Improving Access for Patients with Unmet Medical Needs
1 The Need for Access
Point to any country on a map of the world. That’s where you might find a patient in urgent need of a new therapeutic option. For many patients, access to new medicines via the well-known, well-defined clinical trial process or through commercial channels is not an option. Consider these scenarios:
- The patient does not live in close proximity to a clinical trial site.
- The patient does not meet trial eligibility requirements.
- The patient was enrolled in a trial, the trial has ended, but availability of commercial supply is delayed.
- The patient lives in a country where a new drug is not or will never become commercially available.
For patients with an unmet medical need who require a new therapeutic option, the wait for approval and commercialization may simply be too long.
Fortunately, mechanisms exist in many countries around the world that enable patients in these situations to gain access to medicines that are in clinical development, are unlicensed in a specific market but licensed elsewhere or are in the Marketing Authorization Application (MAA) process, in a business and regulatory compliant and ethical manner.
Such mechanisms are referred to by a number of terms including compassionate use, named patient use, expanded access and early access, to name a few (Table 1.1). Mechanisms can be put in place for groups (i.e., cohorts) of patients or for individuals (i.e., named patients).
This concept paper provides an overview of these programs, the regulatory environment governing them and best practices for design and implementation.
Read more by downloading Improving Access for Patients with Unmet Medical Needs (Published: June 2014).