Cost-effective Next-in-Class Medicines: Accelerated Access Mechanisms

In January 2026, the National Institute for Health and Care Excellence (NICE) published guideline TA1121, recommending acoramidis, a transthyretin stabilizer developed by Bayer, for routine use in the NHS for the treatment of a rare heart disease. Of particular interest is NICE's approach to technology assessment, funding, and implementation.

Disease

Transthyretin amyloidosis with cardiac involvement is a rare, progressive disease caused by amyloid deposition in the myocardium due to instability of the transthyretin protein. The disease leads to restrictive cardiomyopathy and heart failure; hereditary and sporadic (wild-type) forms are distinguished.

Current Therapy Approaches

Tafamidis (Vyndaqel®, Pfizer), a transthyretin stabilizer that has been shown to reduce mortality and hospitalization rates, remains the standard of treatment.

New Drug

ACORAMIDIS belongs to the class of transthyretin stabilizers and is indicated for the treatment of adult patients with hereditary and sporadic transthyretin amyloidosis with cardiac involvement. Within its therapeutic class, the drug is considered both a Next-in-Class and a therapeutic alternative to the existing standard of care (tafamidis) for the same target patient population.

Technology Assessment Approach

TA1121 was assessed by NICE using a cost-comparison approach. The regulator assumed the likely clinical comparability of acoramidis and tafamidis and focused on cost comparisons without calculating an incremental cost-effectiveness ratio (ICER).

The results demonstrated comparable or lower costs with acoramidis, which formed the basis for the recommendation.

• The practical consequence of this approach is the entrenched principle of intra-class choice: when there are several clinically appropriate therapeutic options, priority is given to the least expensive one, which creates a systemic focus on using the more cost-effective option within a therapeutic class.

Access and Implementation Mechanism

The NICE recommendation is subject to the use of the simple discount Patient Access Scheme (PAS)—a commercial scheme offering a discount off the list price for National Health Service England (NHS); the discount parameters are confidential. Access conditions for NHS organizations are implemented through the Commercial Access and Pricing (CAP) Portal; for non-NHS organizations, interaction with the manufacturer is envisaged to clarify the conditions.

Since the recommendation was issued through a cost-comparison process, the drug is subject to accelerated implementation and must be available to patients within 30 days of publication of the final NICE guidance.

The example of TA1121 demonstrates that, when clinically comparable to standard therapy, more cost-effective Next-in-Class drugs can receive accelerated access through simplified assessment procedures and simple pricing agreements.

For CIS countries, the development of similar mechanisms for including such drugs in drug lists and programs is relevant, which allows for increasing the cost-effectiveness of the healthcare system while maintaining therapeutic results.

Source: NICE final guideline TA1121 (January 2026 (https://www.nice.org.uk/guidance/ta1121))