Introduction
The N-of-1+ therapy approach is transforming the drug development landscape and has the potential to facilitate treatment of patients with ultra-rare diseases for whom no previous treatment has been developed. However, while the development of therapies specific to very few patients has a lot of promise, the largest challenge faced by these initiatives is that the drug development, regulatory frameworks and reimbursement systems were not designed for N-of-1+.
Objectives
The overall objective of this Task Force is to connect different N-of-1+ efforts to reduce duplication, achieve global consensus and create a roadmap towards development and implementation of N-of-1+ treatment.
- To produce a reference document summarizing the current state-of-the art, to raise awareness of the N-of-1+ concepts and challenges with all stakeholders
- To identify the major challenges hampering N-of-1+ therapy development and timely patient access, in order to allow for development of proposed solutions and create a better opportunity for strategic planning and delivery of N-of-1 therapies
Timeline
Assembly of the Task Force Group in Q1, 2023.
Members
The TSC is specifically looking for members to contribute to this Task Force with the following qualities and expertise:
- Scientists/Clinicians with experience with N-of-1 treatment development or those working in the ultra-rare field
- Researchers with experience in personalized or core outcome measure development, including digital / software options
- Patient Advocates
- Regulators
- Public or private rare disease research funders
- Industry developers with expertise or interest in N-of-1 treatment, in particular from a platform technology basis
- Those working in ultra-rare disease reimbursement
- Methodologists (including those with experience of statistic modelling in an N-of-1 setting)