PATIENT CONSIDERATIONS FOR OPIOID USE
Opioid use approved FDA to treat certain kinds of acute and chronic pain; also have very serious side effects.
Points to consider by patient:
- Length of treatment
- Reduction of potential side effects
- History of addiction
- Concomitant medications
- Storage, Disposal, Sharing
- Rx for Naloxone
CLINICAL TRIAL ENDPOINTS FOR CANNABIS USE DISORDER (CUD)
ISSUE: 4 M individuals in CUD; projected to increase
ENDPOINT CHOICES: What matters to people with CUD? measurable/standarizable?changes linked to clinical benefits?
FDA TOOLS: Guidances on innovative approaches, Drug Development Tools, Critical Path Innovation Meetings
TARGETED THERAPIES
FOR RARE DISEASES AND DISEASE SUBSETS : Natural History Data, Biomarker Use, Clinical Trial Design
CASE STUDIES: Lumizyme, Carbaglu, Cholbam, Glucarpidase
- Highly plausible mechanistic hypothesis
- Natural history data on untreated patients
- Highly plausible biomarkers
- Serious unmet medical need
- Relatively large treatment effect