Zitter Pulse Check: Orphan Disease


Orphan disease is a high-cost category with an increasing number of therapies that treat the conditions. To help stakeholders absorb datapoints and perceptions from all directions and synthesize those insights into a tangible strategy for orphan diseases, Zitter Insights surveys a leading panel of pharmacy and therapeutics (P&T) decision makers at payers and integrated delivery networks. The Managed Care Biologics and Injectables Index combines deep payer insights with the industry standard in market access information to produce quarterly reports and insights summaries on leading specialty brands.

Payer Coverage

In the U.S., orphan diseases are conditions impacting fewer than 200,000 people. There are more than 7,000 of these rare conditions affecting an estimated 30 million Americans, and new diseases continue to be discovered. Commercial payers managing nearly two-thirds of covered lives said they will solicit greater physician insight on orphan drug management (see chart). In 2022, payers with more than two-thirds of lives expect to increase their parity preferred contracting among two or three agents.

Surveys were collected 2/25/2021 – 4/2/2021

Payers that manage almost half of Medicare lives in 2021 are likely to increase their contracting for sole preferred treatments. That percentage is expected to rise to almost two-thirds of Medicare lives in 2022. Payers representing almost half of Medicare lives said they will solicit greater physician insight on orphan drug management.

Key Findings

Ideal Growth Hormones

Commercial payers with almost half of covered lives said Pfizer Inc.’s Genotropin (somatropin) is their ideal first-line therapy for growth hormone deficiency (see chart); however, payers covering almost three-fourths of Medicare lives said it’s their No. 1 first-line agent. Commercial payers representing almost two-fifths of lives cited Novo Nordisk Inc.’s Norditropin (somatropin) as their top second-line therapy, followed by Roche Group member Genentech USA, Inc.’s Nutropin AQ (somatropin), which was named by payers with almost one-third of lives. Among endocrinologists, more than 90% had prescribed Eli Lilly and Co.’s Humatrope (somatropin) in the 12 months prior to the survey, followed by Genotropin and Norditropin, both prescribed by more than four-fifths of respondents.

Surveys were collected 2/25/2021 – 4/2/2021

Short- vs. Long-Acting Growth Hormones

In 2020 and 2021, the FDA approved the first two long-acting growth hormones: Novo Nordisk’s Sogroya (somapacitan-beco), approved Aug. 28, 2020, and Ascendis Pharma A/S’s Skytrofa (lonapegsomatropin-tcgd), approved Aug. 25, 2021. These agents have weekly dosing compared with daily dosing for the seven short-acting therapies. Approximately four-fifths of endocrinologists said they will prescribe long-acting growth hormones to patients new to therapy, and more than two-thirds said they will switch certain patients from their current therapy to a long-acting one. Payers with almost two-fifths of covered lives said they will manage the long-acting treatments separately from the short-acting ones.

Preferred HAE Agents

The FDA has approved eight agents to treat hereditary angioedema — four for routine prophylaxis against HAE attacks and four to treat acute attacks. Among the prophylactic therapies, more than half of providers cited Takeda Pharmaceutical Company Ltd.’s Cinryze (C1 esterase inhibitor [human]} as their most ideal first-line therapy. For their top second-line treatment, more than two-thirds chose CSL Behring LLC’s Haegarda (C1 esterase inhibitor [human]). The most ideal acute HAE agent was CSL Behring’s Berinert (C1 esterase inhibitor [human]), named by more than half of providers. Respondents were split evenly among three therapies for their preferred second-line drug: Firazyr (icatibant) and Kalbitor (ecallantide) — both from Takeda — and Ruconest (C1 esterase inhibitor [human]) from Pharming Healthcare, Inc.


Mechanism of Action

Message: “Pfizer presented clinical on gene therapy for hemophilia B and fidanacogene eleparvovec. Reviewed MOA, which is an adeno-associated virus (AAV) capsid and a high-activity human coagulation factor IX gene transporter. Both the capsid and the replacement gene are proprietary. The virus vector targets the liver and may not be a cure but could improve a patient from severe to mild. In clinical trials, it ranged from 18% to 80% increases in factor IX levels. In the Phase 1/2 clinical trial in severe or moderately severe hemophilia B, all 15 patients had discontinued routine infusions of factor IX concentrates, and only one patient infused prophylactically during the study period. Phase 3 open-label, multi-center, lead-in study to evaluate the investigational gene therapy is currently enrolled. This model will use the same hemophilia patients and track them to determine their ABR and then admin the gene therapy to see if there is a clinically significant improvement.”

Payer Thoughts: “A little disappointed the MSL couldn’t share any dates but said this is more than two years out.”

FDA Approves First Oral Therapy For Prevention of HAE Attacks

When the FDA approved BioCryst Pharmaceuticals, Inc.’s Orladeyo (berotralstat) last month, the drug became the first oral treatment for prophylaxis to prevent hereditary angioedema (HAE) attacks. According to Zitter Insights, payers with nearly three-quarters of covered lives plan to manage it at parity to other prophylactic treatments.

Subscribers to AIS’s RADAR on Specialty Pharmacy may read the in-depth article online

FDA Approval of Sogroya May Change Prescribing in Growth Hormone Class

When the FDA approved Novo Nordisk, Inc.’s Sogroya (somapacitan-beco) for the replacement of growth hormone in adults with growth hormone deficiency on Aug. 28, 2020, it became the first long-acting agent that the agency had approved. A survey by Zitter Insights shows that many endocrinologists expect to shift prescribing to such agents from the short-acting growth hormones. And many payers say they expect to manage at least one long-acting agent at parity with short-acting growth hormones.

Subscribers to AIS’s RADAR on Specialty Pharmacy may read the in-depth article online

© 2024 MMIT
AIS Health Staff

AIS Health Staff

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