In many cases, patients with serious or life-threatening illnesses cannot wait until a new drug is officially approved and marketed. For these patients, the Early Access Programs (EAP), Compassionate Use Programs (CUP), and Managed Access Programs (MAPs) provide an important treatment pathway. The content and main application methods of these schemes are different, and they are not the same as obtaining clinical trial drugs.
Early Access to Medicines Program (EAP)
EAP is a program for the treatment of patients with serious or life-threatening diseases for new drugs that have not yet been formally approved for marketing. These patients have often tried all existing treatment options and are unable to participate in clinical trials. EAPs are primarily for drugs that have completed early clinical trials, such as Phase II or III, and are supported by preliminary safety and efficacy data.
Mercy Drugs Program (CUP)
CUP is a special type of EAP that is commonly used in patients with rare diseases. Because of the rarity of these patients, it is difficult to find suitable clinical trials to participate in. CUP allows patients to use drugs that have not yet been approved outside of a clinical trial setting, provided that the drugs have a potential therapeutic effect and that there are no other effective treatment options for the condition.
Extended Usage Plan (MAP)
Managed Access Programs (MAPs) are programs designed and administered by pharmaceutical companies to provide patients with serious or life-threatening illnesses with new drugs that have not yet been approved by the market or covered by health insurance. The main purpose of these programs is to enable patients to take these drugs in advance in specific situations, thereby reaping the potential therapeutic benefits.
Similar program in Taiwan: Project Imports Drugs
In Taiwan, the Ministry of Health and Welfare allows the introduction of drugs for personal use or for the treatment of critical or critical patients, which is similar to the EAP. For serious diseases that may not be treated with existing drugs, or emerging drugs that have not yet obtained a drug license in China, and cannot be provided to patients in a timely manner, a specialized teaching hospital or a teaching hospital at or above the regional level may apply for the approval of the manufacturing or import of the project in accordance with Article 48-2 of the Pharmaceutical Affairs Law and Article 2 of the “Measures for the Approval of Manufacturing and Import of Specific Drug Items” announced on September 8, 105; or in accordance with the provisions of Article 2, Paragraph 3 and Article 13 of the Administrative Measures for Drug Sample Gifts amended in 111 and the FDA Yao Zi No. 1081408668 Letter dated October 5, 108 from the Ministry of Health and Welfare, prepare all the required information and apply to the Food and Drug Administration for the import of drug items.
Differences with clinical trial medications
Clinical trials are systematic studies of new drugs, usually divided into multiple phases (Phase I, II, III) to evaluate the safety, efficacy, and optimal dosage of the drug. Patients are required to meet specific enrollment criteria to participate in clinical trials, and the treatment is conducted in a highly controlled environment.
EAPs and CUPs are provided to patients who are unable to participate in clinical trials, allowing them to receive treatment before the drug is officially available. These programs do not have a control group and are not strictly controlled by clinical trials, but they can provide timely medical support to patients who need treatment urgently.
Difference Between MAP and Patient Support Program PAP
Managed Access Programs (MAP) and Patient Assistance Programs (PAP) are both patient expansion programs designed by pharmaceutical companies to provide patients with drugs that have not yet been approved by the market or included in health insurance With the application of PAP towards more diverse applications.
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peculiarity |
Managed Access Program (MAP) |
Patient Support Program (PAP) |
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Target patients |
Patients with serious or life-threatening illnesses that are often unable to participate in clinical trials or have no other treatment options |
Patients who need specific medications but cannot afford or are not yet covered by health insurance do not necessarily have a life-threatening illness |
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Drug status |
Drugs that have not been approved in the market, or have not been approved in some countries |
Drugs that have been approved on the market, but are not yet covered by health insurance or are not affordable for the patient |
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target |
Provide early access to drugs that are not yet approved by the market, meet the needs of patients in urgent need of treatment, and collect real-world data (RWD) to support future market approvals |
Provide financial support or free medication to enable patients to access approved but not covered medications, improving patient access and continuity of treatment |
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Data Collection |
Collect treatment data to inform future drug review and market approval, including patient response to treatment, side effects, and other clinical outcomes |
It mainly tracks the use of drugs and the effects of financial support, and is not usually used as the primary basis for drug review |
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Objectives of Use of Data |
The focus is on collecting real-world data (RWD) to support the safety and efficacy assessment of the drug |
The focus is on providing financial support to ensure that patients have access to treatment on an ongoing basis, rather than collecting clinical data |
─ Evie Wang, Chief Operating Officer of PatientsForce Harvest Health Integration Group
