It’s important for pharma and biotech companies to think about how various stakeholders interact with publicly available information about the company’s medicines in development. Patients around the world have never had more availability to information about their serious or life-threatening disease and treatments available, whether already commercialized somewhere, or globally unlicensed. They may be desperately seeking information for themselves or for a loved one.
It is a fine line to walk, but most pharma and biotech companies of all sizes can do more to help manage expectations of patients and patient advocates. When information on their website is non-existent or incomplete, companies may find themselves receiving inbound emails and phone calls from patients. Companies normally have good systems to provide responses to physicians, but typically have no mechanism to respond to patients. Having a publicly posted expanded access policy can help fill this information gap by informing and managing expectations of patients and patient advocates.
The 21st Century Cures Act and Its Impact on Expanded Access
The 21st Century Cures Act (Cures Act) was signed into law in December 2016 in the United States (US) and was designed to help accelerate medical product development and bring new innovations and advances to patients who need them faster and more efficiently. One provision in the Cures Act was to ensure that a pharma or biotech company developing a medicine in Phase 2 or Phase 3 have a publicly posted policy for evaluating requests for expanded access. Under the terms of the Cures Act, the policy must include:
Contact information for the investigational product developer, manufacturer, or distributor to facilitate communication about expanded access requests
Procedures for making an expanded access request to the company
Anticipated time it will take the company to acknowledge receipt of a request
General criteria used to evaluate and respond to a request
A hyperlink or reference to the clinical trial record specific to the medicine and submitted to ClinicalTrials.gov, if applicable.
Pharma and biotech companies may choose not to offer expanded access for their medicine, but they are still obligated to include a statement to this effect in their publicly available expanded access policy. Companies receiving breakthrough, fast-track status or regenerative advanced therapy designations are required to make their expanded access policies public 15 days after the therapy receives its designation.
Companies may have many legitimate reasons for not making their medicine available to patients via expanded access outside of clinical trials. They are constantly assessing the risk-benefit profile of a medicine in development and this profile will evolve as more safety and efficacy data become available through completed clinical trials. Hence, a company’s view on expanded access may change over time as they collect more data, complete enrollment in pivotal trials, have adequate drug supply, obtain better funding, etc.
Unfortunately, not all companies have received the message about having a publicly posted expanded access policy. In a review conducted in 2021 by Kang, et al[1]., the authors found about one-third of applicable biopharmaceutical companies (32%, 140/423) with an oncology asset in development implemented the Cures Act requirement to have a publicly posted expanded access policy. Further, less than one-third of public policies (31%, 44/140) contained all described requirements of the Cures Act. Larger companies and those with at least one medicine receiving an FDA expedited designation, or at least one FDA-approved medicine were more likely to have a public policy.
The authors conclude based on these results that the Cures Act may be having a limited impact on its goals of supporting timely medical decisions and closing informational gaps for patients and doctors around expanded access to investigational oncology therapies, especially for products sponsored by smaller and newer companies.
Think Global
Even though companies may be enrolling patients into their clinical trials all over the world, their expanded access policy is often very US focused. It is important for companies to acknowledge that early access and compassionate use pathways exist outside the US, and to similarly manage expectations of patients and patient advocates around the world.
What Should Companies Do?
Here are some suggestions for companies to consider related to their expanded access policy:
If a company does not have a publicly posted expanded access policy for a medicine in Phase 2 or Phase 3 that will treat a serious or life-threatening disease, developing one should be a priority.
In my experience, expanded access policies that reflect the ethos of senior members of a company’s management team will have the most profound impact on external stakeholders. This means getting input up front with key internal stakeholders. This obviously takes time but is worth the effort.
If a company is engaging with a patient advocacy group (PAG) associated with the disease, consider getting input from the PAG to better understand the views of patients they support.
Make the expanded access policy easy to find in the company’s website. It can also be published on the Expanded Access Navigator Company Directory at the Reagan-Udall Foundation for the FDA.
Consider including language that helps patients in countries outside the US better understand the company’s overall early access position.
A small biotech may be more restricted around expanded access compared to a large pharma based on what the small biotech faces to keep their development engine moving forward. Nonetheless, in a culture focused on good customer service, we can do better as an industry to help manage expectations of all stakeholders in the drug development process around early access to medicines, even if the message is that clinical trials are the only way to access the medicine and expanded access won’t be considered at this time.
A good resource for further reading on this topic is the Guide for Companies and Sponsors at the Reagan-Udall Foundation for the FDA.
An expanded access policy may be a new concept for some companies with no prior experience or have medicines in the early stages of clinical development. It is important that all companies develop plans for how to address expanded access as they develop their new medicines.
I have experience taking companies through the process to craft or revise their global expanded access policy. Reach out to me at lagusjohn07@gmail.com if your company is looking to speak with an expert to better understand this space.
Access the Advantages of Early Access Programs
John Lagus is the founder of Bluestem Pharma Consulting, LLC. Based in the US, John leverages more than 30 years of experience in pharmaceutical services to guide biopharmaceutical companies as they explore the benefits of early access programs and integrate these programs and related initiatives into their global market access strategies. While these programs can offer a wide range of benefits to both the sponsor company and patients, they remain relatively unfamiliar, are defined by regulatory requirements that vary by geography, and require specialized expertise to execute flawlessly.
John is passionate about helping patients around the world with unmet medical needs get access to lifesaving or life-changing medicines, and supporting companies as they implement these important programs.
1. Kang S, Chang S, Ross JS, Miller JE. Implementation of 21st Century Cures Act Expanded Access Policies Requirements. Clin Pharmacol Ther. 2021 Dec;110(6):1579-1584. doi: 10.1002/cpt.2401. Epub 2021 Sep 12. PMID: 34431083.
