Among the many earth-moving changes on the payer / PBM battlefield was last year’s announcement that Blue Shield of California was breaking away from its relationship with CVS’ Caremark and creating a very different model that would include multiple partners. According to the company, partners include Amazon Pharmacy, Abarca, Mark Cuban Cost Plus Drug Company, and Prime Therapeutics, with Caremark still hanging onto specialty pharmacies.

But wait….. BSofCA just announced another big change as part of its ‘Pharmacy Care Reimagined’ model that will further shake up the marketplace opening the door for other payers to emulate.

In short, BSofCA is dethroning Humira from what has been a sacrosanct position at the pinnacle of its specialty drug formulary. Instead, “Blue Shield is teaming up with drugmaker Fresenius Kabi and Evio Pharmacy Solutions to purchase a biosimilar for Humira at a transparent net price of $525 per monthly dose.” That price is significantly below the market reported net price of $2,100.

Moreover, Blue Shield confirmed that members will pay a $0 copay for the biosimilar. This makes the shift a big win financially for both the health plan….. and members. It is also believed that the no cost to member option will measurably boost persistence and improve overall health outcomes.

Details on how exactly Blue Shield will actually adjudicate and ‘pay’ for the meds were not announced. That, in itself, will be noteworthy. This should be fun to follow.

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Blue Shield of California inks deal to buy Humira biosimilar directly from manufacturer

CLICK HERE to read the article

……………………. catching up on FDA Approvals

The FDA recently approved a new infused therapy, Niktimvo (axatilimab-csfr) from Incyte Corporation and Syndax Pharmaceuticals, indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. 

Graft-versus-host disease is a leading cause of death from bone marrow transplants, which are used to treat certain blood cancers, solid tumors and immune disorders. It is estimated that approximately a third of those who receive bone marrow transplants experience GVHD.

Niktimvo doesn’t work like its predecessors which include Incyte’s Jakafi and Sanofi’s Rezurock. Niktimvo is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody that targets and blocks a protein involved in the development and survival of certain kinds of white blood cells.

Analysts suggest that Niktimvo will launch with a list price of $21,500 / month.

The company did not announce details for distribution.

CLICK HERE to access prescribing information

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FDA approves axatilimab-csfr for chronic graft-versus-host disease

CLICK HERE to access the FDA press release

The FDA recently approved an ORAL therapy, Livdelzi (seladelpar) from Gilead Sciences, Inc., for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

PBC is a rare, chronic, autoimmune disease of the bile ducts that affects approximately 130,000 Americans, primarily women, and is known to cause liver damage and, ultimately, liver failure if untreated. The disease currently has no cure.

Common symptoms include incessant itching or skin-crawling sensations, as well as debilitating fatigue that is made worse by the itching at night.

Gilead confirmed a list price of $12,606 for 30 tablets (10-mg) which works out to $151,272 per year.

Gilead also confirmed that oral Livdelzi will be distributed through specialty pharmacy.

CLICK HERE to access prescribing information

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Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by U.S. FDA

First and Only Treatment to Demonstrate Statistically Significant Reductions Across Key Biomarkers, ALP Normalization and Pruritus Versus Placebo –

CLICK HERE to read the full Gilead press release

PBMs are increasingly under the gun to remedy a variety of practices that are not considered consumer and provider network friendly. It is not surprising, therefore, that a PBM might want to tout some of the things that they have done to move the collective patient journey in a positive direction. But, it is in the short list of things that they are promising in the future that we find of greater interest. 

A short video interview with a senior leader at one PBM, OptumRx, recaps some of their successes over recent years but also opens the window on some noteworthy strategies in development.

The pipeline includes a more aggressive approach to cost containment especially through value-based strategies that go beyond pharmacy, in short, a ‘whole of government approach’….. ummm, a ‘whole of medicine approach’. This means embracing a big tent model of all providers that support specialty patients with complex needs…. often with multiple diagnoses. The evolving model must also develop a true cross-benefits model — Medical and Pharmacy to eliminate existing disparities. There’s more……

If you have 10 minutes, the video interview will add detail and other morsels of concepts upon which to chew. (link below)

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Optum Rx’s Vision for the Future of Specialty Care

OptumRx Healthcare Costs Value-Based Care Patient experience

Rising costs, technological advancements, the growing complexity of patient needs – these are all factors transforming the specialty pharma landscape at present.

CLICK HERE to access the video

…………catching up on FDA approvals

The FDA has approved a new therapy, Imuldosa (ustekinumab-srlf) from Accord BioPharma Inc., as a biosimilar to Janssen Biotech’s Stelara. Imuldosa is available in a subcutaneous form as well as an infusible.  

The approval of Imuldosa marks the fifth ustekinumab approval for the US. 

Imuldosa is slated to launch in May 2025 according to sources.

Imuldosa is indicated for—– 

Adult patients with:

• moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy;
• active psoriatic arthritis;
• moderately to severely active Crohn’s disease; and
• moderately to severely active ulcerative colitis.
• Pediatric patients 6 years of age and older with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy; and active psoriatic arthritis.

It is noted that prescribing information specifies subcutaneous dosing and administration for Psoriasis and Psoriatic Arthritis while specifying intravenous dosing instructions for Crohn’s Disease and Ulcerative Colitis.

CLICK HERE for prescribing information

While Imuldosa did not receive a Black Box warning there are a number of warnings and precautions consistent with the other previously approved Stelara biosimilars.

Pricing and distribution plans were not announced.

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FDA Approves Imuldosa, a Biosimilar to Stelara

CLICK HERE to read the article

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

Here are five LD deals that have been publicly confirmed subsequent to their approvals.

DirectRx to Distribute Ohtuvayre

DirectRx is now part of the U.S. pharmacy network distributing Ohtuvayre (ensifentrine), a newly approved therapy for chronic obstructive pulmonary disease (COPD). The Verona Pharma therapy was cleared by the U.S. Food and Drug Administration as an inhaled maintenance therapy for adults with COPD.

Amber Specialty Pharmacy to Distribute Ohtuvayre

Amber Specialty Pharmacy has announced that it has been named by Galderma to distribute Nemluvio (nemolizumab-ilto) for the treatment of Prurigo Nodularis in adults. The condition is a chronic, debilitating skin condition.

Orsini Specialty Pharmacy Exclusive Distributor for Miplyffa

Orsini Specialty Pharmacy has been chosen as the exclusive provider for Miplyffa (arimoclomol), the First FDA-Approved Treatment for Niemann-Pick Disease Type C, an ultra-rare and progressive neurodegenerative disease. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.

Biologics by McKesson to Distribute Voranigo

Biologics by McKesson has been selected by Servier Pharmaceutical as a specialty pharmacy partner for Voranigo (vorasidenib). Voranigo is approved for the treatment of adults and pediatric patients 12 years of age and older with grade 2 astrocytoma or oligodendroglioma and a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resection.

ONCO360 to Distribute Voranigo

Onco360 has also been selected by Servier Pharmaceutical as a specialty pharmacy partner for Voranigo (vorasidenib). 

Does the US pharmacy marketplace need another discount card?

The answer is a resounding ‘YES’…….. if that new card is the newest thing since sliced bread, the better mousetrap, the nitro charge in the road racer.

As noted in the article below, we will see the launch of a very different type of consumer Rx discount card on January 1st. And that card will be very, very different from the run-of-the-mill discount card.

First, the new card from Rx Save will offer consumers the same discount that they can get through other now available discount cards. BUT….. there will be a big difference. If the consumer wants to pay out of pocket, the Rx Save Card automatically searches deals from multiple discount cards, so they can choose the cheapest option. Now that should get your attention.

BUT WAIT…. it gets more interesting….. the consumer can also choose to purchase the drug through their employer’s insurance using the card at point of sale and the claim will be processed routinely as if it were a traditional PBM channel transaction. This option would be chosen if the cost through the employer’s insurance / PBM is lower than the lowest discount card option (theoretically that is possible but hard to imagine). 

Either way, ya got to be scratching yer head at this point!

We look forward to hearing how effectively this card works post launch.

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New Type of Pharmacy Discount Card to Launch in January

The Rx Save Card is a new, employer-funded, pre-paid pharmacy discount service that allows consumers to pick between PBM prices and prices available through traditional discount cards.

CLICK HERE to access the full article

…………………….catching up on recent FDA approvals

The FDA recently approved a new Sub-q therapy, Yorvipath (palopegteriparatide, developed as TransCon PTH) from Ascendis Pharma, to treat hypoparathyroidism in adults. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs. The disease affects an estimated 70,000 to 90,000 people in the United States.

Yorvipath is a once daily subcutaneous therapy. Dosage is individualized with the recommended starting dosage of 18 mcg once daily and is then titrated in 3 mcg increments or decrements with the goal of maintaining serum calcium within the normal range without the need for active vitamin D (e.g., calcitriol) or therapeutic calcium doses (elemental calcium >600 mg/day). Calcium supplementation sufficient to meet daily dietary requirements may be continued.

Patients must be monitored daily for clinical signs and symptoms of hypocalcemia or hypercalcemia.

CLICK HERE for prescribing information

Ascendis Pharma suggests a $150,000 to $160,000 wholesale acquisition price with about a $130,000 net price.

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FDA approves new drug for hypoparathyroidism, a rare disorder

The U.S. Food and Drug Administration has approved Yorvipath (palopegteriparatide) injection for subcutaneous use in adults with hypoparathyroidism. Yorvipath was not studied in adults with acute post-surgical hypoparathyroidism.

CLICK HERE to read the full FDA press release

……………..catching up on recent FDA approvals

The FDA recently approved a new therapy, Kisunla (donanemab-azbt) from Eli Lilly and Company, indicated for the treatment of adults with early symptomatic Alzheimer’s Disease (AD).  Approval includes people with mild cognitive impairment (MCI) as well as people with the mild dementia stage of AD, with confirmed amyloid pathology. 

Kisunla is administered monthly by infusion and the first and only amyloid plaque-targeting therapy with evidence to support stopping therapy when amyloid plaques are removed. As noted in our Reports on FDA approvals of other recently approved therapies for AD, amyloid is a protein produced naturally in the body that can clump together to create amyloid plaques. It is believed that excessive buildup of amyloid plaques in the brain can lead to memory and thinking issues associated with AD. Kisunla can help the body remove the excessive buildup of amyloid plaques.

The price of each vial of Kisunla is $695.65.  The total cost of Kisunla will vary by patient based on the removal of amyloid plaques to minimal levels as observed on amyloid PET imaging. As such, sample costs associated with the therapy can range from ~ $12,500 (6 months), to $32,000 (12 months), to $48,700 (18 months), etc.

The company indicated that Kisunla will launch through specialty pharmacy limited distribution.

Click here to access prescribing information

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Lilly’s Kisunla (donanemab-azbt) Approved by the FDA for the Treatment of Early Symptomatic Alzheimer’s Disease

Click here to access the full press release

So, you think you know everything about RARE DISEASES and how inexorably linked pharmacy, specifically specialty pharmacy, is to treatment development?

Can you pass the QUIZ?

The article below is a great quick read / study guide for anyone (shall we even say….. everyone) in specialty pharmacy today if they are serious about playing in the ‘rare’ sandbox with sufficient knowledge to earn a passing grade.

Here are some of the essential top line FAQs…..

·        How many people live with a rare disease today in the US?

·        How many people does it take for a drug to qualify for an Orphan Drug designation?

·        How many rare diseases and conditions are there?

·        What is the ARC Program run by CDER?

·        What are the four (4) expedited review programs and how do they differ?

·        And, last but not least, what the heck does CDER stand for? And how about CBER too?

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9 Things to Know About CDER’s Efforts on Rare Diseases

CLICK HERE to read the full article from the FDA

The Orphan Drug Act defines a rare disease as any disease or condition that affects less than 200,000 people in the U.S. There are approximately 25 to 30 million Americans living with a rare disease (about 1 in 10 people). Many rare conditions are life-threatening, and most do not have treatments. Accelerating the development of safe and effective drugs is CDER’s core mission, and CDER understands there are unique challenges in demonstrating the safety and effectiveness of drugs that treat rare diseases.