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01 Dec 2016 Insight Blog

21st Century Cures Act – are you ready?

By Romina Oxborough, PhD, MBA, Director of Clinigen Consulting & Vice-President HBA UK

The US 21st Century Cures Act has been enacted on 13 December 2016 when the US President signed it into law. There are many implications of the act across a multitude of elements of how patients access medicines, however there is a significant change afoot for how companies who provide access to medications via compassionate use/expanded access devise and publicise their policies externally.

A recent survey found that just 19 of 100 pharma2 currently provide a publically available early access policy online, I therefore wonder how many companies are ready to comply with these requirements of the Act.

First of all, here is a summary of the obligations with regards to Expanded Access

  • Manufacturer or distributor of one or more investigational drugs shall make policy on evaluating and responding to requests
  • The policies shall be made publicly available (e.g. via a website)
  • The policy to include: contact information for requests, procedures for making such requests, criteria for evaluating these requests, length of time to acknowledge the request, link or reference to information about the expanded access for a drug.
  • They clarify that the posting of a Policy does not guarantee access.

With just 60 calendar days from the enactment of the Act or the first initiation of a phase 2 or 3 study (as defined by CFR) - whichever is later - until policies should be available, the clock will be ticking for the 81% of companies who have so far kept their policies private, or who do not have a formal policy in place at all.

This means that if you have a product in development, you need to start thinking about your policy now. Defining the criteria for evaluation of the requests, the procedure and length time to acknowledge such request will need some internal discussion as you need to define who the decision-makers are.

I am frequently asked why I focus on a US-only legislation. The first reason is that many pharma companies will adopt a global policy instead of developing regional ones – and I am a big advocate of centralising the management of your unlicensed access around the world for a number of reasons (which I’ll describe in a future blog). The second reason is that I expect other country local regulators to adopt a similar legislation in the near future.

Please contact Clinigen Consulting ( if you’d like to know more about our Policy Development work or listen to a webinar in which Sue Hannah of Bristol-Myers Squibb presented an example of the work we did for them in setting up a best practice expanded access policy.