What is Early Access, and what role can Early Access play as a means to overcome the critical challenges facing biotech and pharma in relation to the innovation and commercialization of novel therapies?

At Inceptua, ‘Early Access’ refers to any regulatory mechanism (e.g.; Named Patient, Expanded Access, Compassionate Use, etc.) which allows for the use of an unlicensed medicine to treat patients in an ethical and compliant manner.

Assets are not necessarily ‘early’ in their development timeline.  More times than not, these are medicines where some reasonable level of efficacy and safety have already been established in clinical trials and may even be advanced as far as commercialization in one or more countries.

Companies who make use of this type of mechanism typically do so in response to unsolicited physician/patient requests for access to their drug, being unapproved in their respective countries.  In making a new therapy more widely accessible in this manner, companies can help to prioritize the unmet needs of patients and engage new physicians, while maintaining a degree of protection over their asset in terms of how, when and where it may be used.

What are the patient benefits of Early Access?

Factors such as country participation and inclusion criteria can often limit the treatment populations for clinical trials; and the commercial launch of a new product does not occur simultaneously in all countries across the globe.  Therefore, the demand for an innovative or life-changing therapy will almost always exceed patients’ ability to access it solely through a clinical trial or commercial pathway. 

One of the greatest benefits of Early Access is that it expands the potential treatment options for physicians and their patients with unmet medical needs when all other clinical and commercial alternatives have been exhausted.

What are the typical case scenarios where an Early Access Program is relevant to consider?

An Early Access Program differs from a clinical trial, in that the primary intent is the treatment of patients vs. the collection of data and information for research purposes.

Along those lines, Early Access programs are typically applicable whenever a life-changing or, in some cases, life-saving treatment cannot be obtained commercially or through a clinical trial; and these scenarios exist across an asset’s lifecycle.

For instance, patients who do not meet study criteria or live in an area or country with no study sites may still benefit from a treatment under an Early Access program.  Companies may also use this type of program to continue therapy for study patients and, thus, bridge the gap between study closure and commercial availability.  Additionally, there are patients residing in countries where local approval and commercialization take years or may never occur who can be treated through this kind of program.

When should pharma companies ideally start considering/planning for Early Access and how is Early Access ideally integrated in the product lifecycle and anchored in the organization?

Over time, planning for Early Access programs has gone from being completely reactive to much more proactive.  Companies are taking greater care to anticipate the attention and unsolicited demand a new, innovative medicine might generate and make plans to address access requests ahead of time.

As is usually the case, starting sooner rather than later is always more beneficial, and companies can certainly formulate strategies to assess the direction of their portfolio and its various assets early on in the development lifecycle; and, in doing so can better identify the potential treatment ‘gaps’ that could exist as products move through Phase 2 and Phase 3 clinical trials into their peri-approval stage and commercial rollout, and be set-up in advance to address those needs, when applicable.

What are the key steps to implementation and deployment of the program, and how does Inceptua Early Access help clients to succeed?     

The successful launch of an Early Access program is frequently the result of thoughtful planning and solid management; and, once again, proactivity is preferred over reactivity.  In advance of any program, Inceptua work with clients to help assess the product landscape and determine program feasibility with respect to potential demand and the various, country-specific regulations and requirements.

As mentioned, the primary objective of Early Access programs is to get medicine to a patient who needs it; and, in that regard, it is critical to have efficient processes in place.  Inceptua has the experience, expertise and infrastructure necessary to support and carry out all activities for the receipt, qualification, approval and fulfillment of requests.