Early access programs (expanded access, compassionate use, named patient supply) are becoming a standard part of the development and launch process for medicines. This change, apparent over the last 5-6 years, has been driven by a greater awareness of such pre-license supply pathways by both physicians and patients, and a recognition by industry that such programs offer wider benefits than the ethical and social responsibility aspects that have largely driven them in the past.
Typically, the regulations state that early access should be for serious, debilitating or life-threatening illnesses, and that there should be no licensed alternative available in the country of intended use. However, these restrictions aren’t usually enforced by the regulators, who are more concerned with the safety profile of a medicine, and are generally happy to allow a physician to use their clinical judgement as to why an unlicensed medicine might be suitable for a patient.
Early access programs offer a wide range of benefits for patients, physicians and pharma companies alike. The opportunity to enter a market early, gain experience outside of the trial population, generate data and potentially generate revenue are all seen as attractive features of early access. They certainly can be attractive, but is opening an early access program always the right thing to do?
Situations where an access program may not be the appropriate option would include;
- Where there have been no requests from physicians for access ahead of approval
- Where the underlying driver is focused on revenue generation
- Where the primary intention is data collection
- Where the data suggests marginal improvement over available therapies
- Where your manufacturing/supply chain is not at full capacity
If any one of these aspects is apparent, it would be advisable to work through a detailed Early Access Feasibility Analysis to gain a deeper understanding of the asset-specific environment before implementing a program.
Factors which lend themselves to successful early access programs include;
- Where you are already receiving requests for early access to the product
- There is no therapy available at all for the indication in question
- Or, data suggests dramatic improvement over existing options
- Well-established and active patient groups
There is no guarantee that these factors will result in an early access program that meets expectations, but they certainly increase the likelihood. There are a lot of intangibles in early access, not least awareness and physician perception of product, but being aware of some of the ‘red flag’ situations will increase your chances of a successful program.
Stuart Bell, Vice President, Inceptua Early Access
Stuart Bell, VP, Inceptua Early Access
Stuart Bell has more than 20 years of healthcare consulting experience, with the last 10 years focused on early access. Stuart has been involved in over 100 early access programs. Stuart is responsible for Inceptua’s consulting activities, including: strategy and policy, feasibility analyses, real-world evidence development and communications. He pioneered the development of global corporate strategies on early access, the concept of detailed Feasibility Analyses for early access, and developed the first early access-specific EDC for real-world data collection.
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