Zitter Pulse Check: Orphan Drugs/Spinal Muscular Atrophy (June 2022)


Orphan drugs 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 almost all covered lives said that management of orphan drug costs through the use of specialty pharmacies was their top method in first-quarter 2022 followed by the addition of site-of-care restrictions for physician-administered therapies (see chart). Respondents said that by the first quarter of 2023, they expected that the most common orphan drug management tactic would be stratifying drugs within a class by their characteristics in order to create separate market baskets.


Among Medicare payers, implementing site-of-care restrictions for physician-administered drugs was the No. 1 management tactic in the first quarter of 2022, with using specialty pharmacies to manage costs in the second spot. Respondents projected that the No. 3 strategy for that time frame — increasing contracting for parity preferred status among two or three therapies — would be the top approach by the first quarter of 2023.

Key Findings

Orphan Drug Payer Priorities

Multiple sclerosis was chosen by nearly all commercial payers as their most prioritized orphan drug category to manage due to its high costs. That was followed by growth hormone deficiency for its multiple treatment options, cystic fibrosis for its large patient population, pulmonary arterial hypertension for its contracting opportunities and hemophilia for its similar therapeutic choices.

SMA Payer Management

Respondents covering almost all commercial lives require prior authorization for all spinal muscular atrophy (SMA) treatments: Biogen’s Spinraza (nusinersen), Novartis Pharmaceuticals Corp. subsidiary AveXis, Inc.’s Zolgensma (onasemnogene abeparvovec-xioi) and Roche Group member Genentech USA, Inc.’ Evrysdi (risdiplam). Among the payers that require this process for only certain SMA agents, most have prior authorization in place for Zolgensma, and some mandate it for Evrysdi. Payers with almost half of the lives do not have step edits in place for SMA agents (see chart). For those that do, requiring a step through Spinraza before Zolgensma for certain patients is the most common strategy. Least common is stepping though Evrysdi before Zolgensma. Respondents representing Medicare lives had similar responses.


Newborn Screening

In July 2018, SMA was added to the list of suggested conditions that states should screen for in their universal newborn screening programs, known as the Recommended Uniform Screening Panel. According to the Centers for Disease Control and Prevention, routine newborn screening conducted by state public health laboratories can detect genetic disorders including SMA in dried blood spots collected from a baby’s heel within the first 24 to 48 hours after birth. The test can show that a baby likely has SMA, and then additional tests can confirm the diagnosis. For pre-symptomatic patients with SMA that is identified through a newborn screening program, payers with about half of covered lives provide full coverage, while those with slightly fewer lives do not provide coverage and said they do not plan to change this policy in the near future.


Prior Authorization

Message: “Live meeting at AMCP conference to continue discussion of our PA policy and to go over some newly released clinical data on non-symptomatic patients for Zolgensma. MSL first went over clinical data, highlighting the effectiveness of Zolgensma on non-symptomatic patients and explaining that SMA is now part of the vast majority of states’ newborn screening panel. This presentation led over to AM that we change PA policy to allow for non-symptomatic patients and also consider opening up criteria to all versions of SMA type. CMO from our side explained that the new data is currently being reviewed, and a decision will be made soon. The other question from AM and Novartis team on the PA was the requirement that patients currently receiving treatment on Spinraza or Evrysdi show signs of deterioration before allowance of Zolgensma.”

Payer Thoughts: “This is obviously an important drug and one that should be administered as quickly as possible to the right patients. We are reviewing the data internally, and I expect a potential change to the policy post the May P&T. The other question that came up in this discussion surrounded the over 100 sites where Zolgensma can be administered. Do more sites lead to more confusion, or some sites navigating PA/paperwork better than others, and maybe delaying treatment? Should the number of sites be reduced? These are questions that our CMO will follow up with Novartis executives at the upcoming Cell and Gene Therapy conference in Boston in early May.”

Recently Approved Evrysdi May Have Big Impact on SMA Class

The spinal muscular atrophy (SMA) therapeutic category continues to expand with the Aug. 7, 2020, FDA approval of Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. Industry experts maintain that the drug has the potential to significantly impact the class for a variety of reasons, including its route of administration and price.

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

Study: ‘Drug Super Spender’ Members Doubled in Four Years

The United States is seeing a surge of innovative specialty therapeutics coming onto the market, many of which offer potential treatments for conditions that previously didn’t have one. But many of these newer therapies, including ones for spinal muscular atrophy, are launching with expensive price tags, driving up spending on health care services. A Prime Therapeutics LLC study found a huge increase in people who have annual drug spend of at least $250,000 — so-called “drug super spenders” — from 2016 to 2019.

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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