The US Food and Drug Administration have invited pharmaceutical companies to make public the contact details and policy under their Expanded Access programme. This FDA programme enables companies to supply drugs in special circumstances, even though they have not yet received marketing authorisation and may still be under development.
The physician-led request to FDA on behalf of their patient must first confirm that the particular drug manufacturer will supply it under the terms of the Expanded Access programme. Such requests may involve critical care and be time-constrained, so it can be difficult to access all the necessary information.
To facilitate this process, the Reagan-Udall Foundation has launched an on-line Expanded Access Navigator. Using dedicated portals, patients/carers and physicians can learn how the Expanded Access programme works and identify possible treatment options and company contacts who can confirm availability of the drug. To encourage use of this programme, the FDA has asked manufacturers to upload information about their expanded access policies and procedures along with contact details for a rapid response to physicians about specific drugs. You can read more about the Expanded Access Navigator on the FDA blog.
Similar schemes are operated in other countries; Early Access (U.K.), Compassionate Use (EU), Conditional Approval (Japan), Special Access (Australia), with other territories considering their impact and potential. One concern is that the impact could be financially damaging for both the payers (in terms of drug cost and administration burden) and the developers (in terms of return on investment, particularly for smaller companies). If you would like to discuss options for enhances access to medicines, whether focussed on investment, strategy, or procurement, please contact Queen’s House Consulting.