AJMC Staff

The FDA has approved risdiplam, the first at-home, oral treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older.

The drug, created by PTC Therapeutics, will be marketed by Genentech as Evrysdi.

The approval was based on “clinically meaningful improvements in motor function and obtainment of developmental milestones,” the companies said.

“Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease,” Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development for Genentech, 

The approval is based on results from 2 clinical studies with more than 450 participants: FIREFISH in symptomatic infants aged 2 to 7 months; and SUNFISH, in children and adults aged 2 to 25 years, which is the first and only placebo-controlled trial to include adults with types 2 and 3 SMA.

Patients previously treated for SMA with other medications were also included.

Other medications for SMA include Biogen’s nusinersen (Spinraza) and Novartis’ onasemnogen abeparvovec (Zolgensma), with the latter being a 1-time gene therapy.

“Infants achieved key motor milestones not normally seen in the natural course of the disease, such as the ability to sit without support. Evrysdi also preserved vital functions and improved survival at 12 months. In particular, Evrysdi improved survival without permanent ventilation compared to natural history when measured at 12 and 23 months,” PTC said.

"Today marks an incredibly important moment for the broader SMA patient community that had been in dire need of safe and effective treatment options," Stuart W. Peltz, PhD, CEO of PTC Therapeutics, 

In the FIREFISH study, 41% of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds, as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Ninety percent of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older, according to the study.

In the SUNFISH study, children and adults treated with risdiplam experienced a clinically meaningful and statistical-significant improvement in motor function at 12 months (1.55 point mean difference; 

 = .0156) compared with placebo (1.36 points [95% CI, 0.61-2.11]; –0.19 points [95% CI, –1.22-0.84], respectively), as measured by a change from baseline in the Motor Function Measure-32 total score.

Risdiplam will be available in the United States within 2 weeks for direct delivery to patients' homes through Accredo Health Group, an Express Scripts specialty pharmacy.

Articles

The FDA has approved risdiplam, the first at-home, oral treatment for spinal muscular atrophy.

FDA Approves At-Home Oral Treatment for Spinal Muscular Atrophy

 cited a study published in the July 2020 issue of 

The Escalation of the Opioid Epidemic Due to COVID-19 and Resulting Lessons About Treatment Alternatives

.” The study indicated that investing in modalities that promote building emotional resiliency and help address the factors that made patients susceptible to opioid reliance in the first place are key to addressing the growing opioid epidemic amid the pandemic.

 spotlighted a study published in the June 2020 issue of 

Value-Based Insurance Design in Louisiana: Blue Cross Blue Shield’s Zero Dollar Co-pay Program

,” found that enrollment in a value-based insurance design program that eliminated pharmacy co-pays for 4 chronic disease drug classes was associated with a large decline in health care spending.

Current Value-Based Care Models Need Greater Emphasis on Specialty Care

,” provided an overview of the impact of specialty care and the opportunity for it to leapfrog primary care as a lead focus for accountable care.

Coverage of our peer-reviewed research and news reporting in the health care and mainstream press.

AJMC® in the Press, August 7, 2020

Talks on New COVID-19 Relief Package Stagnant

 an all-time high at 1.186 million amid the coronavirus disease 2019 (COVID-19) pandemic, negotiations between the White House and Democratic congressional leaders on a new COVID-19 relief package were reported to be on the brink of failure. According to 

, the lack of progress in the high-level talks may spur action from President Donald Trump, who said he would issue a series of executive orders to tackle the economic crisis if no deal can be reached. According to senior administration officials, Trump could issue these orders as early as today.

As more than 100 COVID-19 vaccine candidates continue to be examined in clinical trials, some hints on pricing have emerged among those leading in progress, according to 

. Moderna, whose vaccine known as mRNA-1273 is now in a phase 3 clinical trial, has already made deals at between $32 and $37 per dose with some foreign countries. This has eased some concern among consumer advocates that US taxpayers may not reach fair deals, as Moderna said these indicated prices reflected agreements for smaller volumes and the price would decrease for larger-volume agreements. However, some advocates are still worried, as the US government has invested millions of dollars in developing the vaccine.

“Do Not Travel” COVID-19 Warning Rescinded by Trump Administration

Yesterday, the Trump administration removed its warning against all international travel to Americans amid COVID-19, saying conditions no longer warrant a worldwide alert. As reported by 

, the State Department’s decision to lift its level 4 health advisory worldwide came shortly after the CDC revised its COVID-19 travel advisory information to focus on 20 countries of concern. Similar to the CDC, the State Department will now issue country-specific warnings, which can be accessed 

Negotiations on a new coronavirus disease 2019 (COVID-19) relief package are reportedly on the brink of failure; as more than 100 COVID-19 vaccine candidates continue to be examined in clinical trials, some hints on pricing have emerged; the Trump administration removed its warnings on all international travel to Americans amid COVID-19.

What We're Reading: Relief Package Talks Stagnant; Potential COVID-19 Vaccine Prices; International Travel Warning Rescinded

CMS releases updates on evaluation and management services, telehealth, and accountable care organizations; experts highlight COVID-19 vaccine developments and remaining challenges; and a study examines how state policies may impact life expectancy.

Welcome to This Week in Managed Care, I’m Matthew Gavidia.

CMS Releases E&M Updates, Expands Telehealth, Makes ACO Changes

Among a swath of proposed policy and payment changes announced this week by the Trump administration, CMS continued its effort to simplify coding requirements, quality measurements, and focus on 340B drug costs.

Tuesday, CMS released the proposed calendar year 2021 Physician Fee Schedule, which includes Quality Payment Program changes affecting accountable care organizations and how evaluation and management, or E&M, services are billed, such as examinations, disease diagnosis, risk assessment, and care coordination.

Explaining some of the raft of changes Tuesday, CMS Administrator Seema Verma said E&M codes have not been updated in 2 decades and do not reflect the growing number of chronic conditions and medically complex patients or social determinants of health.

“The value of E&M has decreased as complexity has increased,” said Verma.

In the 340B drug program, CMS would, in 2021, cut the payment rate for drugs to the average sales price minus 28.7%.

CMS said the change would save Medicare beneficiaries an additional $85 million in out-of-pocket payments for these drugs and that the savings would be reallocated among all hospitals on an equal percentage basis, while exempting certain cancer hospitals, rural sole community hospitals, and children’s hospitals.

Experts Highlight COVID-19 Vaccine Developments and Remaining Challenges

Of the more than 160 COVID-19 vaccine candidates being examined in clinical trials, those that are leading the pack are utilizing a new vaccine technology that has never been approved for human use by the FDA, which has contributed to significant unknowns.

In a free, 1-hour webinar from MJH Life Sciences™, the parent company of 

®, 3 top experts discussed vaccine development, the latest clinical trial evidence, and the future of COVID-19 vaccines.

Missed the webinar? You can still register for free and watch the video on demand!

For more coverage of the webinar and to register, 

How Do State Policies Impact Life Expectancy?

, a study found that since 1970, changes in US state policies have played an important role in the stagnation and recent decline in life expectancy, while some policies appear to be key levers for improving life expectancy.

In 2016, life expectancy of American women was 3 years below the female average of high-income countries and 5.8 years below the leading country for life expectancy, with US men also having

shorter life expectancies at 3.4 years below the average and 5.2 years below the leader.

When it comes to root causes of these disparities, one major contributor is a rise in so-called “deaths of despair,” or deaths due to suicide or drug and alcohol abuse. Different regional policies on firearm use and availability of substance abuse rehabilitation programs can alter expectancy gains or declines across state lines.

Local policies were found to affect nearly every aspect of an individual’s life including economic well-being, social relationships, education, housing, lifestyles, and access to medical care, authors point out.

If the status quo is maintained, thereby allowing the current policy direction of states to continue, researchers found the results would yield minimal improvements in longevity. However, authors caution the analyses do not prove state policies have a causal effect on life expectancies.

“Taking these findings together, the slow gains in US longevity may partly reflect the national shift toward some conservative policies that are negatively associated with longevity, such as abortion restrictions, reductions in gun control,” researchers conclude.

Tafasitamab Combination Approved for Adults With R/R DLBCL

Last Friday, the FDA approved tafasitamab to be used in combination with lenalidomide for second-line treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, or DLBCL, meeting the needs of patients who are not eligible for an autologous stem cell transplant.

Approval was based on phase 2 results for 80 patients in the L-MIND trial, which reported an update May 2019 at the American Society of Clinical Oncology,

 with final results published last month in 

FDA showed an overall response rate of 55%, including a compete response rate of 37% and a partial response rate of 18%.

Among non-Hodgkin lymphomas, DLBCL is the most common subtype, accounting for 22% of cases in the United States and 40% worldwide, with about 18,000 US cases per year. Tafasitamab, to be sold as Monjuvi by MorphoSys and Incyte, is a humanized Fc-modified cytolytic CD19 monoclonal antibody being studied in several B-cell malignancies.

“The FDA approval of Monjuvi in combination with lenalidomide helps address an urgent unmet medical need for patients with relapsed or refractory DLBCL in the United States,” said Hervé Hoppenot, CEO of Incyte, in a statement.

And, now our Paper of the Week, which looks back at some of the most important papers over the past 25 years of 

News about updates in E&M codes brings us back to an important 2014 paper from Dr. Richard Young and Tiffany Overton, whose review of data showed that billing codes did not fully reflect the scope of work done by family physicians, and contributed significantly to income disparities between these critical clinicians and specialists.

The disconnect between billing codes and actual work performed revealed a gap not only in income but in the way the physicians’ work is valued.

®, I’m Matthew Gavidia. Thanks for joining us!

Videos

This week, the top managed care news included CMS updates on telehealth and more; COVID-19 vaccine developments and remaining challenges; a study examines the impact of state policies on life expectancy.

Author | AJMC Staff

Articles

FDA Approves At-Home Oral Treatment for Spinal Muscular Atrophy

AJMC® in the Press, August 7, 2020

What We're Reading: Relief Package Talks Stagnant; Potential COVID-19 Vaccine Prices; International Travel Warning Rescinded

This Week in Managed Care: August 7, 2020