Author: Randy Chase

Specialty pharmacies produce a lot of reporting…… but, the scope of this reporting may be missing the mark in the evolving competitive marketplace. The real challenge in this new environment will be all about outcomes. The challenge will be not in analyzing and reporting outcomes but, rather, what interventions were used to improve outcomes. Therein lies the rub.

Two articles were recently published that drilled down into the deeper layer of outcomes reporting by forcing health care providers to demonstrate how they are proactively changing outcomes through targeted interventions. Note that we say ’health care providers’, not just the pharmacy. The way to proactively impact outcomes will require participation by multidisciplined, non-siloed teams. Hospital systems are using this mantra to promote their differentiated specialty pharmacy model. 

One article lays out a roadmap to consider to navigate these new waters starting with a three-step process

• Environmental scan (developing a list of all potential measures, include quality measures / guidelines for each disease state).
• Expert engagement (rely on subject matter experts to identify the most important measures.)
• Implementation (include front line and informatics expert members in a dedicated team.)

Develop a strategy to produce better outcomes documentation include;

• Determine the value story
• Identify the outcomes that demonstrate value (not just numbers). 
• Define outcome goals
• Create targeted data elements to document outcomes
• Collect the data to report meaningful outcomes
• Report outcomes to key stakeholders 

The kind of outcomes reporting suggested will be hard…… no shortcuts here.

However, it may be the key differentiator that the market will start to demand.

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CLICK each title to access the full article

Start Tracking Outcomes in Specialty Pharmacy Now

Using Data-Driven Monitoring Tools Boosts SP Outcomes

Another milestone has been passed in the advancement of specialty pharmacy therapies. The FDA has approved not one, but two, gene therapies for a major disease, Sickle Cell Anemia. Most noteworthy is that one of the approvals offers real proof of concept for CRISPR gene-editing technology.

FIRST….. The FDA has approved a unique therapy, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Casgevy is an autologous genome edited hematopoietic stem cell-based gene therapy. 

Sickle cell affects an estimated 100,000 people in the United States. The disease can impact any race, however, the black community is the most affected with 90+% of all diagnosed cases. In sickle cell disease, the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body’s tissues.

The process for Casgevy therapy requires significant preparation. Stem cells harvested from the patient’s bone marrow are sent to manufacturing facilities where they are edited using CRISPR/Cas9 technology. Once the cells are incubated, they are infused back into the patient during a month-long hospital stay.

Second….. The FDA also approved another therapy, Lyfgenia (lovotibeglogene autotemcel) from bluebird bio. This gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene. Lyfgenia is also an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.

The key difference is that Casgevy introduces a manufacturer, gene-edited therapy vs. Lyfgenia which is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease.

Refer to Prescribing Information for each therapy for more detail on these complex processes especially around dosage and administration.

CLICK HERE to access Casgevy label 

CLICK HERE to access Lyfgenia label 

Neither company disclosed launch pricing at time of approval.

Neither company disclosed distribution plans at this time.

CLICK HERE to read the full press release

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

Orsini Specialty Pharmacy to Distribute Adzynma 

Orsini Specialty Pharmacy has been selected by Takeda Pharmaceuticals as the exclusive specialty pharmacy partner for ADZYNMA (ADAMTS13, recombinant-krhn) for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA is the first and only FDA-approved recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy for people 

Onco360 to Distribute Fruzaqla

Onco360 has been selected as a pharmacy partner by Takeda for Fruzaqla (fruquintinib an oral targeted therapy for certain adults with metastatic colorectal cancer (mCRC).  Fruzaqla is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status. Fruzaqla is a selective oral inhibitor of VEGFR-1, VEGFR-2, and VEGFR-3, which play a pivotal role in blocking tumor angiogenesis.

Biologics by McKesson to Distribute Truqap

Biologics by McKesson announced that it has been selected by AstraZeneca as a specialty pharmacy provider for Truqap (capivasertib), an orally administered drug, in combination with fulvestrant, for the treatment of adult patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1, or PTEN).

Walgreens’ (WBA) AllianceRx Pharmacy to  Distribute XDEMVY

Walgreens Boots Alliance’s WBA wholly-owned subsidiary — AllianceRx Walgreens Pharmacy — will distribute Tarsus Pharmaceuticals’  XDEMVY (lotilaner ophthalmic solution) 0.25%. AllianceRx Walgreens is one of the four specialty pharmacies selected to distribute the FDA-approved treatment for Demodex blepharitis (DB), a highly prevalent eyelid disease.

Biologics by McKesson to Distribute Ogsievo

Biologics by McKesson has been selected by SpringWorks Therapeutics as a limited distribution specialty pharmacy for Ogsiveo (nirogacestat). Ogsiveo is a gamma-secretase inhibitor indicated for adult patients with progressing desmoid tumors who require systemic treatment.

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

The FDA recently approved a new ORAL therapy, Velsipity (etrasimod) from Pfizer, for adults with moderately to severely active ulcerative colitis (UC).  Velsipity is a once-daily, oral, sphingosine 1-phosphate (S1P) receptor modulator that selectively binds with S1P receptor subtypes 1, 4, and 5.

UC is a chronic, debilitating condition affecting about 1.25 million people in the United States. Symptoms often include chronic diarrhea with blood and mucus, abdominal pain, and urgency.  The therapy provides patients the opportunity to achieve steroid-free remission.

Velsipity has launched with a wholesale acquisition cost is $6,164 for 30-day supply or ~$75,000 annually. Pfizer did not disclose distribution details although, given its cost, it is highly likely to be dispensed only through specialty pharmacy limited distribution.

CLICK HERE to read prescribing information

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U.S. FDA Approves Pfizer’s Velsipity for Adults with Moderately to Severely Active Ulcerative Colitis

CLICK HERE to read the full press release

There has been scant news about any big moves by large national specialty pharmacies this year. Today, however, we read about a move by CarelonRx, the specialty pharmacy operated by Elevance Health.

Many of you may be scratching your heads trying to figure out the story behind these entities. We are happy to provide some relief.

In late 2022 Elevance Health acquired long-running BioPlus Specialty Pharmacy as it sought to expand its specialty pharmacy services. (BioPlus was one of the few mature SPs that had not been acquired and taken out of the independent SP column.) More significantly, Elevance is the insurer formerly known as Anthem Health….BioPlus was then integrated into Elevance’s – then branded – PBM, IngenioRx. 

We continue to ask….. Why are insurers keen on integrating SPs into their portfolios?

In a word, money….. and leverage (guess that’s two words). A well-run SP with a solid revenue stream is very profitable. Elevance appears poised to supercharge its market presence both ‘digitally’ and geographically which enables greater steerage of plan members into their specialty pharmacy and supercharge profit opportunity.  And…… PBMs love leverage over manufacturers. Being able to put large specialty purchases on the table goes a long way in manufacturer negotiations.

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Elevance Health’s CarelonRx rolling out new digital pharmacy

Dec 5, 2023

The CarelonRx Pharmacy launch is one of several steps that the company is taking to improve the experience for members.

Elevance Health’s pharmacy benefit manager is launching a new digital pharmacy that aims to make it easier for members to track their prescriptions.

CarelonRx Pharmacy will launch on Jan. 1, and members will be able to connect with pharmacists via text, chat or phone around the clock. The platform will also allow them to check the price of medications for comparison and track their prescriptions throughout the order process, similar to how a customer may follow a pizza order.

……………………………….article continues

CLICK HERE to access the full article

………..catching up on recent FDA approvals

The FDA recently approved a new infused therapy, Pombiliti (cipaglucosidase alfa-atga) from Amicus Therapeutics, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy. 

Pombiliti is approved only in combination with Opfolda (miglustat), an oral drug that is to be administered approximately 1 hour before the infusion of Pombiliti. 

Pombiliti is a recombinant human GAA enzyme (rhGAA). Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. Pombiliti / Opfolda will be distributed in tandem. 

About 5,000 to 10,000 people have been diagnosed globally with Pompe. The majority of patients on current standard of care decline after about two years. The previous standard of care has been Sanofi’s Lumizyme. 

The combo therapy launched at an annual price of $650,000 for a patient weighing about 70 kg.

Pombiliti will be distributed by Orsini Specialty Pharmacy according to a recent press release.

Pombiliti was approved with a black box warning.

CLICK HERE to access prescribing information

In the wacky world of specialty pharmacy, the therapies that get the most attention and press coverage over the past couple of years are the big ticket items. At the tippity top of that list are the new gene and cell therapies that have smashed the multi-million-dollar per patient cost barriers. And, at the very top of that auspicious list, are the new FDA approved therapies for hemophilia with price tags that range from about $3 million to $3.5 million….. yes, the most expensive therapy approved so far.

The article being offered for your reading pleasure today won’t offer any surprises for those of you that regularly support the hemophilia patient community. However, those working  within the specialty pharmacy sphere would benefit from a better understanding of why the gene therapies for hemophilia are actually easier to cost justify than most other gene therapies. 

First, their efficacies are readily measurable vs. traditional blood factor products…. a more apples to apples comparison.

Secondly, gene therapies for other rare conditions are essentially ‘cures’….. where, before hand, there were mostly only therapies that managed symptoms….. a more apples to oranges comparison.

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Why Are Hemophilia Gene Therapies So Expensive?

Biomarin and CSL Behring’s haemophilia gene therapies have boosted innovation in the field, but assessing their cost effectiveness is less than straightforward.

he treatment landscape for haemophilia has undergone significant advancements following two pathbreaking FDA approvals of gene therapies for the inherited blood disorder. However, the cost and the value they provide to patients and society continue to receive scrutiny.

………………………….article continues

CLICK HERE to read the full article

Catching up on recent FDA approvals

The FDA approved a specialty therapy…. with a difference….. it is a topical ointment. The approval for the new therapy, Ycanth (cantharidin) from Verrica Pharmaceuticals, is indicated for Molluscum Contagiosum in adult and pediatric patients 2 years of age and older.  Molluscum Contagiosum is a viral skin infection that cause itchy skin reactions in almost any area of the body. The virus is transmitted by skin-to-skin contact and is most common among children under 10.

Since it is a topical, the first assumption was that specialty pharmacies would be able to dispense this new therapy, however, that’s not the case. It must be administered only by health care providers every 3 weeks as needed. That may be related to one of the serious side effects for Ycanth….. loss of the outer layer of skin at the application site.

The label also includes an unusual patient instruction….. Ycanth is flammable, even after drying. Patients should avoid fire, flame, or smoking near lesion(s) during treatment and after application until removed. 

Cost of Ycanth will depend on the number of lesions. 

A single carton of 12 applicators is priced at $8500 according to GoodRx.

CLICK HERE to read the full FDA press release

CLICK HERE to read the Ycanth prescribing information

Continuing our deep dive into biosimilars

It may well take the wisdom of Solomon to fully appreciate the logic of the recently released guidance (or non-guidance?) on communicating biosimilar interchangeability. 

The guidance treads a fine line. It says that (many??) prescribers are already picking a biosimilar because it was approved as being the same as the reference product and, therefore, can be ‘considered’ interchangeable…. even if the FDA did not pin the official interchangeable designation medal on its chest.

Only four biosimilars have been Olympic interchangeable medal winners to date. The press release below touches on the fact that this designation is important since many state pharmacy laws / regulations require pharmacist substitution. However, qualifying for FDA interchangeable eligibility requires jumping through several more hoops….. and that translates into time and money. None the less, some biosim manufacturers are pursuing the designation post-launch.

So, the FDA is soft peddling the way interchangeability is communicated. They essentially say that it should not be mentioned in the package insert / prescribing information since the prescriber is free to refer to the Purple Book for that information. That is a good example of the definition of the word ‘befuddling’.

In short, this conundrum is likely to continue for some time.

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ARE YOU NOT INTERCHANGED? 

FDA’S RECENT BIOSIMILAR LABELING GUIDANCE CHIPS AWAY AT INTERCHANGEABLE PRODUCT ADVANTAGES

Key Takeaways:

• Interchangeable biosimilar products would not be allowed to disclose or explain their interchangeability designations in their products’ labels under FDA’s new draft guidance.
• FDA justified its reversal on including the interchangeability disclosure in labeling by inferring that prescribers pay little heed to interchangeability information in labeling when considering biosimilars for patients.
• This labeling policy eliminates an important advantage of interchangeability designations and hints at a broader agency trend to minimize the designation’s significance.

The Food and Drug Administration (FDA) released a new Draft Guidance document…..

……….. press release continues

CLICK HERE to read the full press release

Continuing our focus on Biosimilars

The FDA recently approved a new infused biosimilar, Tofidence (tocilizumab-bavi) from Biogen, for the treatment of moderately to severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis. Tofidence is a biosim to reference product Actemra, a first competitor for that brand (although at least there is one additional biosim candidate inching its way towards approval).

The approval of this biosimilar is noteworthy if only for the fact that yet another unchallenged brand has fallen to a biosimilar competitor. As more are approved, biosimilars will be challenging a much larger group of brand name products….. further eroding their near monopoly in many disease categories.

Tofidence was approved without an ‘interchangeable’ designation….. which many still feel is the jewel of biosimdom (yes, new word). It was also approved with a Black Box Warning.

CLICK HERE to view prescribing information

Biogen did not release pricing for Tofidence at time of approval. 

Now, weeks later, pricing info is still not to be found.

Biogen did not release details on distribution.

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FDA Approves Biogen’s Tofidence, a Biosimilar Referencing ACTEMRA

Tofidence becomes the first tocilizumab biosimilar to gain FDA approval in the United States

CAMBRIDGE, Mass., Sept. 29, 2023 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) announced that the U.S. Food and Drug Administration (FDA) has approved Tofidence (tocilizumab-bavi) intravenous formulation, a biosimilar monoclonal antibody referencing ACTEMRA. The Tofidence intravenous formulation is approved for the treatment of ………………………….. press release continues

CLICK HERE to read the full press release