The FDA recently approved a new antiviral therapy regimen, Sunlenca (lenacapavir) from Gilead Sciences, indicated for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations. Sunlenca is given in combination with other antiretroviral(s). 

Sunlenca is the first of a new class of drugs called capsid inhibitors. Sunlenca blocks the HIV-1 virus’ protein shell (the capsid)interrupting multiple essential steps of the viral lifecycle. 

As noted, Sunlenca is supplied as both an oral and subcutaneous injectable regimen. It comes with two induction schedule options (see below). Startup doses and then followed by maintenance injections every six months.

Recommended dosage:

Initiation with one of two options followed by once every 6-months maintenance dosing.

• Initiation Option 1

   Day 1                927 mg by subcutaneous injection (2 x 1.5 mL injections)

600 mg orally (2 x 300 mg tablets)

   Day 2                600 mg orally (2 x 300 mg tablets)

• Initiation Option 2

   Day 1                600 mg orally (2 x 300 mg tablets)

   Day 2                600 mg orally (2 x 300 mg tablets) 

   Day 8                300 mg orally (1 x 300 mg tablet)

   Day 15              927 mg by subcutaneous injection (2 x 1.5 mL injections)

• Maintenance

927 mg by subcutaneous injection (2 x 1.5 mL injections) every 6

months (26 weeks) from the date of the last injection +/-2 weeks.

Gilead confirmed that Sunlenca injection and tablets are expected to cost $42,250 in the first year of therapy and $39,000 annually after that (maintenance injections only).

Gilead did not provide detail on how Sunlenca would be distributed. Given its benign safety profile, it could be expected that the therapy regimen (oral and sub-q injectable) will be provided by specialty pharmacy in limited distribution. Compliance and persistence are central to therapy success.

CLICK HERE to access prescribing information

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FDA Approves Sunlenca – New HIV Drug for Adults with Limited Treatment Options

December 22, 2022 The U.S. Food and Drug Administration approved Sunlenca (lenacapavir), a new type of antiretroviral medication for adult patients living with human immunodeficiency virus type 1 (HIV-1), whose HIV infections cannot be successfully treated with other available treatments due to resistance, intolerance, or safety considerations. After the starting dose is completed, Sunlenca is administered…………………

CLICK HERE to access the full FDA press release

So, can you easily explain the differences between a chemically synthesized drug and a biologic? 

You might be surprised how difficult that has become.

The article below should be thought provoking for those in the specialty pharmacy industry as it goes to the core of what exactly SPs actually sell….. chemically synthesized drugs and biologics. But…. if you were asked to list which column your drugs should be listed you would likely be stumped.

Here’s why….. the Consolidated Appropriations Act of 2020 included new guidance on how drugs can be classified. Specifically, the change states that the purpose of the amendment is to allow for the possibility that a manufacturer may chemically synthesize a biologic product. Huh?? 

Here’s an example…. Vondys 53, a biologic analog made through synthetic chemistry – not by any biologic process – is considered a biologic. The author of the (somewhat ‘tongue in cheek) article below polled pharma colleagues for their opinion as to whether similar drugs – are or aren’t biologic drugs. 

The results: 

41% agreed that these new drugs were biologics, 39% disagreed, 20% responded “Confused” 

….. so am I. 

Non-pharmacists may be a bit intimated by the technical references in the article…. just skip over them as that’s not the point of this Alert. The real purpose is to alert those working in the specialty pharmacy industry that today’s crop of new biologics ‘ain’t your grandfather’s biologics’. If you are going to be selling both chemically synthesized drugs and biologics you should have, at a minimum, a basic understanding of these developments.

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What is a Biologic Drug Anyway?

Or, How I Learned to Stop Worrying and Love My Own Definition

January 19, 2020 — Recently I posted on “biologic drugs” which were approved in 2019. All was well in my little scientific universe, until I received this in an email: 

“You recently reported on “14 novel biologic drugs,” referring to ‘novel’ biopharmaceuticals approved through CDER. But, … You also include several synthetic drugs, which are clearly not biopharmaceutical/biologics.”

Wait, what? Synthetic drugs are clearly not biopharmaceutical/biologics? Sure, the siRNA drug, Givlaari, and the antisense oligonucleotide, Vyondis 53, are both technically made by chemical synthesis. But, not considering these RNA-based drugs “biologic drugs” just felt… wrong.

It got me thinking – what is the definition of a biologic drug, anyway? I immediately started my research the way any good, card-carrying scientist does — I punched it into the Google:

GOOGLE:  A biopharmaceutical, also known as a biologic medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. [source] Wikipedia

Though Wikipedia delivers good answers to probably 90% of my questions, I knew this definition was incomplete – nobody considers natural products like penicillin “biologic drugs.” Not wanting to spend the rest of my Sunday night reading FDA guidance documents, I did instead what any good millennial does – I asked social media.

As one comment pointed out, the term “biologic” itself is anachronistic, coming from a time when chemistry and biology were considered distinct (i.e. before chemists started getting upset about Nobel Prizes in Chemistry going to “biologists”). The Biologics Control Act, which first gave the US government control over the processes to make biological products, was passed in 1902 – long before it was clearly established that proteins are polymers of amino acids and that genetic information is stored chemically in nucleobases.

Digging Deeper

Based on the lack of strong consensus, I decided this was a topic worth diving further into.

Readers likely remember that “biologic” became a charged term in the 2000’s, due to expensive biologic products like filgrastim and epoetin alfa, approved under Biologic License Applications (BLAs) rather than New Drug Applications (NDAs). Since the abbreviated generic drug approval process (ANDA) only applied to originals filed under NDAs, there was no way to create a “generic” biological product without repeating expensive and lengthy clinical trials. This loophole led to the creation of the abbreviated follow-on biologicals approval pathway (aBLA) through the 2009 Biologics Price Competition and Innovation Act (BPCI).

So how are the adjectives, “biologic” or “biological,” legally defined in the US Code of Laws after BPCI in 2009? Turns out, they’re not. The term “biological product,” is legally defined only with examples, not properties:

“The term “biological product” means a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of a disease or condition of human beings.”

42 U.S. Code § 262. Regulation of biological products.

With no basis in the text for determining what an “analogous product” is, no definition of what exactly makes a product “biologic” or “biological,” and with, of all things, arsphenamine (or any other trivalent organic arsenic compound) being the only exemplified exception, no wonder everyone is confused. The FDA and the industry spend an enormous amount of resources deciding new registrants on a case-by-case basis, which was part of the justification for a recent change in the definition. 

A Legal Update on Synthetic Processes

It gets better. The Further Consolidated Appropriations Act, 2020, signed into law in December 2019, amended this definition by removing the phrase “except any chemically synthesized polypeptide”: 

“The term “biological product” means a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (deleted: except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of a disease or condition of human beings.” 

New legal definition of “biological product” after the Further Consolidated Appropriations Act.

The recent FDA press release on the change states that the purpose of the amendment is to allow for the possibility that a manufacturer may chemically synthesize a biologic product. Under the old definition, if an original biologic product was licensed under a BLA, a new chemically synthesized biologic follow-on could not be licensed through an abbreviated aBLA pathway, since it was made by a synthetic process, but also could not be licensed through an ANDA, since the original was not filed under an NDA.

In the end, what this demonstrates is that there are at least two simultaneous uses of the term “biologic” in the industry – an arbitrary regulatory definition to decide what drugs are licensed under BLAs vs. NDAs, and a scientific colloquialism which most of us use on a day-to-day basis. Neither definition is clear and unambiguous, and no single criterion (e.g. chemical process) universally captures all cases. 

Biolognas, Anybody?

Having both uses at the same time is confusing enough to make you want to give “synthetic biologics” a different name without “biologic” in it. But what would we call them? Biologishes? Biolognas? I guess I have a year to think about it before 2020’s Biologic Drug Approvals post. 

But back to my original question– since oligonucleotide drugs are all filed under NDAs, they’re regulated more like most small molecules than most biologics. But just because they’re filed under NDAs, doesn’t mean we can’t still call them biologics (e.g. insulins are all filed under NDAs). Anyway, all this makes me very glad that we have a lot of brilliant professionals working in our very important Regulatory Affairs departments, though I’m glad it doesn’t include me!

We know that there are a limited number of specialty pharmacies in the US. A small number of customers makes it hard for any new product or service to have enough customers to enable a product to break even….. let alone be profitable. So, we are curious when we see new products coming to market that target SPs. Today we are talking about software programs that manage the upstream / downstream patient journey that SPs have managed for decades (often using Excel spreadsheets and even index cards back in the day.)

The new ‘state-of-the-art’ platforms are fairly sophisticated. They capture loads of patient data that can include patient electronic medical records (with permission), physician ordering portals, prescribing protocols that go down to the specific medication profile, patient financial assistance, obtaining prior-authorizations, refills, patient reported side effects / adverse reactions / dosing schedules / dose reductions, and on-and-on-and-on.

As noted in the article below, Henry Ford Health sees new opportunity in this small segment. Why? Because the segment is suddenly much bigger. Hospitals and health systems are increasingly opening owned and operated specialty pharmacies enabling them to  rapidly catch up with well-established specialty pharmacies by installing one of these software platforms. There are a lot more hospitals than stand alone SPs. 

The Henry Ford platform was first developed a decade ago and it sounds like its newest mousetrap is on par with other leading providers of comparable platforms.

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Henry Ford Health’s Specialty Pharmacy Software Goes National

Platform developed by Henry Ford Health now serves systems in seven states, with plans for expansion

DETROIT/PRNewswire/ — Henry Ford Innovations (HFI), one of the nation’s leading healthcare innovation programs, announced today that its specialty pharmacy software platform, DromosPTM™, now serves pharmacies and patients across the country. Developed and implemented by Henry Ford Health in 2013 to address an internal need, the platform quickly grew beyond Detroit and is now used by seven leading specialty pharmacies and healthcare systems from Massachusetts to Utah. Additionally, more systems are planning to adopt the platform in the coming months.

Signifying its progression in technology- and consumer-impacted healthcare, DromosPTM™ fills a long-existing gap in the specialty pharmacy industry by offering efficient patient-focused care and service. Specialty pharmacies differ from traditional pharmacies by coordinating aspects of patient care and disease management. They deliver medications that demand special handling, storage and distribution for often chronic and rare conditions.

These partnerships allow increased functionality and provide patients across the country a better experience and care.

This first-of-its-kind application solves several problems regularly seen in specialty pharmacy cases such as finding financial assistance for costly prescriptions, enhanced monitoring and emphasizing best practices. For example, organizations are leveraging the platform by expanding their pharmacy capabilities within patient portals, allowing patients to easily request often-complicated prescription refills and transfers.

“We are proud to have leading healthcare organizations joining our DromosPTM™ network to transform lives through advanced patient therapy management,” said Lisa Prasad, Henry Ford Health’s Chief Innovation Officer and leader of Henry Ford Innovations. “These partnerships allow increased functionality and provide patients across the country a better experience and care.” 

DromosPTM™ was designed and developed at Henry Ford Health’s specialty pharmacy, Pharmacy Advantage, which is supported by CarepathRx LLC. At the time, Pharmacy Advantage team members recognized an opportunity to improve care administration and outcomes, resulting in the development of the platform and partnership with HFI for licensing and distribution.

“DromosPTM™ is helping the specialty pharmacy industry scale their businesses and improve both operational and clinical outcomes as their organizations continues to grow” said David Shepherd, President and CEO of Community Care Services. “By helping remove barriers from patients, like cost and prior authorizations, DromosPTM™ has created positive patient outcomes already.”

Established in 2011 to develop and commercialize its intellectual assets, HFI has enacted more than 30 licensing agreements, representing over $100 million in future revenue. HFI remains committed to creating best-of-class partnering programs with early-stage and corporate collaborators and serving as a gateway to international healthcare companies.

To learn more about DromosPTM™ please visit https://dromosrx.com or call (888) 376-6670.

SOURCE Henry Ford Health

The FDA recently approved a novel, ORAL monotherapy treatment, Rezlidhia (olutasidenib) from Rigel Pharmaceuticals, Inc., to treat adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. However, the approval included a boxed warning for potentially fatal Differentiation Syndrome.

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.  Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.

Rigel did not announce pricing for the new therapy, nor did they provide details on distribution. Given the segment and relatively small patient base it is likely that Rezlidhia will launch through limited distribution.

CLICK HERE for prescribing information

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FDA approves olutasidenib for relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation

The Food and Drug Administration (FDA) has approved olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. The FDA also approved the Abbott RealTime IDH1 Assay to select patients for olutasidenib.

Approval was based on Study 2102-HEM-101 (NCT02719574), an open-label, single-arm, multicenter clinical trial that included 147 adult patients with relapsed or refractory AML with an IDH1 mutation confirmed using the above assay. Olutasidenib was given orally,150 mg twice daily, until disease progression, unacceptable toxicity, or hematopoietic stem cell transplantation. The median treatment duration was 4.7 months (range: 0.1 – 26 months). Sixteen (11%) patients underwent hematopoietic stem cell transplantation following olutasidenib.

Efficacy was established on the rate of complete remission (CR) plus complete remission with partial hematologic recovery (CRh), the duration of CR+CRh, and the rate of conversion from transfusion dependence to independence. The CR+CRh rate was 35% (95% confidence interval [CI]: 27%, 43%), including 32% CR and 2.7% CRh. The median time to CR+CRh was 1.9 months (range: 0.9 – 5.6 months), and the median duration of CR+CRh was 25.9 months (95% CI: 13.5 months, not reached).

Among the 86 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 29 (34%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 61 patients who were independent of both RBC and platelet transfusions at baseline, 39 (64%) remained transfusion independent during any 56-day post-baseline period.

The most common adverse reactions (≥20%) were nausea, fatigue/malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea, and transaminitis. The prescribing information contains a Boxed Warning alerting health care professionals and patients about the risk of differentiation syndrome which can be fatal.

The recommended olutasidenib dose is 150 mg taken orally twice daily on an empty stomach (at least 1 hour before or 2 hours after a meal) until disease progression or unacceptable toxicity. For patients without disease progression or unacceptable toxicity, treatment is recommended for a minimum of 6 months allowing for clinical response.

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 designated partner(s).

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

Amber Specialty Pharmacy Selected to Dispense TZIELD (teplizumab), a Monoclonal Antibody Treatment to Delay Onset of Stage 3 Type 1 Diabetes in Patients with Stage 2 Type 1 Diabetes

OMAHA, Neb., — Amber Specialty Pharmacy announces that it will begin dispensing TZIELD (teplizumab), an anti-CD3 monoclonal antibody, to delay Stage 3 Type 1 Diabetes (T1D) in patients ages 8 years and older with Stage 2 T1D. Indication for individuals ages 8 years and older with Stage 2 T1D is categorized by the presence of two or more T1D-related autoantibodies and abnormal blood sugars. TZIELD™ is manufactured by Provention Bio, Inc., a New Jersey-based biopharma company.

Orsini Specialty Pharmacy Selected by Provention Bio as a Limited Distribution Partner for TZIELD

ELK GROVE VILLAGE, Ill., /PRNewswire/ — Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases, and gene therapies, has been selected by Provention Bio, Inc. https://www.gilead.com/as a specialty pharmacy for TZIELD® (teplizumab). TZIELD is the first and only treatment indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adult and pediatric patients aged 8 years and older with Stage 2 T1D approved by the FDA.

ELAHERE Available at Biologics by McKesson

Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by ImmunoGen, Inc. as the sole specialty pharmacy provider for ELAHERE (mirvetuximab soravtansine-gynx) for the treatment of adult patients with folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens.

Rigel Pharmaceuticals recently received the green light from the FDA for their oral IDH1 inhibitor, REZLIDHIA™ (olutasidenib).  REZLIDHIA™ is indicated for treatment of acute myeloid leukemia (AML) in a difficult to treat, relapsed or refractory (R/R) patient population who have a mutation in isocitrate dehydrogenase-1 (IDH1).  Mutations of IDH1 are found in 7-14% of patients with AML and there have been few treatment options for these patients, particularly in the second line.  The FDA approval was supported by data from the open-label Phase 2 registrational study evaluating REZLIDHIA™ monotherapy at a dose of 150 mg twice daily in 153 mIDH1 R/R AML patients. The efficacy-evaluable population was 147 patients who initiated REZLIDHIA™ at least six months prior to the interim analysis cutoff date of June 18, 2021, and who had a centrally confirmed IDH1 mutation.  Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.¹ 

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.² Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.³ Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. 

REZLIDHIA™ provided a complete remission (CR) combined with partial hematological recovery (CRh) within less than two months (mean time to response 1.9 months) lasting for over two years (mean duration 25.9 months) and demonstrated a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission.

“The 25.9 months median duration of CR+CRh is a clinically meaningful improvement for AML patients and appears to be longer than currently available treatment options,” said Jorge E. Cortes, M.D., Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator. “Given the limited treatment options for adult patients with mIDH1 R/R AML, who typically have a poor prognosis, REZLIDHIA™ may provide an effective, new treatment option with a well characterized safety profile.”

Complete remission was achieved for over 28 months (mean 28.1 months).  Over 1/3 of patients (34%) treated with REZLIDHIA™ were able to avoid platelet and/or red blood cell transfusions at 56 days and unlike other treatments in the relapsed/refractory AML space, there is no need for cardiac monitoring. REZLIDHIA™ has a black box warning for differentiation syndrome which can be fatal; if suspected, differentiation syndrome should be treated with corticosteroids and hemodynamic monitoring.  

Hats off to Rigel Pharmaceuticals for getting the FDA nod just four months after acquiring the asset.  REZLIDHIA™ has the potential to address an unmet need in a niche patient population typically subject to a poor prognosis.  It is motivating to see companies like Rigel invest in bringing to market therapeutic options for small, tough to treat patient populations.

Click here for the full press release from Rigel Pharmaceuticals: https://www.rigel.com/investors/news-events/press-releases/detail/349/rigel-announces-u-s-fda-approval-of-rezlidhia

Click here for full prescribing information: https://www.rezlidhia.com/downloads/pdf/REZLIDHIA-Full-Prescribing-Information.pdf

Whoa….. we missed covering this FDA approval.

Way back in October the FDA approved a new subcutaneous treatment, Tecvayli from Janssen Biotech, for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy. Tecvayli is a first-in-class, bispecific T-cell engager antibody engineered to redirect body’s immune system to recognize and kill cancer cells.

Multiple myeloma is typically diagnosed in people aged between 65 and 74 and affects more men than women. According to the American Cancer Society’s estimates, close to 35,000 new cases of multiple myeloma are likely to be diagnosed in the United States this year.

The approval, however, comes with a boxed warning and will only be available through a restricted program under a REMS called the Tecvayli REMS because of the risks of cytokine release syndrome, and neurologic toxicity, including ICANS.

Tecvayli is Janssen’s fourth approved treatment for multiple myeloma. A company spokesperson said the therapy will launch with a list price of $39,500 per month, with the overall pricing ranging between $355,000 and $395,000 for a nine-to-10-month course.

Janssen has not yet announced distribution plans. Given the cost and that Tecvayli is a sub-q therapy, it is expected that one or more specialty pharmacies will be selected for a Limited Distribution program.

CLICK HERE for prescribing information

In the spirit of continuing to elevate our client offerings, services and experiences Anton Health is proud to welcome Tara Mautone as the Executive Director of Client Services. Tara joins Anton with over 17 years of commercialization experience from a wide variety of leadership roles within both large pharmaceutical organizations and small, start-up biotech companies. This includes nearly 10 years of building a deep level of expertise in all areas of market access, leading nationally recognized commercial teams in both sales and marketing and being recognized as HBA’s Rising Star and Market Access Leader of the year.

Liz Turner, Anton Health’s VP of Operations shared, “Anton Health is committed to ensuring our clients can successfully navigate an ever-complicating market access environment. Anchored in our one-of-a-kind advisor network, we have a vision of delivering unmatched relevant, real- world solutions through the lens of the customer. Tara has been in the role of our clients responsible for developing market access strategies for pipeline assets at small start-ups and managed the pressures of increased competition in already crowded therapeutic areas. She understands how critical it is for the deliverables promised by a thought partner like Anton Health to be best-in class no matter how big or small the ask.”

We have an unrelenting desire to surpass our client’s expectations. We’re thrilled to have Tara leading the efforts for Anton Health that ensure we’ve optimized every opportunity to see this through.

Tara Mautone is a strategic commercial leader with over 17 years of pharmaceutical and biotech experience. During her career she has launched several products in multiple therapeutic areas, specializing in market access and commercialization strategy. Prior to joining Anton Health, Tara spent two years at Biosplice Therapeutics where she helped build and develop the commercial infrastructure needed to prepare for the organizations first product launch. Prior to that she spent 13 years at Daiichi Sankyo, where she held various roles in Sales Leadership, Sales Training, Market Access, Operations and ultimately, Sr. Leadership roles with broader responsibilities for the US commercial business function. Tara was recognized for her work and leadership by being named HBA’s rising star and Market Access Leader of the year. Tara graduated with a BA in Psychology from Edinboro University. She resides in Millburn, NJ with her husband Anthony, their two daughters, Giada (10) and Marcella (7) and their puppy Bruno.Tara’s phone number is 814-931-9488 and email is tara.mautone@antonhealth.com

Optum is turning to technology to bring down special infusion spend. Others have tried a variety of ideas to achieve this goal going back to the early 2000s with limited results….. but the old saying goes….. Try…. Try…. And Try Again.

Optum is optimistic that they can deliver sustainable savings in the difficult-to-manage infusion settings where placing constraints on hospitals and physicians are often impossible. Instead, the new platform being touted by Optum, branded as Specialty Fusion, makes it ‘easier for clinicians to cut wait time for multiple treatment regimens to be approved, support navigating patient treatment plans, and optimizing health benefits’.

Perhaps one area where Specialty Fusion will help push platform adoption is in the area of cost containment. They say they can realize savings of 17%! The announcement did not address the elephant in the room, the fact that hospital infusions generally cost twice that for the same infusion in an infusion clinic or physician’s office. One would think that even greater saving could be realized by tackling that thorny issue.

As with a lot of new technology, the Specialty Fusion portal will need to be very user friendly and offer meaningful clinical and operational information to gain provider support.

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Optum unveils new platform for managing specialty drug costs

Optum has launched a new solution aimed at the specialty drug market.

Specialty Fusion arms payers and providers with real-time insights into which specialty therapies are the most effective for the patient at the lowest cost. The platform leads to quicker treatment approvals for patients as well as a similar experience for providers at the point of care, Optum said in an announcement.

Internal analysis of the solution suggests it can drive cost savings of 17%.

“The average specialty patient sees more than five care providers per year, while taking more than 10 drugs on average. They often have to wait for multiple treatment regimens to be approved and need better support navigating their treatment plan and health benefits,” said Kerri Tanner, senior vice president at Optum Rx, in a statement.

“We developed a new comprehensive, benefit-agnostic solution that supports streamlined treatment decision-making for care providers, helping patients get on their therapies faster, while driving down high costs,” Tanner said.

Specialty Fusion is currently available to all large health plans, Optum said.

Specialty drugs account for an increasing amount of healthcare spending, with costs expected to reach $505 billion by 2023, Optum said. Finding ways to ensure people can access therapies that they need at a manageable cost has been of critical concern in the pharmaceutical space.

Through Specialty Fusion, providers can kickstart prior authorization through a single portal, and see comparisons for other lower-cost and clinically appropriate options for the patient. The platform is designed to reduce administrative effort and connect patients with these treatments 50% quicker, Optum said.

For example, a doctor could prescribe an immunosuppressive drug that is covered by the patient’s insurance, with a cost of about $1,200. Specialty Fusion would identify alternatives, bringing the cost down by $700 on average.

In addition, the platform arms docs with information on pharmacy networks, best sites of care, dosage management policies and other levers they can use to further lower costs.

“Managing specialty drugs at the earliest possible moment is critical to improving care, clinical outcomes and the patient experience,” said Sarah Dye, senior vice president at Optum Health, in a statement. “Optum Specialty Fusion uniquely partners with the provider to change the status quo of prior authorization, reduce their administrative pain and ultimately help them provide better care for their patients.”

It has finally happened….. the decades’ long wait for the approval of the holy grail in hemophilia treatment….. a gene therapy that replaces the need for frequent blood factor infusions.

BUT….. the approval of this new ‘cure’ needs some explaining….. including why the new therapy was approved with….. count ‘em….. 39 NDCs.

First, this new one-and-done therapy is called Hemgenix (etranacogene dezaparvovec-drlb) and was developed by CSL Behring. 

Second, Hemgenix is indicated only for Hemophilia Type B (congenital Factor IX deficiency).

Type B represents 8,000 individuals in the US vs. Type A (congenital Factor VIII deficiency) at over 25,000.

CSL trials showed that 94% of individuals with Type B had a positive response and realized a 54% reduction in the annual bleed rate…. (not too shabby but far from a total cure). 

Third, is the little sniggly thing related to cost. 

Hemgenix will hit the market at a sizzling $3.5 million making it the world’s most expensive drug. That may stall access for patients as payers struggle to deal with the expense burden.

Fourth, here is what you’ve been waiting for……

Hemgenix was approved with 39 NDCs. 

It is available in multi-vial kits providing 100mL – 480 mL (10mL per vial) as Hemgenix requires weight-based dosing….. hence the 39 unique NDCs.

It remains to be seen how quickly (if?) Hemgenix will see strong uptake. Individuals with hemophilia are very cautious when considering any change in the factor they use due to a historical correlation with triggering inhibitor reactions. As we know, blood factor dispensing is predominantly provided by a small sub-set of specialty pharmacies nationally. It is likely that Hemgenix will be distributed directly to leading hemophilia treatment centers / hospitals due to cost and infusion management. 

CLICK HERE to access full prescribing information