One of the strongest challenges SMA UK is bringing to the NICE committee involves the comparison used to judge cost-effectiveness. The treatments are being compared to “Best Supportive Care” (BSC), and we argue this comparison is fundamentally wrong.
Why Comparing Drugs to BSC Doesn’t Make Sense
- Decline is Certain: SMA is an unforgiving, progressive condition. Without disease-modifying therapies, decline is inevitable. BSC focuses on managing symptoms after a person has lost function (e.g., providing a ventilator when a patient can no longer breathe well).
- Preventing vs. Managing: Risdiplam and Spinraza aim to modify the disease at its genetic root, stopping the decline before it happens. BSC only manages the consequences after the damage is done. You can’t compare a treatment that prevents the disaster to one that just helps clean up the aftermath.
The Necessity of Choice
It’s also crucial to maintain a range of treatment options because one size doesn’t fit all:
- Delivery Matters: Spinraza requires a spinal injection every few months, while risdiplam is a liquid taken orally every day. For a patient, their family, or their doctors, the method of delivery can be the defining factor in whether they can access or sustain treatment successfully, different methods work for different people.
- Individualised Results: Some patients respond better to one drug than the other. Maintaining multiple options ensures that specialist clinicians and patients can select the optimal treatment for the best possible outcome.
We urge NICE to recognise that BSC is not a valid baseline.
Maintaining access and choice for these life-changing drugs is non-negotiable.
SMA UK have been working with NICE to bring you all the latest updates on the appraisal of Risdiplam and Spinraza. Read exactly what process NICE will follow starting with the committee meeting on the 11th November here: https://ow.ly/fFxu50Xj2Oq
