Category: Uncategorized

Now here’s something ya don’t see every day!
A drug approved within the last year being yanked from the market.

This week, the FDA pulled approval for Ukoniq directly related to safety concerns. Earlier this year, the FDA started its investigation when post launch clinical data showed possible increased risk of death from taking Ukoniq.

In February of 2021, Ukoniq, a PI3K / CK1 inhibitor, was granted accelerated approval for the treatment of marginal zone lymphoma (MZL) and follicular lymphoma (FL).

However, PI3K inhibitors started to display clinical concerns resulting in an FDA advisory committee recommendation that halted single-arm clinical studies of PI3K inhibitors to document safety concerns. Further, the FDA’s Oncologic Drug Advisory Committee recommended the use of randomized clinical trials. And the FDA ‘net’ is now expanding with reviews of P13K therapies in general.

One other aspect of this story should also be considered, that being the Accelerated Approval program itself. The number of drugs approved under accelerated approval has mushroomed in recent years….. that was laudable through helping drugs of value reach the marketplace sooner….. until something goes terribly wrong. The boosters quickly become nay-sayers….. especially when deaths are involved….. and the FDA itself is under the microscope for its efficiencies or lack thereof.

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from MedWatch

FDA Approval of Lymphoma Medicine Ukoniq (umbralisib) is Withdrawn Due to Safety Concerns

AUDIENCE: Oncology, Patient, Health Professional, Pharmacy

ISSUE: Due to safety concerns, the FDA has withdrawn its approval for the cancer medicine Ukoniq (umbralisib).

Updated findings from the UNITY-CLL clinical trial continued to show a possible increased risk of death in patients receiving Ukoniq. As a result, the FDA determined the risks of treatment with Ukoniq outweigh its benefits. Based upon this determination, the drug’s manufacturer, TG Therapeutics, announced it was voluntarily withdrawing Ukoniq from the market for the approved uses in marginal zone lymphoma and follicular lymphoma.

BACKGROUND: Ukoniq was approved to treat two specific types of lymphoma: marginal zone lymphoma and follicular lymphoma.

RECOMMENDATIONS: Health care professionals should stop prescribing Ukoniq and switch patients to alternative treatments. Inform patients currently taking Ukoniq of the increased risk of death seen in the clinical trial and advise them to stop taking the medicine. In limited circumstances in which a patient may be receiving benefit from Ukoniq, TG Therapeutics plans to make it available under expanded access.

Patients should talk to your health care professionals about alternative treatments and stop taking Ukoniq.

Health professionals and patients are encouraged to report adverse events or side effects related to the use of these products to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program.

Back in February we sent a report detailing an innovative network approach to alternate site / home infusion and it seems to be a topic that is gaining traction. Amerisource Bergen recently announced a broad home infusion solution for its many hospital clients. 

AB has pushed targeted programs in the past tailored to help clients launch and then manage targeted business segments. For example, they created a program to help Oncologists launch office-based dispensaries for oncolytic drugs….. with the goal of generating office revenue that would otherwise go to specialty pharmacies.

Historically, hospitals have never been keen on home infusion as it has been complicated, messy to manage, and low margin. That is changing with healthy margins for uber-costly specialty meds increasingly being administered in the home. It also enables the hospital affiliated home infusion division to flip a patient back to hospital outpatient if higher levels of care are required.

So, what’s this AB program all about? 

• Clinical expertise and pharmacy design
• Drug sourcing and logistics
• Payer strategy and prescription capture
• Patient access and workflow (inc’l software)
• Revenue cycle management

In short, AB has a soup-to-nuts solution for even the most squeamish hospital to dig into the home infusion muck.

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AmerisourceBergen Launches Home Infusion Solution for Health Systems NationwideAB’s offering works to optimize a health system’s site-of-care strategy and home infusion service line performance

April 28, 2022 — CONSHOHOCKEN, Pa.–(BUSINESS WIRE)–AmerisourceBergen (AB), a global healthcare company, today announces a program designed to help hospitals and health systems launch or optimize home infusion service lines. 

As site-of-care strategies grow in importance, health systems with strong outpatient infusion center performance are increasingly leveraging home infusion to prevent potential care gaps, particularly as therapeutic advancements continue to broaden the range of treatments available for the home. Each phase of AB’s solution is designed to help health systems deploy and operate a clinically focused, sustainable home infusion program for patients that choose to receive care within a health system network. 

While the infusions will be handled by the health systems’ clinical teams directly, AB’s solution serves to strengthen program strategy, business operations, logistics and more. AB has been a leader in specialty pharmaceutical services and distribution for more than 20 years, and health systems can now leverage this expertise as they look to create or refine a home infusion program for their patients. 

More information on AB’s solution can be found here: www.amerisourcebergen.com/provider-solutions/home-infusion.

The core components of AB’s solution are as follows:

“Perceived barriers to entry or prior negative experiences can often deter health systems from investing in home infusion programs, but AmerisourceBergen’s deep understanding of market dynamics, proven solutions, and leading expertise in specialty position us to address all areas needed for program success,” said Dan Teich, Vice President, Client Strategies at AmerisourceBergen. “We help our customers mitigate risk by ensuring that there is ample opportunity to warrant the investment, and that the program itself has all the components needed for successful and sustainable long-term performance.”

As a member of the National Home Infusion Association (NHIA) and their Future of Infusion Advisory Council, AB is committed to leadership and advocacy on behalf of home infusion providers across the United States.

The FDA this week issued final guidance related to the importation of drugs from Canada. Importation was passed late last year but many questions remained unresolved.  The document the FDA just released answered a lot of questions and specialty pharmacies would do well to read through the details to see if there is any benefit to even considering purchasing drugs from Canada.  

In short, a licensed pharmacist, pharmacy, or wholesale drug distributor may participate in programs to import any drug from Canada that could be sold legally there or in the American market, the FDA said. But….. there are a bunch of hoops required to be jumped through first. (surprised??)

One must apply to be an authorized purchaser as a pharmacy or a distributor. The process  doesn’t appear overly difficult, but there is red tape to be cut before one gets to place order #1. Oh, and the state that you are based in must have submitted a management plan and been approved by the FDA since the states’ Boards of Pharmacy will continue in their traditional oversight role.

Here is where it gets interesting for a specialty pharmacy.Take a look at what is excluded from the program——

• Controlled substances,
• biological products, 
• infused drugs (including peritoneal dialysis solutions), 
• drugs that are inhaled during surgery, 
• drugs that are injected intravenously (into a vein), intrathecally (into the spinal
• fluid), or intraocularly (into the eye), 
• drugs that are subject to a risk evaluation and mitigation strategy (REMS), and 
• drugs that are not subject to certain provisions of the Drug Supply Chain Security Act. 
• For a drug product not excluded, FDA will determine on a product-by-product basis. (We believe that aspirin is one of the few remaining drugs that qualify for import….. but don’t quote me!)

With all the exclusions, one must wonder what exactly is left worth going through the trouble to import. Some serious analysis will be required to make that determination.

Since this is a relatively new market phenomenon, one must also wonder how spinning off some purchasing to a Canadian wholesaler will sit with one’s primary US wholesaler. Hmmmm.

CLICK HERE to read the full Final Rule Guidance for Industry 

A significant number of specialty pharmacies have contracted as a 340b dispensing pharmacy often for multiple ‘program eligible’ hospitals. As most know, manufacturers have recently been on the offensive with these programs citing numerous instances of double dipping discounts, providing product to ineligible patients, inaccurate data reporting, and more. With the emergence of hospital owned and operated pharmacies, the challenge of tracking and tracing ‘eligible’ vs. ‘ineligible’ discounts has exacerbated.

As noted in the article below, the already complex process of managing 340B pharmacies has become even more challenging with 16 drug manufacturers now restricting discounts to contracted pharmacies, including some extending to federal grantees.

But the industry is not waiting silently for relief. The AHA, 340B Health, America’s Essential Hospitals, Association of American Medical Colleges, and Children’s Hospital Association recently filed an opinion with the U.S. Courts of Appeals to require drug companies to “fulfill their legal obligations to provide 340B discounted drugs to eligible hospitals and health systems, regardless of whether the drugs are dispensed on site or through contract pharmacies.” They also claim that more than half of 340B hospitals report they do not operate in-house retail pharmacies, and only one in five have their own specialty pharmacy, making contract pharmacies “a necessary and beneficial component of the 340B program.”

Specialty pharmacies working the 340b segment may garner useful insights on the topic by reviewing a survey recently released from the health center pharmacy perspective. It was conducted in the first quarter of 2022 and gathered responses from 75 key leaders, influencers, and decision-makers in health center 340B programs.

CLICK HERE to download the full report

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New Study Provides Insights into 340B Pharmacies Amid Market Upheaval

AMARILLO, Texas, May 18, 2022 /PRNewswire/ — The already complex process of managing 340B pharmacies has become even more challenging for Community Health Centers (CHCs) with 16 drug manufacturers restricting discounts to contracted pharmacies, including some extending to federal grantees, and a widening shortage of pharmacy staff. The federal 340B drug discount program allows CHCs, among other safety net providers, to realize savings on drug costs from mandated discounts from manufacturers. The 340B program has experienced seismic changes in the past two years as manufacturers increasingly restrict access to the discounts and clarity on the legality of the restrictions remains tied up in courts.

CHCs’ 340B pain points are highlighted in a new report released today by Maxor 340B, an industry leader in pharmacy management and comprehensive 340B solutions. The report, titled “The State of Community Health Center Pharmacies Today,” is based on an independent survey of CHC leaders that are actively involved in managing their organization’s 340B programs.

Healthcare consultancy Sage Growth Partners conducted the survey to look at pharmacy-related challenges, needs, preferences and plans of CHCs. The report reveals what CHCs want from their 340B pharmacy partners, as well as their preferred vendor characteristics.

“This survey confirms that managing pharmacies and 340B programs has become even more complex and challenging in the past few years, due to the impact of the pandemic, changing compliance requirements, and uncertainty over participation by drug manufacturers. CHCs may increasingly look to outside partners to help them manage this complexity,” said Mike Ellis, CEO of Maxor National Pharmacy Services. “CHC’s are seeking comprehensive solutions, good service, easy to use technology, transparent and innovative fee structures, compliance expertise, and excellent overall value.”

“Our research shows that those who can offer a transparent and responsive partnership to help CHC leaders simplify 340B programs will have the upper hand going forward,” said Dan D’Orazio, CEO, Sage Growth Partners.

Highlights from the survey include: ” [open link above to read the full article]

Earlier this month the FDA approved an ORAL specialty therapy, Cuvrior (trientine tetrahydrochloride) from Orphalan SA, for the treatment of adults with stable Wilson’s disease who are decoppered and tolerant to penicillamine. Wilson’s disease is a devastating disorder affecting patients worldwide. It is a rare genetic storage disease caused by a defect in a copper transporter gene, leading to copper accumulation in the liver, brain, eye, and peripheral nerves. It is the first drug approved for Wilson’s disease in the United States in more than 5 decades.

Approximately one in 90 people may be carriers of the disease gene. Current prevalence is estimated to be only about 2,000-3,000 cases in the United States. Other affected individuals have been misdiagnosed with other neurological, liver, or psychiatric disorders.

The big question is….. “Is this a new drug??”
In fact, trientine tetrahydrochloride was first approved by the FDA is 1985….. Yes…. 37 years ago!
As we are seeing, there has been an increasing number of ‘old’ therapies being reapproved due to ‘tweaks’ in formulations or reissuing in a different form (usually oral).

CLICK HRE to read prescribing information

Orphalan announced that pricing will be released near to product launch.
Orphalan expects to launch Cuvrior in the United States by early 2023.
Given the very small patient population in the US it is a sure bet that Cuvrior will launch through limited distribution.

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FDA Approves New Drug for Wilson’s Disease

May 03, 2022 — The US Food and Drug Administration (FDA) has approved oral trientine tetrahydrochloride (Cuvrior, Orphalan) for the treatment of adults with stable Wilson’s disease who are decoppered and tolerant to penicillamine, the company has announced.

Wilson’s disease is a rare genetic storage disease caused by a defect in a copper transporter gene, leading to copper accumulation in the liver, brain, eye, and peripheral nerves.

Trientine is a copper-chelating agent that removes copper from the body by forming a stable complex that is eliminated through urine. Trientine may also inhibit copper absorption from the intestinal tract.

In trials Cuvrior met its primary efficacy endpoint by demonstrating noninferiority to penicillamine as measured by nonceruloplasmin copper. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the United States, with about one-third of patients developing intolerance, the company said.

Trientine tetrahydrochloride is already on the market in Europe, where it is sold as Cuprior.

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

Here are several LD deals that have been recently publicly confirmed subsequent to the approvals.

Amber Specialty Pharmacy announces today that they will begin dispensing LENVIMA (lenvatinib). Used by itself, LENVIMA treats differentiated thyroid cancer (DTC) when it can no longer be treated with radioactive iodine, and hepatocellular carcinoma (HCC) when it cannot be removed by surgery. LENVIMA is used with other medications to treat additional types of cancers, including advanced renal cell carcinoma (RCC) and advanced endometrial carcinoma. LENVIMA is manufactured by Eisai.

Biologics by McKesson recently announced that they were selected by CTI BioPharma Corp. as a specialty pharmacy provider of VONJO (pacritinib) for the treatment of intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis.

Biologics also announced that they were selected by Agios Pharmaceuticals as a limited network provider for PYRUKYND (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

We’ve written about the emergence of telehealth services for the past several years. As then, we continue to see positive uptake, albeit much slower than some pundits expected.

Specialty pharmacies have ramped up patient outreach recognizing that more contact directly correlates with better outcomes, avoided adverse events, and better compliance and persistence. Telehealth is one of the methods that continues to show promise as it is applicable to just about any disease / condition under the specialty pharmacy umbrella.

A recent press release caught our eye as it bolsters the notion that telehealth is getting ready for prime time. As described, the Specialty Pharmacy Alliance (HOSP) has worked out a deal for their members to obtain URAC accreditation in Telehealth….. yes, another tutti-fruity accreditation flavor. As SPs search for ways to differentiate themselves adding yet another accreditation is one way to strut one’s stuff.

But, not all pundits are so bullish on telehealth. Doug Long, Vice President, Industry Relations, IQVIA, shared some thoughts on the subject at the recent Asembia conference. He stated that the pandemic did drive telehealth usage and that a recent poll showed that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months.

However, he suggested that the pandemic and the related shift to telehealth had mixed results on the specialty pharmacy market with telehealth reaching only 15% of patients and that the pace has stalled as the pandemic effects have subsided. One reason he suggests is that telehealth platforms don’t have access to the labs and the vitals and things that make doctors comfortable with prescribing new therapies. Long did cite that telehealth is going gang busters in the mental health patient management segment.

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URAC-HOSP Partnership to Focus on Specialty Pharmacy, Telehealth Accreditation

By SPC News Staff
A new partnership between Health System Owned Specialty Pharmacy Alliance (HOSP) and the accreditor URAC will make specialty pharmacy and telehealth accreditations more affordable for HOSP members while fostering collaboration between the organizations.

The partnership, announced during the AXS22/ Asembia Specialty Pharmacy Summit, will broaden the scope of benefits for HOSP members and allow the two organizations to work together on accreditation and certification measures, HOSP Board Chairman Gary Kerr, PharmD, told Specialty Pharmacy Continuum.

“We aim to educate our members and provide them with the tools they need as well as find new ways to work with partners like URAC in the integrated specialty pharmacy ecosystem,” Dr. Kerr said. “Education and collaboration around accreditation also support the HOSP mission to share common experiences and knowledge with industry partners, so we can grow and find timely solutions to our challenges together.”

As telehealth use increases in the wake of the COVID-19 pandemic, telehealth accreditation continues to evolve as well.

“This increase in utilization applies to how pharmacies manage patients and medication adherence during the pandemic,” Jeffrey Carr, URAC’s vice president, business development, said in an email. “URAC does not want price to be a barrier to improving quality. Our partnership allows HOSP members a more affordable option to pursue new pharmacy and telehealth accreditations.”

HOSP is a nonprofit coalition of major health systems focused on integrated specialty pharmacy best practices and advocacy.

“We select partners who align with our commitment and efforts to strengthen patient care through the integrated specialty pharmacy model,” Dr. Kerr said. “Our partners understand the value and importance of collaboration in the advancements in research, advocacy and education that are required to advance the quality of care to patients.”

The rumors about a big divestiture move by Centene Corp. finally materialized last week. Centene announced that they are spinning off both its PantherRx Rare pharmacy business as well as MagellanRx.

The PantherRx Rare divestiture is easier to understand….. sort of.
We were not able to confirm what Centene paid in January of 2021 (only 16 months ago) for the acquisition of PantherRx Rare. PantherRx was just sold for $1.4billion. Analyst estimates of 2022 EBITDA for PantherRx are in the area of $70 million which works out to an EBITDA multiple of ~20x!!! Given the long-term rosy forecasts for rare therapies, it is most curious that Centene would want to cull its herd by selling off perhaps its biggest cash cow.

MagellanRx is the pharmacy division of Magellan Health….. which itself is a division of Centene. Here is where it gets a little complicated. MagellanRx is a PBM and also a large national specialty pharmacy. The specialty pharmacy subdivision consists of a group of acquired providers which include Acaria Specialty Pharmacy (itself subsumed by MagellanRx’ Envolve Health)….. and don’t forget Exactus Pharmacy Solutions and Foundation Care sub-sub-sidiaries.

We remain a bit confused after reading the numerous press releases on the two deals in recent days. There has been no mention of Acaria/Envolve which generated upwards of $2billion in non-rare specialty revenue in 2021 according to published reports. We must assume that these operations are part of the MagellanRx spin off going to Prime Therapeutics. Trying to unpack the numbers becomes very difficult without specific EBITDA data for the PBM vs. the specialty pharmacy operations.

To help clarify (maybe not)….. Centene shelled out $2+billion to acquire Magellan in January of 2021. It appears that Centene will retain Magellan health services (includes a national behavioral health platform) and spin off only the Rx services (PBM and specialty pharmacy) for $1.35billion. Yeah, we need EBITDA to determine if this was a good deal….. and for whom.

The point that seems to have been glossed over is that Centene’s decision to get out of the PBM market comes after it has settled a series of pharmacy benefit management-related Medicaid cases across multiple states. The company said it will reserve a whopping $1.1 billion to settle these cases. That leaves Centene with about $1 billion which the company has already said it will use for stock buyback.

Over the years few new specialty therapies have been approved for Cardiology conditions. That may be about to finally change with a recent FDA approval.

Last week the FDA approved a new ORAL specialty therapy, Camzyos (mavacamten) from Bristol Myers Squibb (via MyoKardia, Inc., a wholly-owned subsidiary), indicated for (deep breath now) the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (obstructive HCM) to improve functional capacity and symptoms. Camzyos is the first and only FDA-approved allosteric and reversible inhibitor selective for cardiac myosin that targets the underlying pathophysiology of obstructive HCM.

Obstructive hypertrophic cardiomyopathy is an often un/mis-diagnosed condition in which the heart muscle thickens and impairs its ability to pump blood. Patients often have symptoms like those seen with heart failure. Symptoms have generally been treated with beta blockers and calcium channel blockers. Camzyos helps heart muscle fibers relax more in between beats, allowing more blood entering the ventricles to be pumped out when they contract helping to resolve symptoms. Echocardiogram assessments of left ventricular ejection fraction LVEF are required before and during treatment.

BMS announced a list price of $89,500 for a year of treatment. BMS also forecasted that they see sales in excess of $2 billion by 2029, slightly under analysts’ consensus.

BMS also indicated that Camzyos will only be available through the Camzyos Risk Evaluation and Mitigation Strategy (REMS) Program due to meaningful risk of heart failure due to systolic dysfunction.

Prescribers must be certified by enrolling in the REMS Program. Additionally, patients must also enroll in the REMS Program and agree to comply with ongoing monitoring requirements.

BMS also announced that Camzyos will be dispensed only to REMS enrolled patients through select, certified specialty pharmacies in a limited distribution program.

CLICK HERE to access prescribing information

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FDA approves new drug to improve heart function in adults with rare heart condition

FDA has approved Camzyos (mavacamten) capsules to treat adults with symptomatic New York Heart Association (NYHA)External Link Disclaimer class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve exercise capacity and symptoms.

Disease or Condition
oHCM is a rare disease that occurs when the heart muscle thickens, mostly in the septum (the heart wall that separates its right and left sides) and obstructs blood flow from the heart to the rest of the body.

The severity of oHCM varies, but it can cause serious symptoms and death. Symptoms include palpitations (the sensation the heart is pounding), shortness of breath, swelling of the legs, and decreased exercise capacity, which significantly impact daily activities and quality of life.

Effectiveness
The effectiveness and safety of Camzyos to treat NYHA class II-III oHCM was studied in adults who were randomly assigned to receive Camzyos or a placebo for 30 weeks. At the end of the study, 37% of participants treated with Camzyos improved on an endpoint measuring exercise capacity and symptoms, compared to 17% of participants in the placebo group.

Safety Information
Camzyos reduces contraction of the heart muscle, which can cause heart failure or totally block functioning of the ventricles (lower chambers of the heart). Patients who have a serious intercurrent illness (such as a serious infection) or arrhythmia (atrial fibrillation or other uncontrolled fast heart rhythm abnormality) are at greater risk of developing impaired heart muscle contraction and heart failure with Camzyos.

Because of the risk for heart failure, patients treated with Camzyos must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Patients must also avoid certain prescription and over-the-counter medicines that interfere with the metabolism (breakdown) of Camzyos.

Because of the potential for developing symptomatic or life-threatening heart failure if monitoring is not followed or if Camzyos is used with medicines that impair its metabolism, Camzyos is only available through a restricted program called the Camzyos Risk Evaluation and Mitigation Strategy (REMS). The REMS program helps ensure safe use by lowering the risk of heart failure due to impaired heart muscle contraction.

We’ve written frequently on two of the biggest trends in channel access, the shift away from buy-and-bill to other, less costly, sites of service as well as payer policies that have shifted reimbursement away from physician / hospital purchased drugs to specialty pharmacies. An article we want to review today is from Drug Channels. It is a great read and includes some fresh data to document these trends.

That article, an excerpt from a larger market report available for purchase from the company, first refreshes our understanding of the types of patient access now being employed. By now we are all familiar with White Bagging and Brown Bagging (if not, read the article). A term that is beginning to catch on is Clear Bagging in which the hospital-owned specialty pharmacy does the fulfillment.

What the data shows is that there has been a significant increase in White Bagging over the past two years with a 20% increase at physician practices and a 25% increase at hospital outpatient departments. Brown Bagging has virtually disappeared in these settings. Strangely, buy-and-bill at Home Infusion companies actually increased by a whopping 41% in the same time period and Brown Bagging more than doubled. Go figure! The data is based on a very large ‘n’ of payers nationally.

There are several points for further consideration.
– First, the list of impacted drugs goes beyond Oncology….. and is growing.
– Secondly, a number of specialty pharmacies have been designated by manufacturers as limited distribution (LD) partners (often exclusive) for certain drugs….. even bypassing traditional wholesalers. Since virtually all of these SPs are now also licensed distributors they can sell direct for professional use as a buy-and-bill drug….. under the medical benefit. (Not popular with hospitals and many providers.)
– Next, Payer and PBM policies have ramped in the past year+ to push these transactions over to the pharmacy benefit (even less popular) through rewriting the patient benefit plan…. including introducing patient OOP if billed as a medical benefit.
– Concurrently, PBMs and Payers have been able to co-opt these dynamics to further their cost management efforts on other, non-LD drugs. The data does not separately break out these LD drugs which further muddies up the total White Bagging picture.
– And finally, we can more easily understand why hospitals are increasingly desperate to redirect fills internally to owned specialty pharmacies to recapture lost buy-and-bill revenue.

White Bagging Update: PBMs’ Specialty Pharmacies Keep Gaining on Buy-and-Bill Oncology Channels
Drug Channels