Category: Uncategorized

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.

Every day we scan hundreds of articles and press releases to find items, events, trends, developments and other flotsam and jetsam related to specialty pharmacy, specialty infusion and the emerging pharmaceutical marketplace in general. Today we ran across an article published in the Irish ECHO that has some interest for us here in this dear land across the Irish Sea.

A company called HealthSolutions (yep, no space) is making some inroads with US-based specialty pharmacies, including Accredo, with a spiffy new device for patients on injectable therapy. Think of it as AI meets sharps container. It is called the SmartSharpsBin….. catchy name huh?. Now a sharps container by itself is not exactly hot news but one that communicates with its owner to remind them of their next scheduled injection and will alert them if the box isn’t fed the last now used scheduled syringe is noteworthy. And, yes, there is also an app for that on your phone! When the container is about to be full, a new container is automatically sent to the patient who then uses that same mailing container to return the used sharps container….. neat, HUH?

Supposedly, health insurers are reimbursing for the device. And, anyone can obtain one independently via membership (multiple plans are available starting at 12 months for a flat $420 or $35 monthly.)

Whether the concept takes hold remains to be seen…. especially at that price point. None the less, this gizmo is one of the newest tech offerings to promote patient compliance that we’ve seen in some time and, who knows, it may be the start of a new thang.

CLICK HERE to visit the SmartSharpsBin web site.

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HealthBeacon Share Price Jumps by 111% in the Past Month

— HealthBeacon has seen its share price jump by 111% in the past month.

— The Inchicore company, which listed on Euronext Dublin in 2021, develops smart tools to assist patients to manage their medications from their homes.

HealthBeacon’s IPO raised €25m, with the company reporting a market capitalisation of €98.4m on the day of its public listing. This figure now stands at around €32m on Euronext Dublin, while shares have slid around 58 per cent in the past year.

Jim Joyce, the company’s founder and chief executive attributes this to a surge in activity in the US, with a few key areas giving “all that optimism that we’ve got the right product fit”. “We’ve done a number of deals with specialty pharmacy organisations in the US,” he said to the Independent. “Organisations like Accredo, where they integrated the technology into their fundamental offering. “That means if you’re on a high-value injectable medication and you’re working with certain large pharmacy systems, you’re going to be offered the HealthBeacon device as part of going on that medication.

More US partnerships are coming down the line with speciality pharmacy organisations, pharma companies, as well as clinical practices, Mr Joyce says.  HealthBeacon’s offerings are reimbursed by insurers in the US.

HealthBeacon has over 20 employees in the US market, with offices in Boston and Orlando. Overall, the company’s headcount stood at around 67 at the end of last year.

The company’s client base grew to 30 last year from 23 in 2021. Sales rose slightly to €2.25m from €2.2m the prior year, while its losses pretax grew to €13m. This was attributed to a global chip shortage and energy crisis, which also weighed on gross margins. However, Joyce is upbeat about what the future holds as the business predicts annualised revenue of €25m by the end of March next year.

Are you familiar with the Preserving Patient Access to Home Infusion Act (PPAHIA)?

You probably should be.  It has been kicking around the halls of Congress since 2021…. without passage.  However, like Whack-a-Mole, it keeps popping up in each new Congress because it has popular appeal and even bipartisan support, at least in the Senate.

If it ever gets passed, the bill will clarify that drugs shall be reimbursement and at what rates.  No small achievement for the home health industry. There’s a big  sticking point however ….. hospitals. The more home infusions the fewer hospital outpatient infusions and related hospital revenues….. and the hospital lobby is on guard. As we know, hospital outpatient infusions generally cost multiples of the same service delivered in the home. 

Besides being able to save millions for patients and Medicare/Medicaid, the bill seeks to correct some changes that were included in the 2018 Cures and Bipartisan Budget Acts to create a professional services benefit for Medicare Part B home infusion drugs. That tinkering “resulted in CMS improperly implementing the benefit by requiring a nurse to be physically present in the patient’s home in order for providers to be reimbursed. As a result, provider participation in Medicare’s home infusion benefit has dropped sharply and beneficiaries have experienced reduced access to home infusion over the last several years.”

Since little legislation seems to be leaking out of Washington these days this bill may have to wait another year…. or more…. before it ever gets passage as common sense might dictate. 

Oh well…

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Lawmakers Reintroduce Bipartisan, Bicameral Legislation to Increase Access to Medicare Home Infusion Benefit

Jun 14 2023 — WASHINGTON – U.S. Sens. Mark R. Warner (D-VA) and Tim Scott (R-SC) joined by U.S. Reps. Vern Buchanan (R-FL-16), Chairman of the House Ways & Means Health Subcommittee, Debbie Dingell (D-MI-06), Diana Harshbarger (R-TN-01), and Terri Sewell (D-AL-07) today reintroduced legislation that will ensure patients maintain access to home infusion therapy. The Preserving Patient Access to Home Infusion Act protects access to Medicare’s home infusion benefit by making clear that pharmacy services for home infusion therapy can be reimbursed and sets an appropriate rate for such services.

“We have seen for years that patients are better off when they can receive quality care from the comfort of their own homes,” said Sen. Warner.   “This legislation would ensure that……..

…………..press release continues

CLICK HERE to read the full press release

FDA has now approved four gene therapies within the past ninety days.

That’s got to be a record!

The FDA recently approved a new infused gene therapy, Elevidys (delandistrogene moxeparvovec-rokl) from Sarepta Therapeutics.   Elevidys is administered as a single-dose intravenous infusion and is the first and only gene therapy approved for Duchenne. It is an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.  Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. This accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.

Elevidys  is the sixth approved therapy in Duchenne and the first therapy to replace the missing dystrophin protein by using a modified smaller version of the dystrophin gene, called micro-dystrophin, to produce a modified micro-dystrophin protein .Duchenne is a fatal genetic disorder that slowly robs patients of their muscle strength.

The therapy was approved without a black box warning but alerts were cited for Acute Serious Liver Injury, Immune-mediated Myositis, and Myocarditis.

Sarepta announced that Elevidys will launch at a one-time treatment cost of $3.2 million.

Details related to distribution and logistics were not released at time of approval, but industry sources have confirmed that at least one specialty pharmacy will be selected as the company’s specialty distribution partner. 

CLICK HERE for full prescribing information

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Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 22, 2023– Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) accelerated approval of Elevidys (delandistrogene moxeparvovec-rokl), …………

CLICK HERE to access the full Sarepta press release