Comparison between the current methodology and that previously used in 20119
2011 | 2019 | |
Method of drug identification | Thorough and systematic search of online databases (PubMed, ISI Web of Knowledge, Embase, Clinicaltrials.gov and Cochrane MS group). | Calls for recommendations from academics, clinicians and people with MS. Systematic search of online databases by medicines discovery catapult. |
Previous clinical trial use | Previously used in a neurodegenerative disease including progressive MS, PD, HD, AD, and ALS. | Human safety data required only. |
Mechanistic targets | Excluded immunosuppressant mechanism of action. Combination treatments excluded. | Priority given to candidates targeting several mechanistic targets. Excluded those with solely immunosuppressant mechanism. Combination treatments accepted. |
Method of administration | Oral. | Any method of administration. |
CNS penetration | Reviewed at selection meeting. | Evidence of BBB permeability required at study entry. |
Safety | Excluded those with significant adverse effects associated with treatment. | Excluded those with significant adverse effects associated with treatment. |
Method of selection | Systematic evaluation of publications pertaining to each candidate. Systematic review of experimental autoimmune encephalomyelitis preclinical data for each candidate. Scrutiny of each drug by an international multidisciplinary committee. | Systematic evaluation of preclinical and clinical publications pertaining to each candidate. Formation of a database of drug CVs. Rating of these by scientific panel. Presentation and decision at international multidisciplinary meeting. |
Input from people affected by MS | Patient representatives acting as external advisors. | 6 members of MS research network on voting panel. Scoring of drug CVs by at least five people with, or affected by, MS. Members of MS research network at treatment selection meeting. |
Peer review | External advisors with a range of expertise including animal models, disease biology, clinical trial design, systematic review and patient representation. | Methodology and final treatment selection sent for external peer review. |
AD, Alzheimer’s disease; ALS, amyotrophic-lateral sclerosis; BBB, blood–brain barrier; CNS, central nervous system; HD, Huntington’s disease; MS, multiple sclerosis; PD, Parkinson’s disease.