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19 Jun 2017 Insight Blog

21st Century Cures Act: Does a public expanded access policy actually mean improved access for patients?

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

The 21st Century Cures Act was signed into law on 13 December 2016 and in accordance with section 3032, it marked the start of a 60 day countdown for pharma and biotech companies developing a phase II or III investigational product to make public their expanded or compassionate access policy via their website. The deadline was 11 February 2017 and, although the US FDA has not yet made it clear if, when and how it will enforce section 3032, we have received interest for support in how to develop a best practice policy, as well as new policies appearing online where they were not previously.

This is encouraging, however the question remains; has the legal requirement for a public expanded access policy in the US actually resulted in improved patient access to early stage medicines, in real terms?

Historically, requesting expanded or compassionate access to experimental medicines has been a complex process, requiring aligned decision-making from cross functional teams within the pharma or biotech company. However, this section of the Cures Act is designed to increase transparency and disclose information on the process and the company’s position on providing access, to better support physicians and their patients. Under the terms of the Act, each expanded access policy must contain:

  • Contact information for requests
  • Procedures for making such requests under the policy
  • Criteria for evaluating and responding to these requests
  • Length of time to acknowledge the request
  • Link or reference to information about the expanded access for a drug.

By making clear who to approach, the company’s process and when to expect an answer, the policy requirements of the Cures Act simplifies how a physician goes about requesting access for an experimental medicine for his or her patient. Having a policy in place also removes “the emotion” from the decision making resulting in an objective, fair, and consistent approach about who may receive access.

From the perspective of pharma and biotech companies, I recently wrote that an ethical compassionate use policy is only possible if the internal access policy is robust and strongly believe that any legislation that can encourage a considered, objective discussion of the ethical, logistical and strategic framework within a company to deal with patient requests is a good thing.

Several aspects of the Act are often misunderstood, for example it does not require every company to have a Managed Access program in place, nor does it assure treatment under an existing program. If, after an intensive, cross-departmental review a decision is made not to allow access to an investigational product to all, or certain patients, the company will have a considered policy in place as to how to respond to such requests.

At Clinigen Consulting we advise companies in evaluating and establishing best practice early access policies. To hear how we worked with BMS to develop theirs, listen to our on-demand webinar, or to learn more about what we do, visit our website for more information. Alternatively, please get in touch at we’d love to hear from you.