Author: Randy Chase

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.

………….. catching up on FDA Approvals

Here’s what we wrote back in July…..

The FDA recently approved a biosimilar….. big news…… huh?

But wait….. on the same day the FDA approved two biosimilars….. bigger news?

The earlier approvals included….. Yesafili (aflibercept-jbvf) from Biocon Biologics) and Opuviz(aflibercept-yszy) from Biogen, Samsung Bioepis.

BUT WAIT….. only weeks later the FDA has approved three more biosimilars for the same reference product! This has to be a record.

Shortly after the approvals originally noted, the FDA approved biosimilar Ahzantive (aflibercept-mrbb) referencing Eylea (aflibercept) from Formycon in late July for age-related macular degeneration.

Then the FDA approved Enzeevu (aflibercept-abzv) in August 2024.

Then the FDA approved another in mid September, Pavblu (aflibercept-ayyh) from Amgen also referencing Eylea .

The therapies are interchangeable biosimilars to reference product Eylea (aflibercept) from Regeneron. Ahzantive and Pavblu join Yesafili and Opuviz as vascular endothelial growth factor (VEGF) inhibitors and each is administered by intravitreal injection every 4 weeks for the first 5 injections, followed by 2 mg (0.05 mL of 40 mg/mL solution) once every 8 weeks.

Regeneron recorded U.S. sales of $5.72 billion from Eylea in 2023. Regeneron’s Eylea intravitreal solution (40 mg/mL) is around $2,000. 

We think we might need a bigger basket!

PBMs have been increasingly under the regulatory microscope for some time…. and recent focus has never been more intense. The pushback from the industry has also been palpable…. no surprise there….. because there is a lot of $$$s involved with even small changes to the industry model.

While the current election has significantly toned down media attention on PBM healthcare hegemony it is opportune to assess what has happened so far and what changes on the regulatory level can reshape the marketplace.

The article below is thought provoking.  

It frames the key issues and lays out some timely questions…..

• How the role of the PBM has changed over time?
• How drug pricing arrangements vary – and also how they don’t?
• Why cross-subsidization of revenue across drug categories is such a big issue and,
• Who are the new players and their connections to the larger system and the value they may bring…..?

……. just to point out a few.

It is fair to say that the outcome of next month’s election…… especially in Congress,  may have a major impact on the near term future (and fortunes) of PBMs in the US.

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Understanding the debate over PBMs

CLICK HERE to read the full article

…………catching up on FDA approvals

The FDA has approved a new therapy, Pyzchiva (ustekinumab-ttwe) from Samsung Bioepis, as a biosimilar to Janssen Biotech’s Stelara. Pyzchiva is available in a subcutaneous form as well as an infusible. Pyzchiva joins the ranks of only a handful of biosimilars to earn the title of interchangeability with the reference product. 

The approval of Pyzchiva marks the third ustekinumab approval for the US as well as the 14th interchangeable biosimilar.

Pyzchiva 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.

Prescribing information specifies subcutaneous dosing and administration for Psoriasis and Psoriatic Arthritis while also specifying intravenous dosing instructions for Crohn’s Disease and Ulcerative Colitis.

CLICK HERE for prescribing information

While Pyzchiva did not receive a Black Box warning there are a number of warnings and precautions.

Pyzchiva  will launch in early 2025 when Stelara’s patent exclusivity expires. As such, pricing and distribution plans were not announced.