Patients Outside the U.S.
Outside the U.S., these access methods are known by a variety of names, including, for example, "named patient programs" (NPPs—in
which physicians can request access to drugs on behalf of individual or "named" patients), "compassionate use," and "temporary
authorization for use."
Regulations differ widely among countries due to differences in national medical practices, resources available, product funding,
hospital structures, and national insurance systems. These programs also allow access, in specific circumstances, to drugs
that are approved in other countries but not yet approved in a patient's home country. Additionally, when there is a small
patient population spread across a large number of countries (as with a rare disease), a company may be able to offer access
to their drug before seeking approval and commercial launch in dozens of geographies that would not otherwise have been feasible.
The thirty countries comprising the European Union (EU)—which fall under the jurisdiction of the EMA—each have their own nationalized
regulations regarding access to unlicensed medications for individual patients and patient groups. For example:
» In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) oversees use and approval of unlicensed medicines.
Permission is obtained prospectively, and use is permitted based on a physician's clinical judgment that an individual patient
has an unmet medical need and has no suitable alternatives available.
» In France, the French regulatory agency (AFSSAPS) is primarily concerned with the science supporting a request that a patient
with a particular indication be allowed access to a drug not approved in France. Additionally, access may be permitted for
single patients (Temporary Authorizations for Use; ATU nominative) or groups of patients (ATU cohort) and permission is obtained
» In Ireland, the Irish Medicines Board requires tight control on distribution of unlicensed medicines with assurances that
the drugs will not be released beyond the intended patients named in NPP requests. Retrospective notification must be completed
within two days of use.
» In Germany, Federal Institute for Drugs and Medical Devices (BfArM) oversees access to unlicensed medicines. Historically,
the primary mechanism has been via single-patient access. However, in 2010 new legislation was put in place for group access.
Countries outside the U.S. and Europe also offer these programs. Canada's "Special Access Program" provides access to non-marketed
drugs to practitioners treating patients with serious or life-threatening illnesses when conventional therapies have failed,
are unsuitable, or are unavailable. Similarly, in Australia, patients can access experimental drugs via the "Special Access
Scheme." In Japan, government-sanctioned "Named Patient Access" allows access to drugs with an expectation that the drug be
approved in the exporting country.
In all of these scenarios, managed access programs provide a route to obtain innovative drugs prior to approval or launch.
To support the needs of these patients, companies should proactively consider offering pre-approval or pre-launch access as
part of their drug development plan in therapeutic areas with critical or unmet patient needs.
John Lagus is Vice President, New Product and Market Development, at Idis. He can be reached at Jlagus@idispharma.com
Kelly Feam is Manger, Regulatory Information and Intelligence, at Idis. She can be reached at firstname.lastname@example.org
Benefit from industry updates and case studies related to this article at the 5th Annual Congress on Access Programs for Investigational and Pre-Launch Drugs, July 19-20, 2012, Philadelphia, PA