And the beat goes on……..

We’ve covered the market moves of both CVS and Walgreens as they transition from a pharmacy centric business model to one of health care provider. The cash flow velocity for each related to multiple acquisitions and some divestitures are all seemingly aimed at carving out a huge niche in controlling…. umm we mean providing…. direct health care services to millions of patients nationally.

Let’s start with a quick recap of how Walgreens’ has recently spent upwards of $16 billion ….

• Acquisition of CareCentrix for $700+ million….. CareCentrix coordinates home care for health plans, patients, and medical providers for 19 million patients in the home setting and approx. 7,400 provider locations.
• Acquisition of specialty pharmacy developer / manager Shields Health Solutions for a $1+ billion.
• Acquisition of primary care network VillageMD for $5.2 billion (majority share). 
• Sold $1.7 billion in shares of Amerisource Bergen to partially fund the $9billion acquisition of Summit Health-CityMD which operates more than 680 provider locations in 26 markets.

Not to be outdone, CVS announced almost $19 billion in deals……

• Acquisition of home care company Signify Health for $8 billion and
• Acquisition of Medicare-focused primary care provider Oak Street Health for $10.6 billion.  

What can one make of all this repositioning?

The legacy pharmacy businesses for each company dovetails well with their healthcare subsidiaries serving as feeders for patient prescriptions….. especially in the specialty medication category.

Second, the market encroachment by hospital / health-system operated retail and specialty pharmacies is mitigated by creating some control over prescribing docs at their subsidiary companies.

And, for CVS, which is also a PBM, the overall environment for PBMs has turned somewhat toxic.

One can only imagine what may be next.

CLICK HERE for full article on Walgreens

CLICK HERE for full article on CVS

The excitement in the air is palpable….  

Yes, another biosimilar to Humira has been approved by the FDA!! Hallelujah!! 

Ummm…..  wait…..  the degree of excitement may be….. exaggerated. 

The FDA did approve yet another biosimilar to Humira but the news wires did not get very hot. Celltrion Healthcare announced that the FDA has approved Yuflyma (adalimumab-aaty) as the ninth biosimilar referencing Humira (adalimumab). If you are keeping count there are now nine biosimilars to Humira since 2016 with yet a tenth in the wings waiting for approval later in 2023.

It is noteworthy that Celltrion is now seeking to obtain an interchangeability designation for Yuflyma which would give it a valuable marketing edge.

Approval was granted with a black box warning for infections and malignancies.

CLICK HERE to access prescribing information.

Celltrion did not announce pricing for Yuflyma but it is expected to mirror pricing for other adalimumab products now coming onto the US market.

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FDA Approves Celltrion’s Yuflyma, the Ninth Adalimumab Biosimilar

May 24, 2023 — Celltrion Healthcare announced the FDA approval of its adalimumab biosimilar, Yuflyma (adalimumab-aaty), making it the ninth biosimilar referencing Humira (adalimumab) to receive regulatory approval in the United States (Table).

Yuflyma is a high-concentration (100mg/mL), citrate-free adalimumab biosimilar. The biosimilar will be offered to patients in a prefilled syringe and autoinjector administration options.

“Currently, more than 80% of patients treated with Humira in the United States rely on a high-concentration and citrate-free formulation of this medication. The availability of a high-concentration and citrate-free formulation adalimumab biosimilar provides an important treatment option for patients with inflammatory diseases who benefit from this effective therapy,” said Jonathan Kay, MD, a professor of medicine and the associate director of the MD/PhD Program at the University of Massachusetts School of Medicine, in a statement from Celltrion.

The FDA approved the biosimilar for 8 indications:

• rheumatoid arthritis
• juvenile idiopathic arthritis
• psoriatic arthritis
• ankylosing spondylitis
• Crohn disease
• ulcerative colitis
• plaque psoriasis
• hidradenitis suppurativa

Yuflyma is the fifth biosimilar and second anti-tumor necrosis factor biosimilar developed by Celltrion Healthcare to receive FDA approval. Yuflyma is also approved in the European Union and Canada.

“Yuflyma offers patients a high-concentration and citrate-free formulation of adalimumab biosimilar, providing an alternative treatment option for patients. It represents a key treatment option in patient care and patient choice…. As a leading global biopharmaceutical company, we are leveraging our unique heritage in biotechnology, supply chain excellence, and best-in-class sales capabilities to expand the availability of high-quality biosimilars for U.S. patients,” commented Tom Nusbickel, chief commercial officer at Celltrion USA, in a statement.

Celltrion said that Yuflyma will launch on the US market in July 2023. The company said that it is also seeking an interchangeability designation for the biosimilar, predicting that the extended approval will come in the fourth quarter of 2024.

The news comes after the US welcomed the first adalimumab biosimilar on the market, Amgen’s Amjevita, in January 2023. It will be the only adalimumab biosimilar competing against the originator until July 2023.

In March 2023, the FDA approved the high-concentration, citrate-free formulation of Sandoz’ Hyrimoz (adalimumab-adaz).

Across 2023, an estimated 10 adalimumab biosimilars will enter the US market, with 9 expected to launch in July 2023. In addition to the aforementioned adalimumab biosimilars, the other FDA-approved adalimumab products are Idacio, Abrilada, Yusimry, Hadlima, and Hulio.

The FDA is also reviewing Alvotech’s adalimumab biosimilar, AVT02, which recently received a second complete response letter (CRL) that cited “deficiencies” in the company’s Iceland-based manufacturing facility. The CRL pushed back the FDA’s decision date for approval from April 2023 to later June 2023.

by Skylar Jeremias

A few weeks ago, we sent a Report asking whether value-based (VB) contracting had a heartbeat. Today we have evidence that a meaningful heartbeat has been detected. 

As reported, 12 % of payers have put their toes into the VB pond. The article below offers new evidence that the heartbeat may be getting louder. Kaiser Permanente has cut a multi-billion-dollar deal with Geisinger Health predicated on the VB model.

The deal sets the foundation for a new entity, Risent Health, with plans to expand to as many as five health systems that will generate north of $30 billion in revenue. Those five or so health systems will also represent upwards of 10,000 prescribing physicians…. all of which can be channeled into select pharmacy fulfillment sources.

Their proposed value-based premise takes on a new dimension. The envisioned large scale enables ‘the entity’ to tackle the big obstacles we noted in our earlier Report holding back VB contracting. Key among these is the ability to analyze outcomes across a very large patient population, critical to obtaining a more accurate determination of meeting… vs. not meeting… contractual VB metrics.

None the less, it will take years to determine the ultimate success of the venture.

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Kaiser to Fold Geisinger into New Value-Based Care Company, Risant Health

The deal creates a new force in the world of value-based health care.

Apr 26, 2023 — Kaiser Permanente and Geisinger Health said they have agreed to create a new company called Risant Health, which plans on making more acquisitions in the years to come.

Under the deal, Geisinger would become a part of Risant and keep its name. The agreement is subject to federal and state regulators.

Like California-based Kaiser and Pennsylvania-based Geisinger, Risant would be a nonprofit; it would be headquartered near Washington, DC.

“In addition to Geisinger, Risant Health will grow its impact by acquiring and connecting a portfolio of like-minded, nonprofit, value-oriented community-based health systems anchored in their respective communities,” according to a statement put out by the companies.

According to The Wall Street Journal, Risant seeks to acquire 4 or 5 more hospital systems and get to total revenue of $30 billion to $35 billion over the next 5 years, and Kaiser is expected to provide about $5 billion in funding to Risant.

Risant Health will operate separately from Kaiser Permanente, while health systems that join the new firm will also retain distinct identities in their communities.

Jaewon Ryu, MD, JD, who became Geisinger’s CEO in 2019, will become the new CEO of Risant Health.

Geisinger includes 10 hospital campuses, a health plan with more than half a million members, and has more than 25,000 employees and more than 1700 employed physicians.

“Through Risant Health, we will make our value-based care expertise, technology and services available to community-based health systems, like Geisinger, to strengthen their ability to provide value-based care models with a focus on high-quality and equitable health outcomes,” said Greg. A. Adams, chair and CEO, Kaiser Permanente, in the statement.

by Allison Inserros, AJMC

From time to time we run across an article that offers more than just recent industry news. Today we stumbled across an article that we suggest be added to your company’s training syllabus. 

The topic has relatively little to do with specialty pharmacy, but it is part of the branch of pharmacy that is above the regular packaged drug dispensed at retail.

The topic of which we speak is Compounding…. a close cousin to specialty poharmacy. There are many similarities between specialty drugs and compounded drugs beyond just cost. As such, all staff working in a specialty pharmacy would benefit from at least a baseline understanding of those similarities and differences to gain as broad an understanding of the segment in which they work.

What follows is a list of key questions that can answer many questions about Compounding.

• How Is a Compounding Pharmacy Different from a Regular Pharmacy?
• What Are the Different Kinds of Compounded Medicines?
• Are There Different Types of Compounding Pharmacies?
• What Governs Compounded Drugs and Compounding Pharmacies?
• Do Compounding Pharmacists Have Education in Making Custom Medicines?
• Why Would I Need to Use a Compounded Drug?
• What Types of Drugs Are Compounded Most Often?
• Why Can Compounding Drugs Be Risky?
• Do Compounded Drugs Cost More Than Manufactured Drugs?
• How Can I Find a Compounding Pharmacy Near Me?

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What Is a Compounding Pharmacy?

When you have to go on a medication, your doctor will probably prescribe a premade one that’s FDA-approved. But sometimes, they may want you to go on a compounded medicine — one specifically made for your needs.

About 1% to 3% of prescriptions in the U.S. are for compounded drugs.

These medicines may be free of certain allergens or have a dosage that’s different than the preset dosages available. They may change the formula for an existing medicine to meet your specific needs.

The risks of compounded medicines are different so the laws that govern compounding pharmacies and compounded drugs differ from a traditional drug manufacturer or pharmacy.

How Is a Compounding Pharmacy Different from a Regular Pharmacy?

Compounding pharmacies sell custom-made medicines. Retail or hospital pharmacies (also known as community pharmacies) sell medicines made by manufacturers, but they can still offer certain compounded drugs. They just may not offer all the exact mixtures you need, especially if what you need must be made in a special setting to ensure safety. In that case, you may need to go to a compounding pharmacy, specifically.

There are about 56,000 community-based pharmacies in the U.S. Of them, about 7,500 pharmacies offer compounding services. Some estimates say that more than 32,000 pharmacies in the country offer some kind of compounding. About 40% of people who can compound drugs work in pharmacies that solely compound drugs.

What Are the Different Kinds of Compounded Medicines?

There are two kinds of medicines made at compounding pharmacies. 

• Sterile compounds are drugs that are injected into your tissue or blood, or put into your eyes. They have a higher risk for contamination than traditional pharmacies.
• Non-sterile compounds come in capsules, creams, and ointments.

Compounding pharmacies have to use pure, pharmaceutical-grade ingredients when they mix up a compounded drug. These must be made at a facility that’s registered with the FDA.

There are differences in how compound and traditional drugs are labeled, too. Compounded medicine labels don’t follow a format like FDA-approved drugs do. They aren’t regulated the same, either.

Are There Different Types of Compounding Pharmacies?

There are two types of compounding pharmacies:

• 503A compounding pharmacies. They can create a medication for you based on your doctor’s prescription. The compounding can’t be done by someone under supervision of a pharmacist. They’re mostly managed by the states.
• 503B compounding pharmacies. These are outsourcing facilities, which can make a medicine based off a prescription. They can also create large amounts of medicines and sell them to medical offices. The FDA manages most of these. They have stricter labeling rules than 503A pharmacies in an effort to avoid confusion.

What Governs Compounded Drugs and Compounding Pharmacies?

Although the FDA has approved the medicines used in a compounding pharmacy, it doesn’t approve compounded drugs like they do with traditional prescription drugs, such as the dosages The FDA can’t confirm if a compounded drug is safe or effective the way that they do with manufactured drugs.

State pharmacy boards manage compounding pharmacies in the U.S. But the FDA can oversee the safety of drugs used in compounding. The Drug Enforcement Administration (DEA) can do the same for controlled substances that are put into compounded drugs. Federal and state authorities can manage the labs and equipment used, too.

Compounded drugs have to follow industry standards, and the United States Pharmacopeia (USP) Convention sets those. If they’re made in an outsourcing facility, they have to follow good manufacturing practice (CGMP) rules that cover how they’re made, processed, and packed. 

In addition to licensed doctors, a licensed pharmacist, or someone supervised by one, can create a compounded drug.

They can be made in a pharmacy or outsourcing facility. The FDA and state pharmacy boards can inspect those facilities.

Do Compounding Pharmacists Have Education in Making Custom Medicines?

Pharmacists learn how to make their own versions of medicines in pharmacy school. Many states test how well they can compound before they get their license.

Many that work in compounding pharmacies have advanced training in compounding. States don’t require specific training, though. There’s no national specialty in drug compounding.

For example, pharmacists can earn a designation as a board-certified sterile compounding pharmacist (BCSCP). This means they meet standards for safety when preparing sterile compounded medications.

Why Would I Need to Use a Compounded Drug? 

If the medicine you need isn’t available in a premade form from a manufacturer, your health care professional might prescribe a compounded drug.

• Pharmacists can mix a drug to:
• Customized dosages
• Add flavor (for a child or pet)
• Change the form (perhaps from pill to liquid)
• Remove allergens or non-essential ingredients

If you’re concerned about taking a compounded drug, talk to your doctor. They may be able to tell if you’re better off on a compounded drug or if a traditional prescription will benefit you. This conversation could be important because a traditional drug may be just as effective — and it’ll probably be cheaper, too.

What Types of Drugs Are Compounded Most Often?

Some drugs are more commonly prescribed in compounded form. These include medicines for:

• Pain: Like baclofen, bupivacaine, cyclobenzaprine, diclofenac, flurbiprofen, gabapentin, ketamine, and lidocaine.
• Hormone replacement: Including, estradiol, estriol, progesterone, and testosterone.
• Dermatology: Doctors often combine different drugs to treat skin-related conditions.
• Animals: It’s common for doctors to prescribe compounded medications for animals and pets.

Compounding pharmacies can be handy when there are drug shortages as well. The pharmacists can essentially make the same formulations as a manufactured drug.

Why Can Compounding Drugs Be Risky?

Some compounding pharmacies and drug preparers may make misleading statements about the safety or efficacy of compounded drugs. They may not follow standards as with traditional, FDA-approved drugs.

If the mixture is contaminated, it can injure or kill people. This happened in 2012 when the New England Compounding Center (NEC) sent out compounded a methylprednisolone acetate solution free of preservatives. It contained fungus that caused 753 infections and 63 deaths.

Be aware about purchasing something from out of state because some states don’t have rules that are as strict as others in terms of quality.

Do Compounded Drugs Cost More Than Manufactured Drugs?

There’s limited data to show price differences between compounded drugs compared to traditional, manufactured drugs.

In 2012, the average cost for an ingredient in a compounded drug was $308.49, while ingredients in non-compounded drugs were $148.75. There was a 130.3% increase in the average ingredient cost for compounded drugs from 2012 to 2013. On the flip side, costs for non-compounded drug ingredients went up 7.7% during that time.

Check with your insurance plan to see how they may cover a compounded drug. They may cover it differently compared to manufactured drug. And you may want to know what they’ll pay before you fill the prescription to avoid any unexpected costs.

by Kristen Fischer

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are several LD deals that have been recently publicly confirmed subsequent to the approvals.

AllianceRx Walgreens Pharmacy will now distribute two medications indicated for adults with certain types of lymphoma

Lunsumio from Genentech is used to treat adults with follicular lymphoma whose cancer has come back or did not respond to previous treatment, and who have already received two or more lines of systemic therapy for their cancer.

Jaypirca from Eli Lilly treats adults with mantle cell lymphoma (MCL) that has come back or did not respond to previous treatment, and who have already received two or more lines of systemic therapy for their cancer, including a Bruton tyrosine kinase (BTK) inhibitor medicine. 

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Orsini Specialty Pharmacy will distribute topical gene therapy Vyjuvek

Orsini Specialty Pharmacy announced that Krystal Biotech has selected it as part of the limited specialty pharmacy network for Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy for the treatment of Dystrophic Epidermolysis Bullosa (DEB). 

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Option Care Health will distribute topical gene therapy Vyjuvek to include vials and pre-mixed therapy direct to health care providers

Option Care Health announced that it was selected by Krystal Biotech as part of a limited distribution specialty pharmacy network for Vyjuvek. Further, Option Care will serve as the only specialty pharmacy provider for mixing and delivering prepared Vyjuvek gel administration syringes to professional health care and home settings.

The FDA recently approved a new subcutaneous therapy, Epkinly (epcoritamab-bysp) from Genmab, for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including those with DLBCL arising from indolent lymphoma or high-grade B-cell lymphoma. DLBCL is a form of non-Hodgkin lymphoma.

DLBCL is an aggressive cancer type that can rapidly progress and resist treatment. The FDA approval of (Epcoritamab) represents a new treatment mechanism of action for third line DLBCL patients. DLBCL is fast-growing and the most common type of lymphoma accounting for 30,400 new cases in the United States each year.

Epkinly was approved with a boxed warning for Cytokine Release Syndrome and Immune Effector Cell-Associated Neurotoxicity Syndrome. Further, the package insert specifies a timed induction schedule over the first month with as dosage is increased. As such, the label also specifies that the therapy must be administered by a trained healthcare professional to monitor for adverse reactions especially over the first three months. 

The company confirmed pricing for Epkinly at $37,500 per month, based on an average length of therapy of around nine months but, indicated that monthly price should decrease after nine months due to less frequent dosing.

Distribution details were not announced. As the therapy must be administered by a health care professional, it is assumed that Epkinly will likely be available through specialty pharmacy distribution.

CLICK HERE to access prescribing information

The FDA just approved a new rare, GENE therapy, Vyjuvek (beremagene geperpavec-svdt) from Krystal Biotech, indicated for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB).  Vyjuvek is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home. It is a biological suspension mixed with excipient gel for topical application.

DEB is a rare and serious disease that affects the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene. Vyjuvek delivers two copies of the COL7A1 gene when applied directly to DEB wounds The COL7A1 gene is responsible for the production of functional COL7 protein that forms anchoring fibrils necessary to bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin).

The new topical stops blisters and wounds from developing on DEB patient skin. Prior to this approval there was no treatment other than simply applying bandages. Vyjuvek both heals wounds and prevents skin from re-blistering while correcting the underlying skin defect of DEB. Further, Vyjuvek doesn’t require supporting technology or specialized expertise making it accessible even to patients who live far away from specialized centers. 

Pricing for Vyjuvek was announced at a list price of $24,250 per vial, which translates to an estimated steady state cost of $631,000 per patient per year.  The label for Vyjuvek specifies that the therapy should be applied by a health care professional.  However, the company also stated that it can be administered in the home.

Administration is a bit complicated requiring atypical syringe filling steps using a syringe with a needle. However, the therapy is applied without penetrating the skin in a droplet pattern. The amount of therapy used depends on the number of wounds to be treated and their size. Assuming a patient or care giver can follow the filling process, the application of droplets on the wound not seem to require a health care professional (people with hemophilia routinely dose intravenously for example). That being said, the package insert does state that a health care professional should apply the therapy IN THE HOME as well.

Distribution details were not disclosed. As Vyjuvek is designated a rare therapy, the small number of patients and its ultra-high cost would lend towards a distribution plan via specialty pharmacy distribution.

Click Here to access the package insert

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Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, Vyjuvek

May 19, 2023

• Dystrophic Epidermolysis Bullosa (DEB) is a serious rare genetic disease that affects the skin and mucosal tissues and is caused by one or more mutations in the COL7A1 gene, resulting in lack of production of functional type VII collagen (COL7) protein
• Vyjuvek is a topical gel that addresses the genetic cause of DEB by restoring functional copies of the COL7A1 gene to patients and is the only medicine available for patients in the US
• Vyjuvek is approved for the treatment of patients six months of age or older with either recessive or dominant DEB
• Vyjuvek is approved to be administered by a healthcare professional either in a healthcare professional setting (e.g., clinic) or a home setting.

Krystal Biotech, Inc.Pittsburgh, May 19, 2023 (GLOBE NEWSWIRE) — Krystal Biotech, Inc. (the Company) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, today announced the US Food and Drug Administration (FDA) has approved Vyjuvek (beremagene geperpavec-svdt) for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB). Vyjuvek is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing. Vyjuvek is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home.

DEB is a rare and serious disease that affects the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene. The COL7A1 gene is responsible for the production of functional COL7 protein that forms anchoring fibrils necessary to bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears with minor friction or trauma. DEB patients suffer from open wounds, which lead to recurrent skin infections and fibrosis that can cause fusion of fingers and toes, and ultimately increase the risk of developing an aggressive form of skin cancer.

“This is a devastating disease,” said M. Peter Marinkovich, M.D., primary investigator of the GEM-3 trial, Director of the Blistering Disease Clinic at Stanford Health Care and Associate Professor of Dermatology at the Stanford University School of Medicine. “Until now, doctors and nurses had no way to stop blisters and wounds from developing on dystrophic EB patient skin and all we could do was to give them bandages and helplessly watch as new blisters formed. Vyjuvek topical gene therapy changes all of this. Vyjuvek both heals patient wounds and prevents skin from re-blistering because it actually corrects the underlying skin defect of dystrophic EB.”

The FDA approval of Vyjuvek is based on two clinical studies. The GEM-1/2 trial was an intra-patient, open label, single center, randomized, placebo-controlled study showing that repeat topical applications of Vyjuvek were associated with durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly with minimal reported adverse events. The GEM-3 trial was an intra-patient, double-blinded, multi-center, randomized, placebo-controlled study that met both its primary endpoint of complete wound healing at six months and its key secondary endpoint of complete wound healing at three months. Vyjuvek was well tolerated with no drug-related serious adverse events or discontinuations due to treatment-related events.

“Data from our GEM-1/2 trial and our GEM-3 trial, published in Nature Medicine and the New England Journal of Medicine, respectively, demonstrated the strength of both studies showing that Vyjuvek safely and effectively improved wound healing,” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “For so many years, all we have been able to offer DEB patients was palliative care, but now, based on the strength of the Company’s clinical trial data, there is a safe and effective FDA approved treatment.”

“Today’s landmark approval of Vyjuvek as the first redosable gene therapy ushers in a whole new paradigm to treat genetic diseases and is an important milestone for patients affected by DEB as well as their families and caregivers,” said Krish S. Krishnan, Chairman and Chief Executive Officer of Krystal Biotech, Inc. “We offer our sincere gratitude to DEB patients, caregivers, investigators, US regulators, and our employees who made this approval possible. For Krystal, this is a transformative achievement that highlights our commitment to developing and commercializing novel therapies for patients with rare diseases and demonstrates Krystal’s capability as a fully-integrated company ready to launch and bring Vyjuvek to patients as quickly as possible and deliver additional transformative medicines to patients as we advance our pipeline.”

Vyjuvek is expected to be available in the United States in the third quarter of 2023, and the Company will begin the promotion of Vyjuvek immediately. To meet the needs of patients, caregivers, and families as they start and continue their Vyjuvek treatment journey, the Company has developed Krystal Connect, a personalized support program. The program includes resources that can answer questions about Vyjuvek, verify health benefits, support treatment planning and administration and provide information about financial assistance for eligible patients. For more information, patients, caregivers and healthcare professionals can call Krystal Connect at 1-844-5-KRYSTAL.

With this approval, the FDA issued the Company a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The PRV program is designed to encourage the development of new drugs for the prevention or treatment of rare diseases.

“With the FDA approval of Vyjuvek, the DEB population has reached a monumental milestone in the treatment of this horrible disorder. Our hopes have now been realized for a safe and effective treatment for one of the most devastating symptoms of the disorder,” said Brett Kopelan, Executive Director of debra of America, the national organization dedicated to improving the lives of all people living with EB in the US. Mr. Kopelan added, “we thank Krystal for their dedication and commitment to bringing Vyjuvek to fruition. People living with DEB will now have a significant chance of having an improved quality of life and debra will continue to work closely with Krystal to assure patients have ready access to Vyjuvek.”

A survey just released by the Pharmaceutical Strategies Group confirmed what we already know….. that the COST of specialty medications is the tipppty toppest key concern of payers. They also updated a basket of other key measures that you will want to consider.

What is surprising in the survey is the agonizingly slow pace of adoption of value-based contracting. The survey found that just 12% of payers are now employing value-based models for pricey specialty therapies. 

Value-based contracting is arguably the fairest way to compensate for uber costly drugs that ‘may’ or may not work….. so why the lack of interest?

The reasons holding back value-based contracting are actually simple. 

It requires a mutually acceptable snd very detailed metric by which value is….. or isn’t….. determined.

It requires tracking and analysis….. and lots of it. 

And it requires patience….. lots of it…..  as it may take many months – or even years – to determine if a therapy finally delivers the results set forth in the metric….. sometimes on a patient by patient basis. 

It is also surprising that SPs haven’t been proactive in value-based contracting. 

OK, it isn’t likely that the payer and manufacturer will invite a lowly intermediary to such lofty discussions. But, that’s unfortunate as the SP can shoulder much of the heavy lifting in tracking, analyzing, measuring, and reporting progress towards the required metric. 

Isn’t that what SPs have been doing for manufacturers for years now?

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Survey: Why value-based contracting for specialty drugs remains rare

By Paige Minemyer, Fierce Healthcare

May 1, 2023 — Insurers gave a mixed review to copayment assistance programs, which offer coupons and other discounts to consumers to cover the cost of high-price drugs. 

Use of value-based contracting for specialty drugs is relatively rare, and payers cite multiple hurdles to rolling out these models, according to a new report.

The Pharmaceutical Strategies Group released its annual look at trends in benefits around specialty medications, where it surveyed more than 180 employers, insurers and labor unions. The survey found that just 12% are using value-based models for pricey specialty therapies.

Why is adoption so low? The surveyed payers said they need to see more evidence that the models are effective and also cited challenges in agreeing on and tracking outcomes. In addition, they said that resources and buy-in were lacking, making it harder to roll out value-based contracting for these therapies.

The survey also found that 14% of employers and 7% of health plans are deploying alternative funding models, while 14% and 33%, respectively, are exploring their use. However, 68% of those polled said they don’t view these models as sustainable.

Specialty pharmacy costs, mental health are top employer concerns. Here’s why

“As plans continue to seek ways to offset specialty drug costs, the market is divided as to whether alternative funding is a positive option or a detriment,” said Michael Lonergan, Pharmaceutical Strategies Group president, in a press release. “Affordability of specialty drugs for plans and members is driving this interest, while there is a concern about sustainability in these models.”

Insurers also gave a mixed review to copayment assistance programs, which offer coupons and other discounts to consumers to cover the cost of high-price drugs. Most (70%) said they were necessary to assist people in affording high-cost medications, however, 68% said that these programs encourage patients to take pricier brand-name drugs rather than select the lower-cost alternative medication.

Given the growing number of specialty drugs with eye-popping prices, costs in this area are a major concern for employers and payers in the long and short term, according to the survey. For instance, less than a quarter (24%) of those surveyed currently had a member taking a gene therapy drug, but 81% expect the affordability of these products to be a significant or moderate challenge in the next two to three years.

Insurers said the biggest concern in this space is ensuring parity in costs across both the medical benefit and pharmacy benefit, according to the survey, while employers said the largest hurdle is the affordability of cost-sharing for members. Due to their size, large employers are better positioned to manage these expenses, the survey said.

As these drugs grow in number, value-based contracts will prove vital, said Renee Rayburg, vice president of specialty at the Pharmaceutical Strategies Group, in the release.

“Rare disease treatments that are being targeted by cell and gene therapies will drive future spending,” Rayburg said. “[Value-based contracts] represent an effort into finding strategies for keeping costly specialty drugs accessible and affordable.”

However, as evidenced by the low uptake of value-based contracting found in the study, disrupting the existing paradigm is difficult. Just under half (49%) of those surveyed said they support eliminating rebates and instead deploying upfront discounts, down from 64% who said the same a year ago.

Most (95%) of the surveyed payers use prior authorization for these drugs, and 85% said they can track and report on their approval and denial rates, up from 66% in 2022. More than half (54%) said they have prior authorization in place for a branded drug that has a biosimilar available, and 51% said they use step therapy, which would require a patient to try a biosimilar first.

Sixty-two percent of those surveyed, however, said they were moderately or greatly concerned about the potential for unintended consequences related to prior auth, such as member dissatisfaction or care delays.

The hottest topic in the media these days is Artificial Intelligence (AI). It seems like everyone is talking about it. But that may be short sighted if you are neck deep into specialty pharmacy.

We’ve sent several reports recently on gene therapies because it is rapidly becoming ‘the next big thing’ in medicine. Analysts say that by the end of 2024 about 20 cell therapy launches and as many as 30 gene therapy launches are expected.  Oh MY!

Within the past few days, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted to back accelerated approval for SRP-9001 for patients with Duchenne muscular dystrophy with a decision due before month’s end. Such approvals are remarkable as these new ‘wunderkind’ therapies started to sprout a only a short time ago. 

So, what is the impact of these ‘one and done cures’? Our conclusion is that the specialty pharmacy model doesn’t coexist well at all. To date, SPs have had not role in dispensing or even distributing gene therapies given the hospital-based process to harvest patient samples for the brewing of the ultimate patient/genetically specific elixir.  Oh MY! Oh MY!

If you are interested in a basic primer around gene therapy CLICK HERE

If you are curious about how many gene therapies have been approved as of 4/2023 CLICK HERE

If you would like to see what others are saying about commercializing specialty drug cell andgene therapies CLICK HERE

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Critical Success Factors for Commercializing Specialty Drugs, Cell and Gene Therapies

Although payers have become somewhat accustomed to the high cost of specialty drugs, the price tags for gene therapies are causing “sticker shock.”

Apr 17, 2023 — Specialty product innovation is increasingly viewed as a journey that begins during pre-commercialization and continues throughout the product life cycle. Pharmaceutical and biotechnology manufacturers now recognize the need to develop, implement and refine a process-driven strategy that is built upon solid structural dynamics and a proactive approach for every phase of commercialization, spanning ideation, clinical research and development (R&D), clinical trials, product launch, market access, distribution and beyond. This strategic foundation gives life sciences companies and their people a clear pathway and focus to ensure that tasks and projects are aligned across teams and departments.

With the development of a potential new specialty drug, cell or gene therapy (CGT) typically requiring a decade or longer, there are critical success factors along every iterative step of the product life cycle that all point toward the same goal: achieving market dominance and capturing substantial market share. .

…………..CLICK ABOVE to access the full article

The FDA recently approved a new subcutaneous specialty therapy, Uzedy (risperidone) from TEVA and MedinCell, an atypical antipsychotic indicated for the treatment of schizophrenia in adults.

It may be surprising that we are reporting on a recent drug approval that was originally FDA approved 30 years ago….. yep, 1993. However, the new and improved Uzedy is a possible game changer for patients with schizophrenia. Till now, patients were prescribed either oral risperidone administered daily or intramuscular injections.

Sticking to the prescribed dosing for risperidone has been a huge challenge for patients with schizophrenia. However, Uzedy is now extended release with either monthly or bi-monthly dosing….. and, is also sub-q which makes this formulation much more patient friendly. The company said 3.5 million people in the U.S. are currently diagnosed with the condition and approximately 80% of patients experience multiple relapses over the first five years of treatment usually coupled with therapy discontinuance. 

Uzedy will be available in the U.S. in “the coming weeks” at a wholesale cost of $1,232 to $3,080 per month, depending on the dosage strength (on par with other IM versions of risperidone). Other injectable forms of risperidone are available at retail so it is likely that Uzedy will similarly be open access.

CLICK HERE for prescribing information

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New treatment provides adults living with schizophrenia a long-acting formulation that offers flexible 1- and 2-month dosing intervals

In a Phase 3 clinical trial, UZEDY demonstrated up to 80% reduction in risk of schizophrenia relapse versus placebo1

UZEDY is a subcutaneous injection from a pre-filled syringe with a 21-gauge needle

APRIL 28, 2023 PARSIPPANY, N.J., TEL AVIV & PARIS–(BUSINESS WIRE)– Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), and MedinCell (Euronext: MEDCL) announced today that the U.S. Food and Drug Administration (FDA) has approved UZEDY (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. UZEDY is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq™, a copolymer technology proprietary to MedinCell that controls the steady release of risperidone. Therapeutic blood concentrations are reached………………..

Click Here to access the full TEVA press release