pLOG

2024-07-29

Proactive Deployment: Early Access to New Drug Treatments

In many situations, patients suffering from severe or life-threatening diseases cannot wait for new drugs to receive formal approval and become available on the market. For these patients, Early Access Programs (EAP), Compassionate Use Programs (CUP), and Managed Access Programs (MAP) provide an important pathway to treatment. The content and main application methods of these programs vary, and they are different from obtaining clinical trial drugs.

Early Access Programs (EAP)

EAP is a program that provides treatment with new drugs that have not yet received formal market approval for patients suffering from severe or life-threatening diseases. These patients have typically exhausted all existing treatment options and are unable to participate in clinical trials. EAP primarily targets drugs that have completed early clinical trials (such as Phase II or Phase III) and have preliminary safety and efficacy data to support their use.

Compassionate Use Programs (CUP)

CUP is a specialized form of EAP, typically used for patients with rare diseases. These patients often struggle to find suitable clinical trials to participate in due to the rarity of their conditions. CUP allows patients to use unapproved drugs outside the clinical trial environment, provided these drugs have potential therapeutic effects and there are no other effective treatment options available for their condition.

Managed Access Programs (MAP)

Managed Access Programs (MAP) are initiatives designed and managed by pharmaceutical companies to provide patients with serious or life-threatening diseases access to new drugs that have not yet received market approval or are not yet covered by health insurance. The primary goal of these programs is to allow patients to access these medications earlier in specific circumstances, thus providing them with potential therapeutic benefits.

Similar Programs in Taiwan: Special Project Drug Imports

In Taiwan, the Ministry of Health and Welfare allows for the import of drugs on a project basis for personal use or for treating critical or severe patients, which is similar to the Early Access Programs (EAP). For serious diseases that cannot be treated with existing medications or for emerging drugs that have not yet obtained market approval in Taiwan, special project importation is possible when timely treatment cannot be provided. Specialized teaching hospitals (such as psychiatric teaching hospitals) or regional teaching hospitals can apply for special project approval for the manufacture or import of such drugs according to Article 48-2 of the "Pharmaceutical Affairs Act" and Article 2 of the "Regulations for the Special Approval of Manufacturing and Importing Specific Drugs" announced on September 8, 2016. Alternatively, hospitals can apply for drug sample imports under the "Regulations for the Management of Drug Samples and Gifts," as amended in 2019, following Article 2, Item 3, and Article 13, and as per the Ministry of Health and Welfare's directive No. 1081408668 issued on October 5, 2019, by preparing the necessary documents for submission to the Food and Drug Administration (FDA) for drug project import approval.

Distinction from Clinical Trial Drugs

Clinical trials are systematic studies conducted for new drugs and are typically divided into several phases (Phase I, Phase II, Phase III) to evaluate the safety, efficacy, and optimal dosage of the drug. Patients participating in clinical trials must meet specific enrollment criteria, and the treatment process is carried out in a highly controlled environment.

In contrast, Early Access Programs (EAP) and Compassionate Use Programs (CUP) are designed for patients who cannot participate in clinical trials, allowing them to receive treatment with drugs not yet officially approved for the market. These programs do not have control groups or the stringent controls present in clinical trials but can provide timely medical support to patients in urgent need of treatment.

Difference Between MAP and PAP

Managed Access Programs (MAP) and Patient Assistance Programs (PAP) are both patient drug extension programs designed by pharmaceutical companies to provide patients with medications that have not yet received market approval or are not yet covered by health insurance. Some pharmaceutical companies consider PAP to be a type of MAP in terms of design, but the main objectives of these programs differ slightly. However, the current trend is moving towards the integrated application of MAP and PAP.

Features

MAP

PAP

Target patient

Patients with serious or life-threatening diseases often cannot participate in clinical trials or have no other treatment options.Patients who need specific drugs but cannot afford them or are not covered by health insurance, not necessarily those with life-threatening diseases.

Medication status

Drugs that are not yet approved on the market or have not been approved in certain countries.

Drugs that are already approved on the market but are not covered by health insurance or are unaffordable for patients.

Target

Provides early access to drugs not yet approved for the market to meet the urgent needs of patients and collect real-world data (RWD) to support future market approval.

Provides financial support or free medication to enable patients to access approved but uninsured drug treatments, improving patient accessibility and treatment continuity.

Data collection

Collects treatment data for future drug review and market approval, including patients' treatment responses, side effects, and other clinical outcomes.

Primarily tracks the effectiveness of drug use and financial support, usually not used as a basis for drug review.

Data usage objectives

Focuses on collecting real-world data (RWD) to support the evaluation of drug safety and efficacy.

Focuses on providing financial support to ensure continuous treatment for patients, rather than collecting clinical data.

 ─ PatientsForce Chief Operating Officer, Evie Wang