro)  somatropin 1999
• Genotropin (Pro) somatropin 2001
• Zorbtive (Pro)  somatropin 2003
• Accretropin somatropin 2008
• Zomacton  somatropin 2015
• Macrilen (Pro)  macimorelin 2017
• Skytrofa (Pro)  lonapegsomatropin 2021
• Sogroya (Pro) somapacitan-beco 2023
• Ngenla somatrogon-ghla 2023

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FDA Approves Pfizer’s Ngenla, a Long-Acting Once-Weekly Treatment for Pediatric Growth Hormone Deficiency

June 28, 2023 — NEW YORK & MIAMI–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. 

CLICK HERE to access the full press release

The outcome of the FTC review of PBM practices and its report on needed corrections may be some of the biggest news items this year. As the article below details, the FC has basically dumped as totally outdated nine advocacy letters dated between 2004 and 2014, These letters advocated against proposals to increase regulatory oversight and transparency of PBMs….. no bias there!

So, where is the FTC shining its regulatory spotlights?

The inquiry is aimed at shedding light on numerous practices that have come under industry stakeholder attacks, including—-

·        fees and clawbacks charged to network pharmacies

·        steering patients towards PBM-owned pharmacies

·        potentially unfair audits of network pharmacies

·        complicated and opaque methods to determine pharmacy reimbursement

·        the prevalence and equity of prior authorizations and other administrative restrictions

·        the use of specialty drug lists and surrounding specialty drug policies, and 

·        how rebates and fees paid by drug manufacturers affect the NET cost of drugs to payers

·        no pass-through of rebate savings resulting and higher out-of-pocket costs to patients.

That’s quite a list! Relief on any one of which could have significant consumer impact.

As part of this inquiry, the FTC has sent compulsory orders to CVS Caremark, Express Scripts, OptumRx, Humana, Prime Therapeutics, and MedImpact.

The next phase of the FTC investigation might really be a doozie!

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FTC Withdraws Advocacy for Pharmacy Benefit Managers

The Commission rescinded earlier guidance that was opposed to increased regulatory oversight and transparency of PBMs.

JUL 25 — Claiming that its old policy statements no longer reflect current market dynamics, the Federal Trade Commission has voted to rescind its prior statements of advocacy for pharmacy benefit managers, effectively ending the agency’s previously stated endorsement of PBMs.

According to the FTC, the new statement pulling its advocacy is a response to PBMs’ continued reliance on older FTC advocacy materials that opposed mandatory PBM transparency and disclosure requirements.

It also warns against reliance on the Commission’s prior conclusions, particularly given the FTC’s ongoing study of the PBM industry to update its understanding of the industry and its practices.

CLICK HERE to access the full article

The FDA recently approved a new therapy, Rystiggo (rozanolixizumab-Noli) from UCB, for the Treatment of adults with Generalized Myasthenia Gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Myasthenia gravis (MG) comes in two main types, generalized and ocular. Generalized MG is the most common type, accounting for 85 percent of people with MG. Generalized MG is generally more serious than ocular MG since it causes weakness in many muscle groups. Common gMG symptoms include difficulty making facial expressions. problems chewing and difficulty swallowing. slurred speech. weak arms, legs, or neck.

Administration of Rystiggo is by subcutaneous infusion. However, prescribing information specifies that it should only be prepared and infused by a healthcare provider.  The most common adverse reactions were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea.

Standard administration schedule is once weekly for 6 weeks, At $6050 / vial,  baseline therapy is a minimum of $36,000. 

Since Rystiggo is administered in clinics it will likely be a specialty distribution product.

CLICK HERE to access prescribing information

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UCB Announces U.S. FDA Approval of RYSTIGGO (rozanolixizumab-Noli) for the Treatment of Adults With Generalized Myasthenia Gravis

FDA approval of RYSTIGGO (rozanolixizumab-noli) has been granted under the Priority Review designation for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive1

U.S. FDA approval is based on the pivotal Phase 3 MycarinG study in gMG2, a large phase 3 study which demonstrated treatment with rozanolixizumab-noli resulted in statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.

Additional treatment option provides opportunity for U.S. clinicians to tailor therapeutic approach based on individual patient needs

The year was 2019…… and that BUZZ you were hearing was all about drone deliveries of everything from pizza to prescriptions. But by 2020…. mostly crickets!

After that warm start, the zeal for drone deliveries cooled. A number of companies, including UPS, FedEx, CVS, Walmart, Amazon and more were saying that drone delivery would the next best thing…. some day. 

So, what’s happened since?

In short, very little has been said publicly, but it seems that experimentation continues at a quiet pace while ‘proof -of-concept’ is still being nailed down.

What has been learned is that drones can reliably cover a round trip of about 150 miles flying at speeds of 60+ MPH. A delivery van would have difficulty consistently matching that speed….. but they might have 50 or more deliveries to make…. not just one. Standard drones have a weight limitation of about 5 pounds, which is OK for most prescriptions. Commercial drones run more than $10,000 each with high-capacity drones running multiples of that amount. Then there is the expense of maintenance and who the heck is going to remotely ‘pilot’ the flights? Cost per delivery by van runs less than $10 today according to Amazon and other national companies. The cost per drone delivery will need to come down significantly to compete.

But, Michigan Medicine is bravely stepping outside the hanger as an early adopters of the drone concept. As noted in the article below, they have contracted with Zipline, a third-party drone delivery service to start deliveries in a limited area starting in 2024. Zipline seems to have a solid track record in the drone segment with “hundreds of thousands of deliveries”.

The question is….. Will Michigan Medicine be a trend setter?

NEWS FLASH: A press release today announced that Ohio Health Corp has just signed a deal with drone operator Zipline. Ohio Health is central Ohio’s largest health system is working with Zipline toward a launch in 2024. The first phase would be deliveries between OhioHealth facilities, with deliveries to as many as 2 million patients rolling out in 2025! 

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Michigan Medicine will use drones to deliver prescriptions in 2024

Michigan Medicine announced March 15 they will be partnering with Zipline, an autonomous drone delivery service, to distribute prescription medications to some Washtenaw County patients’ homes in 2024.

The partnership aims to help deliver pharmaceuticals across the county and is projected to double Michigan Medicine’s prescription fulfillment. The partnership is a part of Michigan Medicine’s broader strategy to expand specialty pharmacy services, which provide medications to patients with complex diseases.

……… CLICK HERE to read the full article

If you haven’t added the term ‘blockchain’ to your vocabulary, then it is high time that you do…. starting right now.

Blockchain is a system of recording information in a way that makes it difficult or impossible to change, hack, or cheat the system. Sound interesting? 

How about using this technology for something practical….. like tracking real-time pricing of drugs in the marketplace based on the input of many verified and trusted customers. Got your attention?

Well, the first hospital in the US has just announced it has signed on to the concept and expects the result will be a tsunami of customers ready to also jump on this newfangled bandwagon.

So, what exactly is blockchain?

A blockchain is essentially a digital ledger of transactions that is duplicated and distributed to each participant in the network of computer systems on the blockchain. Each block in the chain contains many transactions, and every time a new transaction occurs anywhere on the blockchain, a record of each transaction is added to every participant’s ledger. The decentralized database managed by multiple participants is known as Distributed Ledger Technology (DLT) see graphic above.

Do hospitals use blockchain?

As noted in the press release below, hospitals are starting to embrace blockchain. Blockchain will be used for health record-keeping, clinical trial findings, patient monitoring, tracking safety protocols, display information & transparency, and much more. It will support hospital financial management and minimize the time and cost of data transformation. Industry pundits also predict that Pharma and any organizations involved in high-volume healthcare transactions will also seek to join.

What are the risks of blockchain technology in healthcare?

The biggest concerns about implementing blockchain in healthcare are technological limitations, speed, and scalability. Storing large records on the blockchain, such as full electronic medical records or genetic data records, would be inefficient and costly. It is also currently difficult to query data within a blockchain, limiting clinical & statistical research. Perhaps the most vexing concern is its lack of confidentiality (e.g., patient privacy). As such, many questions will need to be answered before blockchain reaches full deployment in healthcare….. but now you know enough to sound more smarter.

Baptist 1st system to use blockchain for drug pricing

Baptist Health, a nine-hospital system in Louisville, Ky., is the first health system in the U.S. to use

MediLedger’s blockchain technology to review prices for procured drugs.

The system teamed up with MediLedger, a blockchain pharmaceutical technology company, to ensure the 40,000 pharmaceutical products it buys every year are “the correct negotiated contracted prices,” Thomas Matanich, system director of pharmacy contract management for Baptist Health, said in a July 18 news release.

The blockchain technology will help the system find and address price discrepancies before purchasing a

pharmaceutical product, the release said. “It’s very interesting nobody has taken on that bull by the horn,” Nilesh Desai, chief pharmacy officer at Baptist Health, told Becker’s.

Page Twenter – Beckers Hospital Review

The FDA recently approved a new therapy, Litfulo (ritlecitinib) from Pfizer, Inc., a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata.

Alopecia areata is an autoimmune disease characterized by patchy or complete hair loss on the scalp, face, or body.  It has an underlying immuno-inflammatory pathogenesis and develops when the immune system attacks the body’s hair follicles, causing hair to fall out.  This hair loss often occurs on the scalp, but it can also affect eyebrows, eyelashes, facial hair, and other areas of the body.  Alopecia totalis (total scalp hair loss) and alopecia universalis (total body hair loss) are types of alopecia areata.

Alopecia Areata impacts nearly 7 million people in the U.S. and approximately 147 million people globally and can affect people of any age, gender, race, or ethnicity and can cause considerable burden beyond hair loss.  Nearly 20% of people with alopecia areata are diagnosed before the age of 18.

The therapy was approved with a Boxed Warning that includes Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events (Mace), and Thrombosis.

Pfizer announced that a full year supply of Litfulo carries a list price of $49,000, similar to other specialty dermatologic treatments.

Distribution and logistics for the therapy were not announced but it is highly likely it will launch through specialty pharmacy limited distribution.

CLICK HERE to access prescribing information

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FDA Approves Pfizer’s LITFULO for Adults and Adolescents with Severe Alopecia Areata

LITFULO is the first and only treatment for severe alopecia areata approved for patients 

June 23, 2023 — NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved LITFULO (ritlecitinib), a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. The approved recommended dose for LITFULO is 50 mg. It is the first and only treatment approved by the FDA for adolescents (12+) with severe alopecia areata.

“While patients may start to develop symptoms of alopecia areata at any age, most people start showing signs in their teens, twenties, or thirties,” said Dr. Brittany Craiglow, Associate Professor Adjunct – Dermatology at Yale School of Medicine. “LITFULO is a particularly important treatment option for younger patients with substantial hair loss, who often struggle with such a visible disease.”

LITFULO is a kinase inhibitor which inhibits Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases.

“LITFULO is an important treatment advancement for alopecia areata, an autoimmune disease that previously had no FDA-approved options for adolescents and limited options available for adults,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “With today’s approval, adolescents and adults who struggle with substantial hair loss have an opportunity to achieve significant scalp hair regrowth.”

The FDA approval was based on results of clinical trials in alopecia areata. The ALLEGRO Phase 2b/3 trial, which enrolled 718 patients with 50% or more scalp hair loss as measured by the Severity of Alopecia Tool (SALT), evaluated the efficacy and safety of LITFULO at 118 sites in 18 countries. In this pivotal study, 23% of patients treated with LITFULO 50 mg had 80% or more scalp hair coverage (SALT≤20) after six months compared to 1.6% with placebo. The efficacy and safety of LITFULO were consistent between adolescents (12 through 17 years of age) and adults (18 years of age and older). The most common adverse events (AEs) reported in at least 4% of patients with LITFULO include headache (10.8%), diarrhea (10%), acne (6.2%), rash (5.4%), and urticaria (4.6%). Full results from the ALLEGRO Phase 2b/3 study were published by The Lancet in April 2023.

“People living with alopecia areata are often misunderstood, and their experience is frequently trivialized as ‘just hair.’ However, it is a serious autoimmune disease that can have considerable negative impact beyond the physical symptoms,” said Nicole Friedland, President and Chief Executive Officer of the National Alopecia Areata Foundation (NAAF). “We believe the approval of LITFULO is a significant advancement for the treatment of alopecia areata, particularly for teens. It’s exciting to see more FDA-approved treatments becoming available for this community.”

View the full Prescribing Information. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.