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Specialty pharmacies should be well into forecasting for the upcoming year. Cash in fuels cash out….. and you know that more of the former is critical for growth. So, good forecasting provides a roadmap for marketing, sales, purchasing, HR, etc., to ramp up for the new year — or, heaven forbid, plan to trim expenses. Remember, Finance takes an approved forecast and translates it into a company budget.

All good forecasts rely on published reports from pharmacy market analytic resources. Successful forecasts aggregate as much data as possible from multiple sources. So, before you submit your 2022 forecast check out the 2020 Specialty Trend Report from Pharmaceutical Strategies Group (PSG). 

CLICK HERE to download the PSG Report

By now you should know the highlights. 2020 was a sour year for the economy and its impact to specialty pharmacy should not be overlooked. But, there was some palpable market rebound in 2021…. but we won’t see that data till about April 2022. So, the trends through the downturn are harbingers for the upturn. And, the 2-5-year estimates in the report are credible metrics to moderate your optimism (or pessimism) for 2022. 

The press release below raised eyebrows. At first, we thought that Omnicell had acquired ReCept Specialty Pharmacy. ReCept is a four decades long running specialty provider and has grown to eleven locations mostly in the southern states. But our quick scan of the release proved first impressions wrong.

ReCept has sold off only the assets of a relatively small division, one that provides specialty pharmacy management services for health systems, clinics, and physician groups. Services include operational expertise, HR, technology / integration, workflow management, payer access assistance, and other aspects of managing a specialty pharmacy.

What is noteworthy is that these services are being offered to help health system owned (HOSP) pharmacies compete with independent specialty pharmacies. Is this a case of ‘If ya can’t beat em’… join em’? ReCept would not be the first SP to make this tack. Walgreens, for example, has been active in the HOSP space for several years.

Perhaps the most eye-popping aspect of the announcement was that this division only generated $24 million in the prior year period….. but was acquired by Omnicell for $100 million. That’s not 5X EBIDTA…. It’s 5X Revenue. Well played ReCept….. well played!

ReCept will continue to operate its specialty pharmacies and retail locations. 
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Omnicell to Acquire ReCept

Strategic acquisition of leading specialty pharmacy management services provider supports the Autonomous Pharmacy vision and expands Omnicell’s Advanced Services portfolio

Addresses significant need for access to and management of complex medications, expected to improve profitability and patient care

December 02, 2021  — Mountain View, Calif.–(BUSINESS WIRE)–Omnicell, Inc., a leading provider of medication management solutions and adherence tools for health systems and pharmacies, today announced it has entered into a definitive agreement with ReCept Holdings, Inc., a leading provider of specialty pharmacy management services for health systems, clinics, and physician groups, to acquire its business for total aggregate cash consideration of $100 million, subject to customary adjustments. The acquisition will broaden Omnicell’s portfolio of capabilities and services to address the significant need to improve access to and care coordination for complex medications.

“As medical innovations drive new, more complex drugs, pharmacy requires more specialized tools and expertise to help manage the patient’s treatment journey”

Studies have shown that specialty medications represent over 50 percent of the country’s total spending on retail, mail-order, and provider-administered drugs.1 Used for treatment of complex conditions, these medications often require intensive patient management and specialized workflows for dispensing and care coordination. Specialty pharmacies serve as the connection between patients, prescribing physicians, and payors to ensure streamlined access and adherence to these specialty drugs, helping to maintain continuity of care throughout the process, and are expected to improve margin and profitability. As an Advanced Services organization, ReCept provides implementation and managed services for health systems and other provider organizations to optimize their specialty pharmacy programs and the related pharmaceutical aspects of patient care.

The ReCept acquisition will add solutions for specialty pharmacy management services, including specialty pharmacy expertise and operational capabilities, human resources, technology and integration, workflow management, payor access assistance, and other aspects of managing a specialty pharmacy, to Omnicell’s broader medication management infrastructure. This total solution for provider groups, federally qualified health centers (FQHCs), and health systems supports on-site management of specialty pharmacy services, including payor contracting, staffing, licensing, quality assurance, 340B administration, and preferred pricing agreements designed to improve margin and profitability, while keeping the patient at the center of care.

“As medical innovations drive new, more complex drugs, pharmacy requires more specialized tools and expertise to help manage the patient’s treatment journey,” said Randall Lipps, chairman, president, CEO, and founder of Omnicell. “The ReCept acquisition is a strategic investment that expands our portfolio to address the significant market need for specialty pharmacy solutions. Our entry into this rapidly expanding area of practice is a natural progression on the path to the Autonomous Pharmacy and the goal to enhance care, improve efficiency, and drive profitability. We are excited to add these critical solutions to the Omnicell portfolio for our health system clients and prospects.”

“Specialty pharmacy is a key component of medication management. By joining with Omnicell we look forward to maximizing the benefits of our managed services capabilities as part of a broader, intelligent infrastructure that will deliver value for our pharmacy partners,” said Carmine DeNardo, president and chief executive officer, ReCept. “The synergies with Omnicell’s advanced pharmacy technology solutions create a comprehensive strategic partner that will help give health systems broader and deeper expertise across the full spectrum of medication management.”

Compelling Strategic BenefitsDifferentiated Portfolio of Solutions: ReCept provides a full suite of turnkey specialty pharmacy management services, including specialty pharmacy expertise and operational capabilities, technology and integration solutions, assistance in increasing access to payor networks and limited distribution drugs, and performance analytics and clinical outcomes assessment tools. These capabilities are complementary to Omnicell 340B, EnlivenHealth™, and IV Compounding Services, and can also support health systems moving to a consolidated service center approach to pharmacy operations.

Extends the Autonomous Pharmacy Vision: The ReCept acquisition furthers Omnicell’s commitment to deliver the hardware, software, and services to support the Autonomous Pharmacy vision, while advancing the evolution of the pharmacy care delivery model.

Complementary to Existing Solutions: The combination of ReCept’s specialty pharmacy expertise and operational capabilities and Omnicell’s commercial capabilities and client relationships, as well as its 340B program administration solutions and EnlivenHealth’s patient and member management tools, is expected to provide meaningful differentiation in the large and growing specialty pharmacy managed services organizations.

Transaction HighlightsUnder the terms of the agreement, Omnicell will pay total aggregate cash consideration of $100 million, subject to customary adjustments, at closing as provided for in the agreement and plan of merger. The transaction, which is expected to close by the end of the year, is subject to Hart-Scott-Rodino clearance and the satisfaction of other customary closing conditions. The ReCept business that is being acquired recorded approximately $24 million of total revenue (unaudited) for the 12 months ended September 30, 2021.

Omnicell will use cash available on its balance sheet to fund the transaction. Upon closing, the transaction is expected to be accretive to Omnicell’s non-GAAP EBITDA beginning in the first quarter of 2023.

About ReCeptReCept is the national leader in pharmacy and specialty pharmacy management solutions for health systems, federally qualified health centers, Ryan White HIV/AIDS programs and large physician provider groups. With over 40 years of experience in retail and specialty pharmacy management, ReCept has developed a proprietary and turnkey implementation and management platform, leveraging industry best practices to help pharmacies achieve their full potential. ReCept leads its customers through the entire pharmacy lifecycle, from store buildout to licensing to supporting ongoing operations to ensure future success. As a respected partner in the industry focused on quality patient care since 1978, ReCept helps its customers tackle the barriers associated with specialty medications, increasing patient access, improving patient adherence, and driving better outcomes for patients and prescribers. ReCept is a portfolio company of Generation Partners. To learn more about ReCept, visit: https://receptrx.com.

About OmnicellSince 1992, Omnicell has been committed to transforming the pharmacy care delivery model to dramatically improve outcomes and lower costs. Through the vision of the autonomous pharmacy, a combination of automation, intelligence, and technology-enabled services, powered by a cloud data platform, Omnicell supports more efficient ways to manage medications across all care settings. Over 7,000 facilities worldwide use Omnicell automation and analytics solutions to help increase operational efficiency, reduce medication errors, deliver actionable intelligence, and improve patient safety. More than 60,000 institutional and retail pharmacies across North America and the United Kingdom leverage Omnicell’s innovative medication adherence and population health solutions to improve patient engagement and adherence to prescriptions, helping to reduce costly hospital readmissions. To learn more, visit www.omnicell.com.

Just before Thanksgiving the FDA approved a new ORAL therapy, Livtencity (maribavir) from Takeda Pharmaceuticals, with an indication for the treatment of adults and pediatric patients (12 years of age and older and weighing at least 35 kg) with post-transplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet.

Post-transplant cytomegalovirus (CMV) is a rare disease and is one of the most common infections experienced by transplant recipients. The incidence rate varies by specific diagnosis with 16-56 % in Solid Organ Transplant (SOT) recipients and 30-70 % in Hematopoietic Stem Cell Transplant (HSCT) patients.

Takeda has not yet released the cost of Livtencity.

Takeda subsequently announced that the therapy will be available only through a select network of specialty pharmacies and distributors.

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FDA Approves First Treatment for Common Type of Post-Transplant Infection that is Resistant to Other Drugs

November 23, 2021 — Today, the U.S. Food and Drug Administration approved Livtencity (maribavir) as the first drug for treating adults and pediatric patients (12 years of age and older and weighing at least 35 kilograms) with post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral treatment for CMV. Livtencity works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication.

“Transplant recipients are at a much greater risk for complications and death when faced with a cytomegalovirus infection,” said John Farley, M.D., M.P.H., director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research. “Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern. Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.”

CMV is a type of herpes virus that commonly causes infection in patients after a stem cell or organ transplant. CMV infection can lead to CMV disease and have a major negative impact on transplant recipients, including loss of the transplanted organ and death.

Livtencity’s safety and efficacy were evaluated in a Phase 3, multicenter, open-label, active-controlled trial that compared Livtencity with a treatment assigned by a researcher running the study, which could include one or two of the following antivirals used to treat CMV: ganciclovir, valganciclovir, foscarnet or cidofovir. In the study, 352 transplant recipients with CMV infections who did not respond (with or without resistance) to treatment randomly received Livtencity or treatment assigned by a researcher for up to eight weeks.

The study compared the two groups’ plasma CMV DNA concentration levels at the end of the study’s eighth week, with efficacy defined as having a level below what is measurable. Of the 235 patients who received Livtencity, 56% had levels of CMV DNA below what was measurable versus 24% of the 117 patients who received an investigator-assigned treatment.

The most common side effects of Livtencity include taste disturbance, nausea, diarrhea, vomiting and fatigue. Livtencity may reduce the antiviral activity of ganciclovir and valganciclovir, so coadministration with these drugs is not recommended. Virologic failure due to resistance can occur during and after treatment with Livtencity, therefore CMV DNA levels should be monitored and Livtencity resistance should be checked if the patient is not responding to treatment or relapses.

Livtencity received Breakthrough Therapy and Priority Review designations for this indication. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.

The FDA granted the approval of Livtencity to Takeda Pharmaceuticals Company Limited.

Last week the FDA approved a new infused therapy, Fyarro (sirolimus protein-bound particles for injectable suspension / albumin-bound) from Aadi Bioscience, for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

Fyarro is the first and only approved therapy for adults with this condition, an ultra-rare and aggressive form of sarcoma with a strong female predominance. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract.

It is estimated that there are only about 100-300 new patients per year in the United States.

Fyarro’s wholesale acquisition cost was announced at $6,785.00 for a single-use 100 milligram vial. Utilization is dependent on the patient’s body mass and any dosing modification resulting in a cost of approximately $468,000 per year. Aadi said that “Discounts, such as Medicaid rebates, sales to 340B covered entities and VA facilities among others, will reduce the net price by approximately 15% to 20%.”

Given the small patient population in the US it is expected that Fyarro will be launched through a specialty pharmacy distribution (direct to physician) program.

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Aadi Bioscience Announces FDA Approval of its First Product FYARRO for Patients with Locally Advanced Unresectable or Metastatic Malignant Perivascular Epithelioid Cell Tumor (PEComa)

LOS ANGELES, Nov. 23, 2021 — Aadi Bioscience, Inc., a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that the U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is the first and only FDA-approved treatment for advanced malignant PEComa in adults.

Neil Desai, Ph.D., Founder, Chief Executive Officer and President of Aadi, stated, “We are thrilled to have received full FDA-approval of FYARRO. The approval of FYARRO is a momentous event not just for Aadi but, importantly for advanced malignant PEComa patients. We reiterate that all of us at Aadi are incredibly grateful to all of the people with advanced malignant PEComa, their families and caregivers, as well as the healthcare professionals who made the FYARRO clinical studies possible.”

“The approval of FYARRO, the first approved drug for advanced malignant PEComa, an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease,” added Andrew Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute and the Principal Investigator in the pivotal AMPECT registrational trial. “In our AMPECT trial, FYARRO demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, with an acceptable and manageable safety profile. This is a drug that will be welcomed by the physician community as the only approved therapeutic option for patients with advanced malignant PEComa.”

In the Phase 2 registrational AMPECT trial the overall response rate as assessed by independent review was 39% (12/31) with 2 patients achieving a Complete Response after prolonged follow up. The median duration of response has not been reached with a median follow-up of 36 months, and a range of 5.6 to 55.5+ months and ongoing. Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to 2 years. As is the case with other therapeutics of the mTOR class, the FYARRO prescribing information includes warnings and precautions related to stomatitis, myelosuppression, infections, hypokalemia, hyperglycemia, interstitial lung disease, hemorrhage, and hypersensitivity reactions. Grade 3 non-hematologic events occurring in more than 10% of patients included stomatitis, rash, fatigue and infections. Grade 3 laboratory abnormalities occurring in more than 10% of patients that worsened from baseline included lymphocytopenia, increased glucose, and decreased potassium. For detailed important safety information, please see below.

Brendan Delaney, Chief Operating Officer of Aadi, added, “We have built a strong commercial team and devised a thoughtful strategy in preparation for FYARRO’s launch. With FYARRO’s demonstrated clinical profile we believe it will become a standard of care for advanced malignant PEComa. We look forward to engaging with physicians to educate the market about this new treatment.”

About Malignant PEComa
Advanced malignant PEComa, defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,’ are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. While there is no formal epidemiology for malignant PEComa, with a female predominance) and can have an aggressive clinical course including distant metastases and ultimately death. The estimated prognosis based on retrospective reports is 12-16 months. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.

I’ve editorialized about the encroachment of health system owned specialty pharmacies (HOSPs) into the independent SP space since 2013. Each prescription that is directed ‘in-system’ is a prescription that doesn’t go to an independent…. or even a big box SP.

Consider the article below. One key line reads….. “there hasn’t been one organization that represents all the interests of the industry in one place.”

It is curious that the HOSP Alliance feels that the HOSP segment voice is not now being adequately represented. You may, however, get a different view by visiting the NASP web site. As most of us know, NASP is one organization that has represented all the interests of the SP industry for years. But, the fastest growing NASP segment (based on a quick count of corporate sponsors on the NASP site) appears to be, you may have guessed it, health system owned specialty pharmacies. Upwards of 30 health systems are current NASP sponsors, including Shields/ExceleraRx and TrellisRx (companies that have led the charge to form HOSPs). That’s more than 20% of all NASP sponsors!

So, ask yourself….. “Why does the HOSP Alliance feels that it now needs its own ‘industry’ voice?”

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How a health system alliance is advocating for hospital-owned specialty pharmacies

In October 2020, seven health systems united to form the Health System Owned Specialty Pharmacy Alliance, or HOSP, to advocate for health system-owned specialty pharmacies.

The group has grown significantly since its launch, with nearly 30 health systems now participating. Here, Gary Kerr, PharmD, the president of HOSP’s board of directors and the chief pharmacy officer at Springfield, Mass.-based Baystate Health, answers questions about the group’s work and plans.

Editor’s note: Responses have been lightly edited for clarity and style.

Q: Why do health system-owned specialty pharmacies need their own voice?
Dr. Gary Kerr: HOSP acts as the “face and voice” of the integrated specialty pharmacy industry, advocating for and uniting members around common industry interests and concerns. The industry needs its own voice because while there are several organizations that represent many general interests impacting health system specialty pharmacy, there hasn’t been one organization that represents all the interests of the industry in one place. Our goal is to help bring the industry together, so health system-owned specialty pharmacies have a seat at the table and ensure that their interests are represented. That said, we recognize there is some great work underway by other organizations, and we don’t intend to duplicate efforts. Rather, we are supportive of that work and intend to focus on the areas where there are gaps.

Specialty pharmacies focus on delivering medications for more complex and chronic illnesses. More than 1 in 4 specialty pharmacies are now owned by health systems, a trend that is continuing to grow as hospitals look for ways to improve patient care. The integrated specialty pharmacy model helps keep a critical piece of patient care — medication management and treatment — inside the health system, instead of outsourcing to a more fragmented approach to care. From a physical plant perspective alone many of these system-owned specialty pharmacies operate on campus along with the clinicians’ offices, share the electronic medical record with the prescribers and control all aspects of the land-based courier services.

Q: How many health systems are involved in HOSP?
GK: We had a great first year, and membership has increased significantly year over year. We currently have nearly 30 health systems participating, and we are growing fast. We also unveiled an active campaign this fall, now underway, to identify and embrace new partnerships beyond the core of the health system specialty pharmacy members. The HOSP Partnership Program offers a unique way for like-minded organizations to collaborate with and support our industry.

Q: What does HOSP’s collaboration with health systems look like?
GK: We don’t collaborate with health systems, but rather our membership is composed of health systems who are generally represented by their system’s integrated specialty pharmacy leadership. Members actively and thoughtfully collaborate to share emerging best practices, discuss innovative work processes and patient care solutions and develop and advance a unified voice on a variety of policy issues. Members are also focused on full engagement on how to demonstrate and share the value of the integrated specialty pharmacy model, by shaping the metrics data aspect of the care model.

Q: What have been HOSP’s biggest accomplishments since its launch?
GK: HOSP has done a lot in one year. We’ve established a very strong board of directors and have been steadily increasing our membership base. We have four dedicated hardworking committees. One is the innovation committee; whose goal is to increase awareness and influence actions to improve quality of care and patient outcomes with improved patient management software integration in health systems-owned specialty pharmacies. They have dug deeply into the patient journey to find where there are gaps that technologies and other solutions could help address. We’ll be highlighting them more in the near future.

Another committee you’ll be hearing about is our Health Economics and Research Outcomes committee, which owns HOSP’s work to develop and collaborate on the most valuable data (metrics) that helps promote the value of the integrated, specialty pharmacy care model. Our advocacy committee just sent a letter introducing HOSP and outlining our top priorities to HHS Secretary Xavier Becerra and top congressional officials.

We’ve also made some great inroads in beginning some new initiatives that we’ll be advancing in 2022, including our HOSP best practice groups, which bring leaders across membership together to share best practices on a variety of issues.

Q: What initiatives does HOSP have planned for the future?
GK: In addition to growing our membership and keeping our committees focused on the great work we are producing. We’re planning an in-person member event in early 2022. Since we launched during the COVID-19 pandemic, we have yet to get together in person, so we’re very excited to finally get together in the same space.

Last week the FDA approved a new subcutaneous therapy, Voxzogo (vosoritide) from BioMarin, to improve growth in children five years of age and older with achondroplasia, the most common form of dwarfism, as well as open epiphyses (growth plates).

Achondroplasia is the most common type of short-limbed dwarfism, with a global incidence of 1 in 15,000 to 40,000 newborns. Most cases are not inherited, but rather result from new mutations (80%) in the FGFR3 gene causing abnormal formation of cartilage and bone and ultimately shorter bones; however, if both parents have achondroplasia, then there is a 50% chance that their children will be affected.

Voxzogo is an analog of C-type natriuretic peptide (CNP), a positive regulator of bone growth. Vosoritide also inhibits fibroblast growth factor receptor 3 (FGFR3), which has a negative effect on bone growth. Vosoritide has a longer half-life than its endogenous form. Across the trial studies, vosoritide was generally well tolerated, with transient injection site reactions and hypotension as the most common TEAEs.

BioMarin announced that the list price per year will run $320,000 per year. After rebates and discounts, Voxzogo will net about $240,000 per patient per year. Given the low incidence of the indicated conditions, it is expected that the therapy will launch in limited distribution.

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FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism

SILVER SPRING, Md., Nov. 19, 2021 /PRNewswire/ — Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow. Achondroplasia is the most common form of dwarfism.

“Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States and underscores the FDA’s commitment to help make new therapies available for rare diseases,” said Theresa Kehoe, M.D., director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research. “With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.”

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. Voxzogo works by binding to a specific receptor called natriuretic peptide receptor-B that reduces the growth regulation gene’s activity and stimulates bone growth.

Voxzogo’s safety and efficacy in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase 3 study in participants five years and older with achondroplasia who have open epiphyses. In the study, 121 participants were randomly assigned to receive either Voxzogo injections under the skin or a placebo. Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo.

The most common side effects of Voxzogo include injection site reactions, vomiting and decreased blood pressure. Voxzogo’s labeling also lists decreased blood pressure as a warning and precaution, which means it is a potentially serious side effect.

The FDA approved Voxzogo under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need, based on a surrogate or intermediate clinical endpoint. A condition of this accelerated approval is a post-marketing study that will assess final adult height. This application also received priority review designation.

The FDA granted the approval of Voxzogo to BioMarin.

Specialty pharmacies do a fair amount of business with Medicaid. In many states the Medicaid program allows any willing provider access, sometimes across state lines. The downside is that reimbursement margins may be very thin for the pharmacy. None the less, one element of sustaining any business is cash flow. Even with thin margins, Medicaid sales can be a reliable cash flow generator. As such, keeping an eye on trends in the Medicaid universe is a prudent business practice.

Magellan Rx recently published a nifty analysis for your reading pleasure. It details trends over the past few years. Knowing where the trends are heading can help with allocating resources to build new referral relationships, tweak operational / service planning, and more. The report also includes a bunch of useful charts and graphs.

Key 2020 findings include:

• In 2020, specialty drugs accounted for 51.4% of the net cost in Medicaid
• Specialty drugs only accounted for 1.3% of utilization.
• Traditional net cost trend for Medicaid FFS was positive for the first time in five years driven by new-to-market drugs.
• Medicaid FFS top drug classes remained almost identical to previous years
• HIV/AIDS and antipsychotics accounting for more than 19.8% of the total net drug spend.
• Most key conditions will experience increased trend over the next three years. 
• Conditions with generic drug introductions or specialized management strategies will trend lower.
• The 2020 virus impacted patient continuity of care triggering technology-based solutions (e.g., video conferencing, shift to home care, and more).

CLICK HERE to access the full Magellan report.
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Long Predicted Medicaid Specialty Drug Trend Finally Surpasses 50% of Pharmacy Spend in the Latest Magellan Rx Management Medicaid Pharmacy Trend Report

November 15, 2021  — PHOENIX–(BUSINESS WIRE)– Magellan Rx Management, the full-service pharmacy benefits management division of Magellan Health, Inc. (NASDAQ: MGLN), released its sixth annual Medicaid Pharmacy Trend ReportTM, the industry’s leading report exclusively detailing trends in the Medicaid pharmacy fee-for-service (FFS) space.

The Medicaid Pharmacy Trend Report highlights the evolving landscape of Medicaid, made even more dynamic from the events of the last two years. The report includes an in-depth analysis of class and drug trends, forecasting of Medicaid key conditions, drugs in the pipeline, and Medicaid pharmacy economics. This is also the only detailed industry source for the analysis of Medicaid pharmacy fee-for-service (FFS) gross, net, and forecasted drug cost trends.

“As our nation continues to navigate the lasting impacts of the COVID-19 pandemic, there is no doubt that states are facing unprecedented challenges across all areas of government, especially related to ensuring the mental and physical well-being of their citizens,” said Meredith Delk, PhD, MSW, general manager and senior vice president, government markets, Magellan Rx Management. “The Medicaid Trend Report illustrates critical data-driven observations and solutions. The valuable insights in this report can assist states on their mission to ensure a high quality and cost-effective prescription drug program for their most vulnerable populations.”

“Our ability to offer comprehensive and configurable solutions that fundamentally connect the dots for our customers and their members around the efficacy of drugs, quality care and payments is key,” said Delk.

The Magellan Rx Management Medicaid Pharmacy Trend Report includes data from Magellan Rx’s Medicaid FFS pharmacy programs in 25 states and the District of Columbia. The material is reviewed and supported by a team of Magellan Rx experts with broad national expertise, including 369 years of combined pharmacy benefit administration (PBA) experience.

Last week the FDA approved a new therapy, BESREMi (ropeginterferon alfa-2b-njft) from PharmaEssentia, for the treatment of adults with polycythemia vera (PV). The drug was granted an orphan designation. BESREMi is administered subcutaneously.

Polycythemia Vera (PV) is a cancer that originates from a disease-initiating stem cell in the bone marrow leading to a chronic increase of red blood cells, white blood cells, and platelets. The cancer then frequently results in cardiovascular complications as well as secondary myelofibrosis or leukemia.

BESREMi is a long-acting interferon and is administered once every two weeks until hematologic targets are realized. Patients with a complete hematologic response can then be treated every four weeks.

Analysts expect the annual cost per patient per year (PPPY) to be in the $40,000 range. Details related to channel access were not released. As an orphan drug, BESREMi will very likely be released through limited distribution.

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U.S. FDA Approves BESREMi (ropeginterferon alfa-2b-njft) as the Only Interferon for Adults With Polycythemia Vera

November 12, 2021 — BURLINGTON, Mass.–(BUSINESS WIRE)–PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi for the treatment of adults with polycythemia vera (PV).

“With the availability of an FDA-approved, next-generation interferon for this indication, it’s time that we focus on preserving the long-term health of patients with polycythemia vera.”

BESREMi is an innovative monopegylated, long-acting interferon, which exhibits its cellular effects in polycythemia vera in the bone marrow. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infections disorders. PharmaEssentia is preparing to make BESREMi available in the coming weeks in the U.S.

“The FDA approval of BESREMi for people with polycythemia vera represents the next step in advancing patient care as it provides a critical addition to managing not only symptom burden and near-term complications, but also treating the cancer early, which may help reduce the risk of disease progression over time,” said Srdan Verstovsek, M.D., Ph.D., Director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center. “With the availability of an FDA-approved, next-generation interferon for this indication, it’s time that we focus on preserving the long-term health of patients with polycythemia vera.”

“The reality of living with a rare and chronic cancer like polycythemia vera is that it is often under recognized and the limited treatments available cannot properly address the disease beyond the symptoms. Our community welcomes the FDA approval of a new treatment that has the potential to deliver what has been unavailable for so many patients hoping for a better outlook,” said Ann Brazeau, CEO of MPN Advocacy and Education International.

The U.S. FDA approval was based on safety from the PEGINVERA and PROUD/ CONTINUATION-PV studies and efficacy data from the PEGINVERA clinical study program. The study showed that after 7.5 years of treatment with BESREMi, 61% of patients with PV experienced a complete hematological response (defined as hematocrit <45% without phlebotomy for at least 2 months since last phlebotomy, platelets ≤ 400 x 109/L, leukocytes ≤10 x 109/L, normal spleen size (longitudinal diameter ≤12 cm for females and ≤ 13 cm for males). Importantly, 80% of patients achieved a hematological response (based on objective laboratory parameters only, with the exclusion of normal spleen size and thrombosis). These parameters are the most commonly used metrics to make therapeutic decisions.1 In the pooled safety population of patients treated with BESREMi, the most common adverse reactions (incidence >40%) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. Serious adverse reactions (incidence > 4%) were urinary tract infection, transient ischemic attack and depression.1

“We are incredibly proud to deliver on our goal of bringing treatments like BESREMi to the polycythemia vera community where there is clear unmet need for more effective, tolerable and durable treatments to preserve patients’ health and well-being,” said Ko-Chung Lin, Ph.D., Co-Founder and Chief Executive Officer for PharmaEssentia and inventor of ropeginterferon alfa-2b-njft. “As we begin working closely with the community to integrate this important treatment into clinical practice, we also continue to expand our scientific efforts to unlock the full potential of our pioneering molecule.”

The FDA just approved a new ORAL therapy, Scemblix (asciminib) from Novartis, for patients with Philadelphia chromosome-positive chronic myeloid leukemia previously treated with two or more tyrosine kinase inhibitors (TKIs). Studies show that around 55% of CML patients are intolerant to TKIs and 70% receiving TKIs as a second-line treatment fail to achieve a significant response. Patients who acquire T314I mutations also tend to be resistant to existing TKI therapies. The drug also received full approval for adult patients with Ph+ CML in CP with the T315I mutation.

Chronic myeloid leukemia (CML) is the most common myeloproliferative disorder accounting for ~20% of all leukemia cases. Its annual incidence has been estimated at between 1 and 1.5 cases per 100,000 and its prevalence at around 1 in 17,000. The Philadelphia chromosome is seen in more than 90% of patients with CML.

Pricing for Scemblix was not released. Channel access was not announced. However, given the small patient base for its indication it would be expected that Scemblix will launch via limited distribution.

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FDA approves Scemblix (asciminib) for Philadelphia chromosome-positive chronic myeloid leukemia

On October 29, 2021, the Food and Drug Administration granted accelerated approval to asciminib (Scemblix, Novartis AG) for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), and approved asciminib for adult patients with Ph+ CML in CP with the T315I mutation.

ASCEMBL (NCT03106779), a multi-center, randomized, active-controlled, open-label clinical trial, is evaluating asciminib in patients with Ph+ CML in CP, previously treated with two or more TKIs. A total of 233 patients were randomized (2:1) and stratified according to major cytogenetic response (MCyR) status to receive either asciminib 40 mg twice daily or bosutinib 500 mg once daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was major molecular response (MMR) at 24 weeks. The MMR rate was 25% (95% CI: 19, 33) in patients treated with asciminib compared with 13% (95% CI: 6.5, 23; p=0.029) in those receiving bosutinib. With a median duration of follow-up of 20 months, the median duration of MMR has not yet been reached.

CABL001X2101 (NCT02081378), a multi-center, open-label clinical trial, is evaluating asciminib in patients with Ph+ CML in CP with the T315I mutation. Efficacy was based on 45 patients with the T315I mutation who received asciminib 200 mg twice daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was MMR. MMR was achieved by 24 weeks in 42% (19/45, 95% CI: 28% to 58%) of the patients. MMR was achieved by 96 weeks in 49% (22/45, 95% CI: 34% to 64%) of the patients. The median duration of treatment was 108 weeks (range, 2 to 215 weeks).

The most common adverse reactions (≥20%) are upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. The most common laboratory abnormalities are decreased platelet counts, increased triglycerides, decreased neutrophil counts and hemoglobin, and increased creatine kinase, alanine aminotransferase, lipase, and amylase.

The recommended asciminib dose in patients with Ph+ CML in CP, previously treated with two or more TKIs, is 80 mg taken orally once daily at approximately the same time each day or 40 mg twice daily at approximately 12-hour intervals. The recommended asciminib dose in patients with Ph+ CML in CP with the T315I mutation is 200 mg taken orally twice daily at approximately 12-hour intervals.

This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 4 months ahead of the FDA goal date.

This application was granted priority review, breakthrough designations, fast track designation, and orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

We missed one…….

At the end of August the FDA approved a new therapy, Skytrofa from Ascendis Pharma (lonapegsomatropin-tcgd), indicated for pediatric patients that have short stature due to inadequate secretion of endogenous growth hormone. Skytrofa is the first weekly subcutaneous injectable growth hormone for children with growth hormone deficiency (GHD).

According to the American Academy of Pediatrics, growth hormone deficiency among humans is a rare condition and affects less than one in 3,000. In 2020 there were 74 million kids under the age of 18 in the US. So, we can estimate that there are as many as 25,000 children eligible for hGH.

Skytrofa joins a growing number (pun intended) of competing growth hormone products.
– Accretropin
– Humatrope
– Norditropin
– Norditropin
– Nutropin
– Nutropin AQ
– Nutropin Depot
– Omnitrope
– Saizen
– Serostim
– Skytrofa
– Tev-Tropin
– Zomacton
– Zorbtive

Analysts indicate that the hGH market globally surged in 2019-2020 and is expected to realize a robust 5% compound annual growth rate for the foreseeable future.

Skytrofa is dosed based on weight. It is supplied in nine quantities from 3mg through 13.3mg in prefilled cartridges. Most noteworthy is that this therapy is also supplied with an electric auto-injector (the instructions for use resemble something from IKEA). Each cartridge includes a chamber for the dry powder and a sterile water chamber for mixing with the powder….. inside the electric injector. Safety instructions state not to use near a microwave oven…. now that is something you don’t see in prescribing information every day. The electric injector is replaced after every six injections.

Skytrofa is supplied in a 4-dose carton for once weekly administration. A carton of Skytrofa with a GoodRx discount costs $2,500 – $2,700 depending on pharmacy. Finally a new therapy that is not in limited distribution.

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FDA Approves Weekly Therapy for Pediatric Patients with Growth Hormone Deficiency

FDA has approved Skytrofa (lonapegsomatropin-tcgd) injection to treat pediatric patients age one year and older who weigh at least 11.5 kg (25.4 pounds) and have short stature due to inadequate secretion of endogenous growth hormone. Skytrofa is a human growth hormone (hGH) approved for pediatric patients to take by under-the-skin injection weekly. Other available FDA-approved hGH formulations for pediatric patients with growth hormone deficiency must be administered daily.

Growth hormone deficiency is a disorder characterized by inadequate growth hormone production from the anterior pituitary gland, a small gland located at the base of the brain that produces several hormones. Children with this disorder can receive growth hormone to stimulate growth.

Skytrofa was studied in a 52-week trial that enrolled 161 pre-pubescent research participants (average age was 8.5 years) with growth hormone deficiency who had received no previous growth treatment. Participants were randomly assigned to receive either weekly injection of Skytrofa or daily injection of somatropin, an FDA-approved human growth hormone. Efficacy was determined by the increase in growth rate after 52 weeks of treatment. Weekly Skytrofa treatment for 52 weeks resulted in a growth rate increase of 11.2 cm (4.4 inches) per year and was not inferior to daily somatropin treatment.

The most common side effects of Skytrofa include viral infection, pyrexia (fever), cough, nausea and vomiting, hemorrhage (blood loss), abdominal (stomach) pain, arthralgia (joint pain), arthritis, and diarrhea. Patients with acute critical illness, allergies to somatropin or any Skytrofa excipients, active malignancy, certain types of diabetic retinopathy (eye-related diabetes complications), and Prader-Willi syndrome with other risk factors should not take Skytrofa. Children with closed epiphyses (end part of a long bone) should also not take the drug.

Patients taking Skytrofa are also at increased risk of neoplasms (abnormal tissue growth), intracranial hypertension (buildup of pressure around the brain), fluid retention, hypoadrenalism (underactive adrenal gland), hypothyroidism (underactive thyroid), slipped capital femoral epiphysis (a pediatric hip condition), progression of preexisting scoliosis, and pancreatitis (inflammation of the pancreas).

Skytrofa may interact with glucocorticoid (a type of steroid) treatment, oral estrogen, insulin, and other antihyperglycemic (blood sugar-reducing) agents.