The International Alliance of ALS/MND Associations encourages regulatory agencies around the world to develop a framework for global alignment in evaluating potential new therapies for people living with ALS/MND.
The Alliance believes that the ALS/MND community and all stakeholders will benefit from a more cooperative global approach to weighing the risks and benefits of new therapies, especially because drug development and clinical trials take place across many countries. People living with ALS/MND, wherever they live, have an extraordinary unmet need and want the fastest and widest possible access to therapies that may help them. Given how devastating ALS/MND is, this community cannot wait through excessive procedural delays caused by sequential reviews and hand-offs between regulators.
Pivotal (late-stage) clinical trials in ALS/MND are often conducted in several countries, and these global trials are increasingly important for testing the safety and effectiveness of ALS/MND drugs for approval. Drug development may benefit from more uniform global standards of treatment, which can lead to better trial designs.
The Alliance calls for a cooperative, harmonized pathway to approval that makes the evaluation of ALS/MND treatments more effective and faster, guided by the following principles.
Principles
- The perspectives, expertise, and experiences of people living with ALS/MND, their caregivers, and their health care providers, including their views on benefits and risks, should be at the core of clinical development and global regulatory review discussions.
- Regulators in each country and region align on regulatory review, with the purpose of driving outcomes.
- Regulators develop harmonized guidance and process pathways that encourage sponsors to engage with regulators before submitting applications for ALS/MND products, and to submit those applications to participating countries for concurrent review.
- Regulators establish ongoing, regular interaction to share information and engage with each other.
- The stakeholder community, working with regulators, develops objective measures of progress and uses them in reporting.
- Each regulatory authority identifies a Global Regulatory ALS/MND Lead.
- A global lead convener role is established, with responsibility that rotates among regulators.
- Regulators are transparent with all stakeholders in the ALS/MND community.
Read the full report
These principles were developed at a roundtable on October 5, 2021, that brought together people living with ALS/MND, caregivers, ALS/MND organizations, and industry representatives from more than a dozen countries.
Read the Regulatory Pathways Roundtable Report (PDF, 11 pages) →