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FDA Approves ORAL Tx for Rare Hemolytic Anemia – Pyrukynd

Last week the FDA approved a new oral therapy, Pyrukynd (mitapivat) from Agios Pharma, to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency. PK is a disorder in which red blood cells are destroyed faster than they can be made.

PK deficiency is rare, with an incidence of three to nine cases per one million people. Best estimates are that PK deficiency affects approximately 3,000 people in the U.S. However, PK deficiency is often misdiagnosed or goes undiagnosed, making it difficult to determine its frequency in the general population.

Pyrukynd became the first FDA-approved medicine for the disorder. It is an inherited disease that can cause lifelong complications including gallstones, pulmonary hypertension, osteoporosis and more. Patients with PK deficiency have a range in severity of symptoms, which include fatigue, unusually pale skin, jaundice (yellowing of skin and eyes), shortness of breath, and a fast heart rate. Patients can also develop an enlarged spleen, too much iron in their blood from repeated blood transfusions, and gallstones (small deposits in the gallbladder or bile ducts).

Pyrukynd must be closely monitored for dose titration from 5 mg up to the maintenance dose of 50 mg over the first 3+ months of therapy.

Pyrukynd will launch at an annual price of $334,880, before insurer’s discounts.

Given its high cost and very small population in the US, it is highly probable that Pyrukynd will be placed into exclusive distribution with a specialty pharmacy.

FDA approves treatment for anemia in adults with rare inherited disorder

February 17, 2022
FDA has approved Pyrukynd (mitapivat) tablets to treat hemolytic anemia (a disorder in which red blood cells are destroyed faster than they can be made) in adults with pyruvate kinase (PK) deficiency.

Disease or Condition
PK deficiency is an inherited disorder that causes premature red blood cell destruction, which leads to anemia (a decreased number of red blood cells).

PK deficiency is rare. In clinical practice, its frequency is approximately three to nine cases per one million people. However, PK deficiency likely is misdiagnosed or undiagnosed, making it difficult to determine its frequency in the general population.

The effectiveness of Pyrukynd was evaluated in two studies. One was a randomized, double-blind, placebo-controlled clinical study of 80 adults with PK deficiency who did not receive regular blood transfusions. The other was a single-arm study of 27 adults with PK deficiency who received regular blood transfusions. In these studies, patients received Pyrukynd up to 50 mg orally twice daily after an initial dose titration (adjustment) period. Participants received Pyrukynd for an average duration of about 24 weeks in the randomized study and an average duration of about 40 weeks in the single-arm study.

In the randomized study, the effectiveness of Pyrukynd was based upon hemoglobin response defined as a 1.5 g/dL or greater increase in hemoglobin concentration from the beginning of the study that was sustained at two or more scheduled assessments. Hemoglobin is a measure of the amount of red blood cells that are not destroyed. At the end of the study, 40% of participants receiving Pyrukynd had a hemoglobin response compared with no participants receiving placebo.

In the single-arm study, effectiveness was based on reduction in transfusion burden, defined as at least a 33% reduction in the number of red blood cell units transfused during the last 24 weeks of treatment compared with the historical transfusion burden on the individual participant (standardized to 24 weeks). Thirty-three percent of participants who received Pyrukynd met this reduction in transfusion burden, including 22% of participants who did not require any transfusions over the last 24 weeks of treatment.

Safety Information
Because of drug interactions, patients should either avoid using Pyrukynd with certain other medications or adjust the Pyrukynd dose. Abruptly stopping Pyrukynd could worsen premature red blood cell destruction. Patients should avoid suddenly stopping or pausing Pyrukynd and follow their health care provider’s instructions for discontinuing treatment.

The most common side effects of Pyrukynd are decreases in estrone and estradiol (types of the estrogen hormone) in men, increased urate (a type of salt in the body), back pain, and joint stiffness. The effects of Pyrukynd on estrone and estradiol could not be reliably assessed in women because of normal changes in these hormones during the menstrual cycle and use of hormonal contraception.

This application received orphan drug designation, fast track designation, and priority review.


Another Nifty Approach to Lower Specialty Infusion Spend

Last week we sent you a review of an article detailing the creation of a network of alternate site infusion providers by AscellaHealth. On Friday we ran across the article below detailing a new program, Optum Specialty Infusion.

It is noteworthy that, while the approaches differ, both programs have the same goal…. to contain the skyrocketing cost of specialty infusions. Neither currently appear to rely on benefit changes to drive the change in utilization (at least yet).

Optum is going more high-tech. It appears that when requesting a prior authorization for infused drugs the prescribing physician is presented with a menu detailing the differences, across benefits, for infused medications for the specified indication/diagnosis. Since so many patients are shoulder coinsurance for infused medications, a shift in therapy can make a huge out-of-pocket difference for a patient. Also, patients have been known to skip scheduled infusions because they can’t handle their financial share.

It would be nice to know how much information is available to the physician at the time of processing the prior auth from the ‘menu’. We saw direct links to clinical studies and Journal articles on such platforms some years back when they were first being touted for Oncology. Such detail would further reinforce the selection of a lower cost drug. Also, a reference to ‘across benefits’ suggests that oral meds may be included in the menu. Last thought….. the Optum approach doesn’t shift where the therapy is delivered, which should create less conflict with hospital sites of service.

At this point Optum Specialty Fusion is available only to large health plans.

Optum Launches Specialty Fusion to Simplify Specialty Care and Lower Costs

First-to-market solution helps health care payers manage the increasing cost of specialty medicines, delivering up to 17% savings across medical and pharmacy spend

February 17, 2022 — EDEN PRAIRIE, MINN. : Optum today announced the launch of Optum Specialty Fusion, a first-of-its-kind specialty medication management solution, to simplify care for patients with complex conditions and lower the cost of expensive specialty drugs.

With specialty drug costs projected to reach $505 billion by 2023, health plans and care providers are seeking ways to ensure people get the treatments they need more affordably. Using Optum’s data and clinical expertise, Specialty Fusion provides health care payers and care providers real-time insight into the most effective specialty treatment at the lowest cost to the patient. Results include a simpler experience for providers and faster treatment approvals for patients, while delivering 17% total cost savings in medical and pharmacy spend.1

“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, Optum Rx. “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.”

In line with Optum’s commitment to develop solutions that provide more transparency and better connect the pharmacy and medical benefits across health care, Specialty Fusion was designed to streamline and simplify the medication management process. Using Specialty Fusion, care providers can initiate the approval process for specialty drugs requiring prior authorization using a single portal. Treatment options are then compared against dozens of clinically appropriate, lower-cost options across benefits in seconds. Specialty Fusion eliminates the need for a cross-benefit prior authorization process – a burden for providers – and secures treatment approval in real time. This reduces administrative hassle and results in an expected 50% faster access to therapy for patients, while lowering costs.

For example, when a provider prescribes an immunosuppressive drug on the medical benefit, treatment may cost $1,200. Using Specialty Fusion, a provider can see coverage recommendations and select a therapeutically equivalent treatment for $700 less, on average. Informed by Optum data and insights, Specialty Fusion provides savings options such as preferred products, dosage management policies, best sites of care and additional cost-control levers such as available discounts and pharmacy networks [possibly AscellaHealth??].

“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, Optum Health. “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.”


Wagons ‘ho to Alternate Site Utilization

Payers have been struggling for years to move infusion site of care services out of higher cost hospital outpatient administration to alternate sites….. community physician offices or stand-alone infusion clinics. There is no debate that every patient so moved saves significant dollars with no loss in quality of care and, very often, greater patient convenience. That’s a Win-Win.

So, just like an improvement over sliced bread, AscellaHealth has come up with a bright idea….. create a network of non-hospital infusion providers that allows insurers to more readily push members into the lower cost network sites. It takes the PBM concept and significantly expands its functionality.

It will be interesting to see how benefit design can be crafted to fully complement utilizing the network. Assuming it gains traction, it could be a great model for the future.

Ascellahealth Makes Case For Alternate Sites

HME News Staff

February 8, 2022 — BERWYN, Pa. – AscellaHealth has released a white paper that details the value of site of care (SOC) optimization for infusion therapies, a leading strategy for reducing the cost of specialty biologic medications, improving outcomes and enhancing access to more convenient, quality focused settings.

“Recognizing the importance of SOC optimization, AscellaHealth offers a Home Infusion Pharmacy Network as part of our overall strategy to cut costs without compromising quality of care,” says Dea Belazi, president and CEO, AscellaHealth. “Our pharmacy providers were chosen for their national reach, high-touch capabilities and thorough home infusion expertise, enabling care delivery in the highest-quality, lowest-cost care settings and enhancing outcomes via increased compliance rates.”

In the four-page paper, “Site of Care Optimization Lowers Specialty Drug Costs, Reduces Hospital Visits, Ensures Care Quality,” AscellaHealth makes the case that the exact same medications, administered using the same method, can be more than two- to three-times more expensive in the hospital setting vs. a standalone infusion center.

“Insurance companies, self-funded companies and other stakeholders will find significant value in applying a high level of expertise to the management of these specialty drugs by working with AscellaHealth and its partners in SOC optimization,” Belazi said. “Flexibility of alternate sites also contributes to a higher quality of life by improving patient access to care, reducing time away from work or school, and offering a degree of independence to patients living under otherwise rigid medical parameters.”


Is Something ‘Rare’ in Your Specialty Pharmacy Future?

Is it the new ‘must-have’ in specialty pharmacy?
So it would appear……it’s an accreditation in Rare Disease Pharmacy.

A couple of years ago it was bragging rights to be able to say, “My specialty pharmacy is accredited as a Rare Disease Pharmacy.” Well, the numbers are starting to swell, and may eventually dilute that differentiation if too many SPs jump on that band wagon.

A quick Google search popped up 10 SPs that now have the RARE certificate on their lobby walls (from URAC or ACHC). These include Orsini, PantherRx Rare, Soleo Health, Fairview, Accredo, Premier, Vanderbilt, Perform, and Chemistry Rx…. there are likely more. But the number of SPs that are in now process or planning on going the rare route might be more telling.

We have huge admiration for SPs that get accreditation. We’ve guided literally dozens of SPs going through the SP accreditation process….. starting as early as the mid-1990s. The designation is a demonstration of the commitment to quality that every payer and manufacturer has come to regard as a gold standard in the industry.

Manufacturers are more likely to be impressed with the extra accreditation since virtually ALL rare therapies are launched through exclusive or limited distribution. URAC’s web site includes the following as a key benefit of a rare disease accreditation…. “[It] Elevates contracting power, credibility and differentiation over competitors as payers and stakeholders are assured of organizational competency.”

Evernorth’s Accredo Earns URAC Rare Disease Pharmacy Center of Excellence Designation

BLOOMFIELD, Conn., Feb. 10, 2022 /PRNewswire/ — Accredo®, the specialty pharmacy subsidiary of Evernorth, Cigna Corporation’s health services business, was awarded URAC’s Rare Disease Pharmacy Center of Excellence designation. The URAC accreditation recognizes Accredo for demonstrating commitment to quality health services, patient safety and improved outcomes for patients living with rare diseases.

“The patients we serve are living with the most complex health issues and they deserve the highest quality of specialty care,” said Matt Perlberg, president, pharmacy businesses, Evernorth. “This accreditation is an important indicator of excellence and reflects our ongoing focus on improving health outcomes for patients with rare diseases.”

An estimated 25 to 30 million people in the U.S. suffer from a rare disease and approximately two-thirds are children. Accredo’s rare disease therapeutic resource center provides specialty pharmacy and in-home clinical care for patients living with 23 different rare diseases. The company’s care delivery team includes approximately 500 specialist pharmacists and 600 field-based nurses as well as social workers and dieticians. Accredo has already earned URAC Specialty Pharmacy Accreditation, effective through January 1, 2024.

“Pharmacies and related pharmaceutical services play a critical role in the health care delivery system now more than ever. Given the complicated nature of chronic disease, pharmacies like Accredo do much more than simply fill a prescription. As trusted experts at the frontline of care delivery, they often provide targeted patient support and education to ensure proper medication management,” said URAC President and CEO Shawn Griffin, M.D. “By achieving URAC accreditation, Accredo demonstrates excellence in quality care delivery and their long-term commitment to ensuring patient safety and improving outcomes.”

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