How do new drugs for rare diseases enter medical insurance?

Scotland's latest decision to introduce a new oral drug for spinal muscular atrophy into the national health system

Whether medical insurance can include the latest new drugs for rare disease treatment is a world problem

No, this is the approach taken when a new oral drug entered Medicare Scotland

At the time, the Scottish health insurance payer SMC opposed the inclusion of the new drug in the National Health Service (NHS)

In Scotland, the NHS is equivalent to the country's public hospitals, and access to this system could make new medicines available to patients at low cost or even free of charge

When this new drug, Risdiplam, listed in Chinese as Evrysdi, was in front of the Scottish Medicines Consortium, the Scottish medical cost watchdog, the medical insurance review agency responded, "NO"

This is not surprising, as new drugs are inherently expensive, especially for rare diseases

Evrysdi is approved for the treatment of a rare form of spinal muscular atrophy (SMA) caused by a genetic defect

Evrysdi is the first oral treatment for spinal muscular atrophy, providing a new treatment option for this devastating disease that can be taken without leaving home

How can Scotland's health insurance payer, SMC, not only see the price of new drugs, but also the benefits they bring to patients?

Risdiplam was jointly developed and marketed by Swiss pharmaceutical company Roche and PTC Therapeutics

It is understood that Evrysdi is priced at US$11,671 in an 80ml dosage form (0.

In China, the retail price of 60mg/bottle is 63,800 yuan, and the annual treatment cost is between 800,000 and 2 million yuan

Here's what Roche did in changing the views of Medicare Scotland:

01.

NICE, a health care cost watchdog, has veto power over whether new drugs can enter the National Health Service
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Patients in England, Wales and Northern Ireland can receive treatment from Roche at special, confidential health insurance rates at low or no cost through a three-year managed access scheme
.

In Scotland, patients will then be able to access treatment first through the UK-wide Early Access scheme, which launched in September 2020, and will receive either low-cost or free Evrysdi treatment directly from the NHS after March this year
.

Meanwhile, SMA UK, the organization for patients with spinal muscular atrophy, announced that SMC will provide low-cost or no-cost medical care to patients with spinal muscular atrophy (SMA) who are eligible to take Evrysdi
.

These eligible patients included: 2 months of age and older, clinically diagnosed with SMA type 1, 2 or 3, or 5q SMA with 1 to 4 copies of SMN2 (survival of motor neuron 2)
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Evrysdi works by increasing levels of neuromotor neurons (SMNs)
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SMN is a protein critical for motor neuron and muscle health, however, SMA patients are unable to produce normal SMN
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Evrysdi is an oral formulation that is administered once daily at home by mouth or feeding tube to complete treatment
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The safety and efficacy of the therapy were established based on data from the ongoing Phase 2/3 Firefish FIREFISH (NCT02913482) and Sunfish SUNFISH (NCT02908685) trials
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The trial involved a total of 221 patients with SMA types 1, 2 and 3 aged 1 month to 25 years
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RESULTS: Evrysdi safely and significantly improved survival, breathing, swallowing, and motor development measures in infants with SMA type 1, and improved motor function in children and young adults with SMA types 2 and 3
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Evrysdi's clinical program also includes two global Phase 2 trials testing the therapy in different patient populations - JEWELFISH (NCT03032172) and RAINBOWFISH (NCT03779334)
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Interim data from these studies showed that presymptomatic infants given Evrysdi for at least a year achieved the same markers of motor development as healthy children
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In contrast, patients under 60 who had used other disease-modifying treatments more than doubled their SMN levels and showed stable exercise capacity after using Evrysdi
.

The RAINBOWFISH trial is still enrolling presymptomatic neonates at 6 weeks of age with a genetic diagnosis of SMA
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At the same time, based on the favorable interim findings of the RAINBOWFISH trial, Roche is submitting a priority review application to the regulatory agency in the United States, requesting that the use of the therapy be expanded to the treatment of infants under 2 months of age before SMA symptoms appear
.

Reference article:

https:// https://smanewstoday.
com/news-posts/2022/02/11/evrysdi-available-soon-eligible-sma-patients-via-scotland-nhs/

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