Executive Summary Project Name: Advanced clinical fellowship in neuromuscular disease with focus on hereditary transthyretin amyloidosis service development

Joint working between (1) the National Hospital for Neurology and Neurosurgery (NHNN), (2) the National Amyloidosis Centre (NAC) and (3) Alnylam UK Ltd (Alnylam) in relation to the development and implementation of a multidisciplinary team and service evaluation Start Date and Duration: 15 November 2024 to 31 March 2026

Project Summary: As part of the existing clinical process, patients diagnosed with hereditary transthyretin-mediated amyloidosis (hATTR) and with suspected peripheral neuropathy (Patients) at the NAC are referred by their managing health professional to the NHNN. The joint working project will develop and implement a process to triage and then assess these Patients at a multidisciplinary team meeting. The project will also include an evaluation of the service pathway for diagnosis and treatment of hATTR in the UK (Service Evaluation).

Expected Benefits: Anticipated benefits for Patients:

  • Fewer but more effective and directed visits to hospitals.
  • Better information on their condition and treatment options due to assessment being undertaken by experts in hATTR and peripheral neuropathy.
  • Patient assessment is co-ordinated and more appropriately based on clinical need than under the current system. This is turn would provide patients with a better experience of the healthcare system.
  • The development of a procedure to support earlier diagnosis of patients with hATTR.

Anticipated Benefits for NHNN:

  • Opportunity to advance clinical understanding of hATTR and peripheral neuropathy alongside the specialist expertise at the NAC through discussion and evaluation of Patient cases.
  • Opportunity to increase the capacity of the NHNN to assess Patients based on clinical need within a shorter timeframe.
  • Opportunity to improve the service for hATTR patients across neurology/neuropathy care including at the NHNN.

If deemed necessary, provide data to support a business case to be shared with relevant decision-making body agreed by the Parties to improve the provision of neurology/peripheral nerve clinical input into the care of patients with hATTR under the care of the NAC.

Anticipated Benefits for the NAC:

  • Opportunity to advance clinical understanding of hATTR and peripheral neuropathy alongside the specialist expertise at the NHNN through discussion and evaluation of Patient cases.
  • If deemed necessary, provide data to support a business case to be shared with NHS England (or other relevant decision-making body agreed by the Parties) to improve the provision of neurology/peripheral nerve clinical input into the care of patients with hATTR under the care of the NAC.
  • Service Evaluation will provide data to the NAC which may be useful for its operations. Anticipated Benefits for Alnylam: o Alnylam is expected to benefit through the potential earlier diagnosis of hATTR patients with polyneuropathy.
  • Improvement in patient care through alignment with local or national guidelines.

Anticipated Benefits for Alnylam:

  1. Alnylam is expected to benefit through the potential earlier diagnosis of hATTR patients with polyneuropathy.
  2. Improvement in patient care through alignment with local or national guidelines.