The rare disease markets in the United States are very lucrative and worth billions, especially over the past ten years.

To make an impact from a commercial perspective the agents need to demonstrate a significant impact beyond their generic alternatives; in terms of overall endpoint efficacy or the potential reverse of the course of disease.

This would primarily mean targeted gene therapy and from a Phase 2 and 3 perspective the data needs to be designed to show a separation from placebo with efficacy. 

This means the Clinical Research Organization (CRO) needs to be highly effective and very much aligned with the life science company for end results.

There have been two recent gene therapy start-up companies who have devastating study results because of CRO alignment. 

Some other factors to cover, if introduced in the United States, have there been any Phase 2 or 3 studies in US locations? If not, it could cause delay as there can sometimes be disagreement on outside US study veracity.

Other factors, include physical access to the clinicians involved with care; many institutional accounts have set policy against company access. The majority of rare disease providers are in these accounts.

Then, there is current legislation such as, the Inflation Reduction Act and its direct involvement in controlling Medicare prices. 

Not to mention the many state Medicaid plans and how to best position a product for reimbursement 

These are the top-level considerations for introduction of a rare disease product in the United States.  

There are many other details for a successful product launch and we can provide this guidance to you as requested.