Saturday, June 22, 2024

Cost-Effectiveness Scrutiny by NICE Challenges Approval of Biogen’s ALS Treatment

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The United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE) has a reputation for being particularly stringent in its evaluation of the cost-effectiveness of treatments. In recent years, there have been several high-profile cases where companies have been rejected based on their pricing of treatments. In 2021, Bluebird Bio’s treatment, Zynteglo (betibeglogene autotemcel), was rejected by NICE, despite the company agreeing to supply the treatment at a confidential discount. More recently, the institute surprised many by rejecting AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) due to the use of a disease severity modifier threshold.

Biogen recently faced a setback during NICE’s review process. The case involves Qalsody (tofersen), an antisense oligonucleotide licensed from Ionis Pharmaceuticals and used to treat amyotrophic lateral sclerosis (ALS). Last year, the treatment received accelerated approval from the FDA, despite some contention over its clinical efficacy.

In the UK, the contention revolves around NICE’s choice of appraisal route for the drug. The ‘Single Technology Appraisal (STA)’ route makes it less likely for the treatment to be deemed cost-effective, potentially limiting current patients’ access to the treatment in the long term. The STA route is undertaken by NICE when a single, new technology is compared with the standard of care for the indication in question. Its purpose is to appraise the health benefits and costs of those technologies based on clinical and economic evidence, principally provided by the manufacturer or sponsor.

Cost-Effectiveness Debate: Biogen Appeals NICE’s Decision on ALS Drug Qalsody’s Appraisal Route

Biogen pushed for Qalsody to be analyzed under the highly specialized technology (HST) route, which is reserved for drugs that treat very rare conditions and must fulfill six other criteria. Some criteria include that the target patient group is clinically distinct, the condition is chronic and severely disabling, and the technology requires significant national commissioning. The key difference between the two routes is the cost threshold. The STA route has a cost threshold of £20,000 to £30,000 per quality-adjusted life year (QALY), while the HST route increases this budget to £100,000 per QALY.

Biogen applied for the HST route for Qalsody but was designated STA. The company appealed the decision, arguing that since the treatment is for patients with motor neuron disease (MND) who have SOD1 gene alterations, it should be classified as targeting a very small population. The MND Association stated that this subset of patients comprises only 2% of people with MND, estimating this to be between 60-100 individuals in the UK. Products for rare diseases affecting fewer than one in 50,000 people in England are appraised through the HST route.

NICE decided to review Qalsody through the STA route because it did not consider SOD1 MND a ‘clinically distinct disease,’ stated the UK Motor Neuron Disease Clinical Studies Group (UK MND CSG) in an open letter. UK MND CSG responded, “This is an outdated concept when it is recognized that disease classifications based solely on clinical features are inadequate for making precise diagnoses and targeted treatments. It is at variance with the consensus of global neurodegenerative disease experts that SOD1 is a distinct entity with a different pathological basis from the other 98% of MND.”

Cost-Effectiveness

NICE’s Cost-Effectiveness Criteria Likely to Block Approval of Biogen’s Qalsody in the UK

NICE’s decision means it is ‘highly unlikely’ that Qalsody will receive a positive appraisal from NICE, according to UK MND CSG. The open letter indicates that Biogen may not submit an application to the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for approval. This would result in a significant loss for Biogen in the European market and a commercial impact on a product sold in small volumes. In the US, the product is priced at $14,230 per dose, delivered once every 28 days after initial loading doses. Bloomberg estimates peak annual sales of approximately $200 million.

For patients, this means those in England are unlikely to gain access to the treatment beyond current Biogen programs. The company confirmed that the Early Access Program and ongoing Qalsody clinical trials will remain open to eligible patients. However, with NICE’s decision on the route designation being final, another case may emerge where a drugmaker chooses not to market its drug in the country. “Of further concern is the chilling impact this will have on pharmaceutical companies bringing innovative science and clinical trials to the UK, given the perception of no effective route to market for precision medicine or rare disease therapies,” stated UK MND CSG.

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This situation highlights a broader challenge for pharma and payers worldwide. The rise of precision medicine, rare disease treatments, and advanced technologies like gene therapies has led to difficult decisions on access to treatments. As these treatments target fewer people or require fewer doses, the price tags have increased accordingly. Regardless of NICE’s final decision, the debate over pricing and access is likely to continue.

 

Resource: Pharmaphorum, May 22, 2024

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