Real World Insights

Nov 01, 2012
By Pharmaceutical Executive Editors

Sue Barrowcliffe
Managed access programs (MAPs) are designed to provide access to medicines outside of the clinical and commercial setting, for patients who have no other available treatment options. Such programs provide patients with access to treatment in special cases, but companies can also use information gathered under compliant MAP programs to gain insight into how a product might perform in the real world, ahead of regulatory decisions and commercial launch.

When are MAPs relevant?

MAPs can be applicable whenever there are patients with unmet medical needs wanting access to a medicine which shows evidence of efficacy and safety relevant to their condition and which are unavailable through the normal commercial or clinical trial routes. This situation arises in a variety of circumstances:
» During Phase II/III for patients who fail to meet the strict inclusion/exclusion criteria of a clinical trial or access to a clinical trial site.
» In the time after clinical trials and before commercial availability, while waiting for NDA/marketing authorization submissions to be submitted and approval granted.
» Following first global approval of a new medicine, commercial availability in other markets can take months or years.

While keeping focus on creating access for patients, there is a unique opportunity to observe how the product is used and performs in this wider setting. By its very nature, the context of a MAP is closer to a real world setting than to a clinical trial setting. This information is useful to the manufacturer, and will also be welcomed by regulatory agencies and health technology appraisal bodies.

It is important to recognize that MAPs are neither clinical research nor seeding trials. In this column, we look at the types of insight that can be gained, and how the data might be used.

Key principles

MAPs utilize regulatory mechanisms in place around the world that allow a physician to treat a patient with an unlicensed medicine. These regulatory mechanisms have a variety of names including expanded access, named patient, and compassionate use, and the exact approach to be used may vary depending on whether the product is globally unlicensed or available in some countries. The key to such programs is that irrespective of the regulatory mechanism, it is the physician who requests access to a medicine that they feel would benefit the patient.

The scope for data capture beyond the required adverse event information includes patient demographics, medical history, baseline symptoms, drug and dispensing information, laboratory data, and outcomes. The general guidelines for collecting data as part of a MAP are meant to ensure that physicians don't have to perform additional tests beyond what he or she would normally do as part of his or her clinical practice, unless there are tests or follow-up required for safe use of the product. This is not a limitation but a benefit, since it keeps the environment as close to the "real world" as possible.

The intended use of the data from a MAP will have some influence over the approach and tools used. In all countries the collection of adverse event information is required and can be used to get a real-world view on the safety profile of a product. However, in countries where the treating physician is responsible for the reporting, it may be more challenging for the manufacturer to get comprehensive information on the safety profile. So in cases where a company wants to collect robust data on a specific outcome measure or follow an important laboratory safety parameter, it would be more appropriate to focus on countries that have group access mechanisms where a protocol covering the required assessments is agreed on with the regulatory agency, and physicians are obliged to comply.

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