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Can You Explain 503A?

We sent a Report on compounding back in July. At that time we were alerting those pharmacies that engage in compounding that numerous new standards were being rolled out. Today there is another issue to which compounders need to be very attuned. If your specialty pharmacy isn’t into compounding….. read on regardless and learn something about your industry.

The focus of today’s Report is on 503A compounding and the regulations that restrict that practice. In short, sections 503A (and 503B) of the FDA Act set conditions under which compounded drug products are exempt from certain federal requirements.  One common exemption is when a drug in short supply. That has never been as tangible a problem as it has been of late with a big increase in the number of drugs not available either due to shortages, product discontinuation, or other supply chain issues. However, there is a BIG caveat….. compounding should not be conducted “regularly or in inordinate amounts.”  That means that a compounder should not produce the ‘copy’ in large batches (i.e., manufacturing levels) and that each dose compounded should be a result of a valid prescription (i.e., not anticipated demand).

Manufacturers are alert to compounders that cross the line….. going from reactive compounding to proactive manufacturing. That’s when PHARMA lawyers get involved with lawsuits claiming everything from patent infringement, to trademark infringement, to unfair competition….. and so forth and so on. There are federal and even state laws and Boards of Pharmacy regulations that have cropped up around these issues….. the lawyers are the only ones who are happy.

If you are interested in digging into the nitty gritty of the maze that surrounds compounding and 503A spend a few minutes to read the article.

CLICK HERE to access the full article


Legal Battles Intensify: Pharmaceutical Manufacturers’ Lawsuits Targeting Compounding Pharmacies

Analyzing the patent and trademark challenges faced by 503A compounding pharmacies.

Pharmacy compounding is a fundamental component of pharmacy practice and is essential to the provision of health care. However, for years, pharmaceutical manufacturers have been filing lawsuits against compounding pharmacies, accusing the pharmacies of infringing on their patents.

This rise in legal actions is driven by the growing prevalence of compounding practices over the years, despite the heightened regulatory oversight imposed on 503A compounding at the federal level through the Drug Quality & Security Act (DQSA),1 and at the state level through state boards of pharmacy.

Lawsuits often have claims based on…….  the Pharmacy Times article continues


Ambulatory Infusion – It’s Alive!

In recent weeks two leading specialty pharmacies announced their plans to strategically expand their footprint in the specialty pharmacy ambulatory infusion space. Curiously, that falls on the heels of Walgreens’ divestiture of Option Care which, only a year ago, was the largest infusion provider in the US. That move may have been heavily influenced by Walgreens’ desire to pay down debt, continue support of the company’s strategic priorities, and further drive forward its transformation to a consumer-centric healthcare company.

The two providers of which we speak are Soleo Health and Hy-Vee Health. One may ask if they saw Walgreens’ exit as an wiggle room to further grow their positions in the ambulatory infusion segment.

Soleo Health announced that it is opening an ambulatory center in Cincinnati offering a full range of specialty infusion therapies as well as pharmacy dispensing services. More recently, Soleo announced the opening of a Boise, Idaho, pharmacy and infusion center location. The new facilities add to Soleo’s 23 pharmacy locations nationally as well as more than 40 infusion centers throughout the U.S.

Hy-Vee announced the opening of its first Hy-Vee Health infusion clinic in West Des Moines, Iowa. The freestanding clinic is staffed by licensed physicians and nurses. Aaron Wiese, president of Hy-Vee, Inc., said “Our vision for Hy-Vee Health is to provide convenient, cost-effective health solutions for patients across the United States.” Even more recently Hy-Vee announced the opening of a new clinic in Chicago.

If one looks at the number of ultra-high-cost infused therapies approved in the past few years one can make a strong case for ambulatory specialty infusion….. especially if it is a lower cost site of service vs. hospital outpatient facilities….. something that payers are getting more bullish about.


Limited Distribution Updates

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 is a basket full of LD deals that have been recently publicly confirmed subsequent to FDA approval.

AcariaHealth to dispense Elevidys

AcariaHealth announced that it has been selected by Sarepta Therapeutics, Inc. as part of the limited distribution network for gene therapy Elevidys (delandistrogene moxeparvovec-rokl), approved for the treatment of ambulatory pediatric patients aged four through five years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. 

Amber Specialty Pharmacy Selected to Dispense Vowst

Amber Specialty Pharmacy has been selected as one of only two pharmacies nationally selected by Aimmune Therapeutics, to support the launch of Vowst indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI).

Orsini Specialty Pharmacy to dispense Vowst

Orsini Specialty Pharmacy announced that Aimmune Therapeutics has selected it as a limited distribution partner for Vowst (fecal microbiota spores, live – brpk) indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI). 

Orsini Specialty Pharmacy to dispense Austedo

Orsini Specialty Pharmacy announced that they now has access to Austedo XR (deutetrabenazine) for treating Tardive Dyskinesia (TD) and Chorea associated with Huntington’s Disease (HD).

Orsini pharmacy selected to supply Roctavian

Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A. The one-time treatment from BioMarin Pharmaceutical is the first gene therapy ever approved by the FDA for eligible adults.

PANTHERx Rare selected to supply Rystiggo

PANTHERx Rare announced that it has been selected by UCB as a limited distribution partner to provide Rystiggo (rozanolixizumab-noli) FDA-approved treatment for both anti- acetylcholine receptor (AChR) anti- muscle-specific tyrosine kinase (MuSK) antibody positive adult patients with generalized myasthenia gravis (gMG).


FDA Approves Oral Tx for AML – Vanflyta

Catching up on recent specialty approvals————

The FDA recently approved a new ORAL, kinase inhibitor therapy, Vanflyta (quizartinib) from Daiichi Sankyo Company, Ltd., in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated.

Vanflyta was approved with a Black Box warning and REMS. 

CLICK HERE to access prescribing information

AML is one of the most common forms of leukemia in adults and an estimated 20,380 new cases diagnosed in the U.S. in 2023.  Up to 37% of newly diagnosed patients with AML have a FLT3 gene mutation and approximately 80% of these are FLT3-ITD mutations, which drive cancer growth and contribute to increased risk of relapse and shorter overall survival.  The five-year survival rate for patients with FLT3-ITD AML has been reported at approximately 20%.

VANFLYTA is the first and only FLT3 inhibitor to be approved specifically for FLT3-ITD positive AML and across the three phases of treatment – induction, consolidation and maintenance in patients without transplant – for newly diagnosed AML.

Daiichi Sankyo will launch Vanflyta at $546 per tablet. Total cost per patient will vary based on number of cycles (induction, consolidation, maintenance). By comparison, Xospata, another FLT3 therapy, comes with the highest price tag at $750 a day.

Distribution logistics were not announced at the time of approval. However, it is expected that Vanflyta will launch through specialty pharmacy limited distribution.


Vanflyta First FLT3 Inhibitor Approved in the U.S. Specifically for Patients with Newly Diagnosed FLT3-ITD Positive AML

First and only FLT3 inhibitor approved across three phases of AML treatment, when added to chemotherapy, improved overall survival

Jul 20, 2023 TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)– Daiichi Sankyo (TSE: 4568) today announced that Vanflyta (quizartinib) has been approved by the U.S. Food and Drug Administration (FDA) in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated.


ABC Shares the abcs on Biosimilar Trend

Earlier this week we sent a Report on biosimilar utilization and trends. Today we follow up with some stats from another credible source that add a few new insights.

AmerisourceBergen Corp (ABC) recently published results from a survey of payers related to treatment of biosimilars within their coverage policies. The findings are worth considering for future planning.

Top line highlights—

Organization types: 51 payors / advisors representing 300 million covered lives

(about 90% of the U.S. population)

  • 55% health plans
  • 24% integrated delivery networks, and
  • 22% pharmacy benefit managers
  • 45% said their organization would allow multiple biosimilars
  • 35% said they would select a single preferred biosimilar and
  • 4% said they would prefer the reference product.
  • 16% selected ‘Other’ (hmmmmm??)
  • 96% and 82% of respondents, respectively, said that lower price and contracting arrangements (legal speak for rebates) continue to drive preference for a reference product.
  • 88% of respondents said that interchangeability will inevitably force the shift to greater biosimilar adoption.


What’s Driving Biosimilar Decision Making? AmerisourceBergen Payor Survey Has Some Answers

Biosimilar competition is heating up, with eight biosimilars to adalimumab (Humira, AbbVie) already launched in 2023 and more on the horizon. Other reference products also have anywhere from two to five biosimilar competitors. With this intense level of competition, payors are more likely to select multiple biosimilars for the same product, rather than a single biosimilar or the reference product only, according to a survey from AmerisourceBergen…..

CLICK HERE to read the full article

by Gina Shaw, Pharmacy Continuum


New Insights on Payer Policies for Biosimilars

A recently released study on payor practices related to biosimilars was detailed in an article published by the Center for Biosimilars….. and some of the findings are thought provoking.

Here are the highlights from the study:

  • The study identified 1181 coverage decisions for 19 commercially available biosimilars, representing 7 reference products and 28 indications
  • Health plans dictated coverage exclusions or step therapy limitations on biosimilars in 229 (19.4%) of the 1181 coverage decisions
  • Payers were more likely to restrict biosimilar coverage for pediatric populations whereas the reverse was observed for all other categories, including Oncology, for specialty biosimilars.
  • Biosimilar utilization was higher for diseases with US prevalence higher than 1,000,000 cases
  • Biosimilar utilization was higher if the health plan did not contract with 1 of the 3 major pharmacy benefit managers
  • Payers were less likely to enforce restrictions on the biosimilar if:

— the biosimilar was indicated for cancer treatments 

— if the product was the first biosimilar to market

— there were two available biosimilar competitors, and

— could generate annual list price savings of more than $15,000 per patient. 

  • However, payers were most restrictive if the biosimilar did not have a cost-effectiveness measure available.

While many of the findings are confirmatory, the report doesn’t address the underlying dynamics of how rebates affect payer policies over preferred utilization of reference products.


Payers Reveal Most Significant Factors Impacting Biosimilar Coverage Decisions

Jun 29, 2023

Justina Petrullo    AJMC Center for Biosimilars

It was found that cancer treatment, pediatric population, and coverage restriction of reference products were revealed as some of the strongest factors associated with biosimilar coverage decisions by US commercial health plans relative to reference products.

Cancer treatment, pediatric population, and coverage restriction of reference products were discovered as some of the most significant factors associated with biosimilar coverage decisions by commercial health plans in the US relative to their reference products, according to BioDrugs.

CLICK HERE to access the full article


Is Congress Ready to Stomp on Step Therapy?

We’ve written recently on legislation grinding its way through Congress targeting the PBM industry and its various business practices that industry stakeholders say are unfair and must be remedied. The article below adds yet another practice to the list we’ve already shared…..Step Therapy.

Step therapy has been around for many decades in one form or another. In its earliest iteration it was simply – ‘Let’s try this first to see if it works…. and then we can try these ten other drugs to see what happens!’ Things have advanced significantly since then. What we have now is ‘Let’s try this less costly, legend therapy before we try the newest, uber-expensive therapy.’ 

While there is an argument for efficiency in managing overall healthcare spend, step therapy can be problematic for many patients who expend time that they may not have experimenting with a therapy that may only manage symptoms vs. a new therapy option that may be disease modifying. 

It appears that language restricting step therapy has been included in the Pharmacy Benefit Reform Act slowly making tis way through the US Senate. The bill has been bouncing around the Senate since 2017 even with bipartisan support. Given the momentum that other bills are gaining in Congress it is hoped that the step therapy bill will be incorporated in a final version.

It is noteworthy that thirty states have already enacted step therapy legislation similar to the provisions in the Safe Step Act. In those instances, the state laws apply only to health plans subject to those states’ laws but do not impact ERISA qualified health plans.

CLICK HERE to read the full article to learn about provisions in the bill that specify timing requirements for step therapy appeals as well as five exceptions where a patient may qualify for an exemption from step therapy.


Step Therapy

Jun 19, 2023 — Legislation designed to smooth the path to exemptions from step therapy has been incorporated into the Pharmacy Benefit Reform Act, one of several bills targeting pharmacy benefit managers that is making legislative headway in the Senate. Legislation that aims to make it easier for patients to get exemptions from the step therapy requirements has been folded into one of the several U.S. Senate bills targeting pharmacy benefit managers (PBMs), and patient and provider organizations are optimistic about PBM legislation — in one form or another — becoming law.

“PBM reform is going to pass in this Congress — (although) I wouldn’t say by the end of this year — and it will include step therapy reform,” Erin McKeon, associate director, federal advocacy, for the Crohn’s & Colitis Foundation.

The current Congress, the 118th, is scheduled to adjourn at the…………

CLICK ABOVE to access the full article


FDA Approves Weekly Sub-q hGH – Ngenla

If you’ve been waiting anxiously (must ask why?) for yet another human growth hormone to be approved….. then wait no longer.

The FDA recently approved a new therapy, Ngenla (somatrogon-ghla) from Pfizer, a once-weekly, human growth hormone (hGH) analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone.

Human growth hormone works by replacing insufficient growth hormone in the body. Ngenla is administered subcutaneously and must be dosed based on patient weight. Ngenla is available in two single-patient-use, disposable, prefilled pens: a 24 mg/1.2 mL (20 mg/mL) with 0.2 mg increments and also a 60 mg/1.2 mL (50 mg/mL) pen with 0.5 mg increments.

Various clinical sources cite prevalence levels of ~1/ 4000-10000 children aged 3+-18 years old with inadequate secretion of endogenous growth hormone. There are an estimated 65 million children between 3 and 18 years old in the US. That translates into a potential target patient population of only about 5,000-10,000 patients who may require therapy.

Pfizer confirmed that Ngenla will launch at a monthly list price of about $8,300 for a child weighing 35 kg. It is expected to become available starting in August. Distribution details were not disclosed.

Here is a recap of the now 14 various hGH products approved by the FDA:

  • Geref Diagnostic sermorelin 1990
  • Norditropin (Pro) somatropin 1995
  • Norditropin FlexPro somatropin 1995
  • Serostim (Pro) somatropin 1996
  • Saizen (Pro) somatropin 1996
  • Geref  sermorelin 1997
  • Nutropin AQ (Pro)  somatropin 1999
  • Genotropin (Pro) somatropin 2001
  • Zorbtive (Pro)  somatropin 2003
  • Accretropin somatropin 2008
  • Zomacton  somatropin 2015
  • Macrilen (Pro)  macimorelin 2017
  • Skytrofa (Pro)  lonapegsomatropin 2021
  • Sogroya (Pro) somapacitan-beco 2023
  • Ngenla somatrogon-ghla 2023


FDA Approves Pfizer’s Ngenla, a Long-Acting Once-Weekly Treatment for Pediatric Growth Hormone Deficiency

June 28, 2023 — NEW YORK & MIAMI–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. 

CLICK HERE to access the full press release


FTC Saying PBMs Got Some Splainin’ to Do

The outcome of the FTC review of PBM practices and its report on needed corrections may be some of the biggest news items this year. As the article below details, the FC has basically dumped as totally outdated nine advocacy letters dated between 2004 and 2014, These letters advocated against proposals to increase regulatory oversight and transparency of PBMs….. no bias there!

So, where is the FTC shining its regulatory spotlights?

The inquiry is aimed at shedding light on numerous practices that have come under industry stakeholder attacks, including—-

·        fees and clawbacks charged to network pharmacies

·        steering patients towards PBM-owned pharmacies

·        potentially unfair audits of network pharmacies

·        complicated and opaque methods to determine pharmacy reimbursement

·        the prevalence and equity of prior authorizations and other administrative restrictions

·        the use of specialty drug lists and surrounding specialty drug policies, and 

·        how rebates and fees paid by drug manufacturers affect the NET cost of drugs to payers

·        no pass-through of rebate savings resulting and higher out-of-pocket costs to patients.

That’s quite a list! Relief on any one of which could have significant consumer impact.

As part of this inquiry, the FTC has sent compulsory orders to CVS Caremark, Express Scripts, OptumRx, Humana, Prime Therapeutics, and MedImpact.

The next phase of the FTC investigation might really be a doozie!


FTC Withdraws Advocacy for Pharmacy Benefit Managers

The Commission rescinded earlier guidance that was opposed to increased regulatory oversight and transparency of PBMs.

JUL 25 — Claiming that its old policy statements no longer reflect current market dynamics, the Federal Trade Commission has voted to rescind its prior statements of advocacy for pharmacy benefit managers, effectively ending the agency’s previously stated endorsement of PBMs.

According to the FTC, the new statement pulling its advocacy is a response to PBMs’ continued reliance on older FTC advocacy materials that opposed mandatory PBM transparency and disclosure requirements.

It also warns against reliance on the Commission’s prior conclusions, particularly given the FTC’s ongoing study of the PBM industry to update its understanding of the industry and its practices.

CLICK HERE to access the full article


FDA Approves Sub-q Tx for gMG – Rystiggo

The FDA recently approved a new therapy, Rystiggo (rozanolixizumab-Noli) from UCB, for the Treatment of adults with Generalized Myasthenia Gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Myasthenia gravis (MG) comes in two main types, generalized and ocular. Generalized MG is the most common type, accounting for 85 percent of people with MG. Generalized MG is generally more serious than ocular MG since it causes weakness in many muscle groups. Common gMG symptoms include difficulty making facial expressions. problems chewing and difficulty swallowing. slurred speech. weak arms, legs, or neck.

Administration of Rystiggo is by subcutaneous infusion. However, prescribing information specifies that it should only be prepared and infused by a healthcare provider.  The most common adverse reactions were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea.

Standard administration schedule is once weekly for 6 weeks, At $6050 / vial,  baseline therapy is a minimum of $36,000. 

Since Rystiggo is administered in clinics it will likely be a specialty distribution product.

CLICK HERE to access prescribing information


UCB Announces U.S. FDA Approval of RYSTIGGO (rozanolixizumab-Noli) for the Treatment of Adults With Generalized Myasthenia Gravis

FDA approval of RYSTIGGO (rozanolixizumab-noli) has been granted under the Priority Review designation for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive1

U.S. FDA approval is based on the pivotal Phase 3 MycarinG study in gMG2, a large phase 3 study which demonstrated treatment with rozanolixizumab-noli resulted in statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.

Additional treatment option provides opportunity for U.S. clinicians to tailor therapeutic approach based on individual patient needs

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