When diving into the world of (orphan) medicines, you’ll encounter a slew of abbreviations for organizations and institutions. Commonly used abbreviations include CBG, ZIN, IGJ, EMA, VWS, CCMO, and others. Below is an overview of the key abbreviations and the roles of these entities.

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Medical-scientific research

METC (medical ethics review committee):
For medical-scientific research involving test subjects, METC approval is required. A detailed dossier must be submitted to ensure the safety of participants.

CCMO (central committee on research involving human subjects):
The CCMO ensures the protection of people participating in medical-scientific research by overseeing METCs.

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Product development and registration

MAH (marketing authorization holder):
An MAH holds a marketing authorization, allowing the sale, supply, import, export, or provision of a medicine to patients. Without this authorization, such activities are prohibited. Obtaining this requires rigorous assessment and usually involves clinical studies. For rare diseases, finding enough patients for these studies can be challenging. Sometimes, existing data from repurposing efforts can be used.

CBG (medicines evaluation board):
The CBG decides whether a medicine can be sold in the Netherlands (or whether an MAH receives authorization). Strict requirements must be met, and the CBG continues monitoring medicines post-approval to ensure patient safety. The board also focuses on clear patient information and collaborates with ZIN to expedite medicine availability through parallel registration and reimbursement procedures.

EMA (European Medicines Agency):
The EMA is the European counterpart to the CBG, evaluating the effectiveness and safety of medicines for the European market. It also determines whether a drug qualifies as an orphan medicine, meaning it is effective for a rare disease affecting fewer than 1 in 2,000 people. The drug must also outperform existing treatments for the condition.

FDA (Food and Drug Administration):
The FDA is the U.S. equivalent of the EMA, responsible for ensuring the safety and efficacy of drugs, as well as food and cosmetics, for the American public.

Notified body:
A notified body assesses whether medical devices, such as bandages, wheelchairs, medical instruments, or prosthetics, meet legal requirements. Approved devices receive a CE mark.

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Reimbursement

ZIN (national health care institute):
ZIN advises the Ministry of Health, Welfare, and Sport (VWS) on whether a medicine should be reimbursed under basic health insurance. This involves balancing effectiveness and affordability. For drugs with uncertain efficacy, ZIN uses conditional admission policies. Each country has its own methods for determining reimbursement.

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Post-marketing

Lareb (Dutch pharmacovigilance center):
Lareb collects, records, and analyzes reports of medicine side effects. Patients can report side effects via Lareb’s website, which helps improve understanding of drug safety. This is crucial for orphan medicines, often tested on small groups, to monitor rare side effects post-study.

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National and European authorities

EC (European Commission):
The EC drafts and enforces legislation, including EU regulations and guidelines for medicines and orphan drugs.

VWS (Ministry of Health, Welfare, and Sport):
The Ministry of VWS oversees topics such as medicine affordability, reimbursement, and counterfeit prevention. It negotiates drug prices, including expensive orphan drugs.

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Regulatory oversight

IGJ (health and youth care inspectorate):
The IGJ monitors healthcare safety, including the production, research, and transportation of medicines. It also oversees the import of unavailable medicines from abroad, requiring documentation from the prescribing doctor or pharmacist.