Managed access program

Managed Access Program (MAP) is an umbrella term used when defining an ethical solutions-based approach to provision of unapproved medicines for patients with unmet medical needs, typical of the following scenarios when drugs:
 * Are still in clinical development and have yet to be approved
 * May never be approved, but still have medicinal value for a very small population
 * Are approved in one country, but not in another
 * Have been discontinued in a particular market
 * Are an alternative to a drug discontinued globally
 * Are approved but not yet commercialized
 * Are used off-label in some countries

A variety of terms are used to describe these programs including:


 * Expanded Access
 * Early Access
 * Compassionate Use
 * Named Patient
 * Pre-Approval Access
 * Unlicensed
 * Off-Label

These regulatory programs are put in place on a country-by-country basis and can vary significantly across markets.

Geographic Differences
In the United States, the Food and Drug Administration (FDA) has sanctioned expanded access programs, which provide access to investigational drugs, since 1987. This access is typically offered in later stages of development, after earlier studies have established a relatively high degree of certainty in safety and effectiveness.

FDA regulations define three types of access:


 * Single-patient INDs: a request from a physician to the FDA that an individual patient be allowed access to a drug


 * Intermediate-size Patient Populations: when the FDA receives a significant number of requests (about 10 to 100) for single-patient access to an investigational drug for the same use, they may ask the trial sponsor to consolidate these requests.


 * Treatment INDs and Treatment Protocols: access for a large number of patients who would not otherwise qualify for clinical trials

In the European Union, named patient programs allow access to investigational drugs, as well as access to drugs approved in other countries but not yet in a patient’s home nation. For example, the programs allow patients to use drugs in the time period between centralized European Medicines Agency (EMA) approval and the launch in their home counties, a period which can range from one year to 18 months.

Other countries implement other forms of MAPs. Canada uses “Special Access Programs” to provide access to non-marketed drugs to practitioners treating patients with serious illnesses when conventional therapies have failed, are unsuitable, or are unavailable. Australia provides patients access to experimental drugs via the “Special Access Scheme”, while Japan’s “Named Patient Access” makes drugs available if they are approved in the exporting nation.

Patients
MAPs provide significant benefits to patients with unmet medical needs who have exhausted all the available treatment options. When patients with life-threatening illnesses, including cancer, infectious diseases and rare diseases, run out of therapeutic options, MAPs offer a route to obtaining other treatment methods.

Healthcare Providers
MAPs provide physicians and pharmacists with a clearly defined pathway to access new treatment options for patients in critical need, when not available through commercial routes or the clinical trial setting. In addition, MAPs provide healthcare professionals with educational support to enable them to use the drug appropriately in a way that will bring maximum benefits to the patient and minimize the chance of any adverse events.

Drug Manufacturers
MAPs offer a variety of benefits to the sponsoring pharmaceutical company. First, these regulatory programs provide companies with additional opportunities to assess a drug prior to the full launch. Companies can generate additional safety and real world data that helps in understanding how the drug will be used in clinical practice. In addition, they can manage risk in the pre-launch setting through tactics such as appropriate adverse event capture and screening patients to ensure the correct ones are selected to take the medicine.

MAPs help a company build relationships with both doctors and patients. The programs give more physicians experience with using the new medicine, creating advocates for the drug prior to its launch. They also allow the company to meet patient demands for treatment, exposing more people to the drug in a way that minimizes risk and is fully compliant with regulations.

Finally, MAPs provide financial benefits as well. They block patients from turning to competitors’ drugs and also generate additional revenue, which is especially important for small companies.