MY APPROACH
Where I focus
Rare disease and gene therapy programs rarely fail on the science. They fail on the decisions made around it, and nobody finds out until after approval.
Clinical strategy ↓
Post-approval readiness ↓
Expanded access ↓
Before approval ↓
When things go sideways ↓
Clinical strategy
Endpoints, comparators, trial design, evidence gaps, and what each choice will cost you three years from now. Heaviest pre-clinical through Phase II.
Post-approval readiness
Post-marketing requirements, long-term follow-up, registries, medical readiness, and the evidence you should already be collecting. Heaviest Phase II onward.
Expanded access
Program design, operational oversight, and identifying which data can be used for regulatory purposes, which depends on how it was structured before it was collected.
Before approval, while it is still cheap
• A primary endpoint that will not support the label you are building the company around.
• Natural history data you needed to start collecting three years ago.
• An expanded access program generating data you will not be permitted to use.
• A post-marketing commitment you are about to agree to and cannot staff.
• Sites and countries chosen for enrollment speed that will not survive the registrational plan.
On the day everything goes sideways
• A clinical hold, and a leadership team that has never been through one.
• A study that read out and did not answer the question you needed answered.
• A CRO relationship that has stopped working in the middle of the trial.
• A post-approval commitment slipping, and a regulator asking why.
• A board meeting in eleven days and nobody in the building who has done this before.
Case Studies
Coming soon.
Key case studies and examples of my work supporting rare disease programs will be added here.
