ORPHAN DRUG
SEARLY ACCESS TO ORPHAN DRUGS
FOR RARE DISEASES



An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.

Availability of orphan drugs
The granting of marketing approval does not mean the drug is available throughout all countries. The marketing approval holder must decide beforehand on its commercial status within every country and 



the drug will then go through numerous steps in each country in order to condition its management, and usually its price. Despite joint efforts from many stakeholders, the heterogeneous approaches among countries make patients’ access to orphan drugs more complex.

Early access to a drug for patients may be possible before its marketing authorization is granted to the pharmaceutical industry that develops it, most often during the third phase of the clinical trial and when its safety and efficacy are strongly assumed.

At SWISS GLOBAL PHARMA we strive to provide you with early access to such drugs be it through Expanded access programs (EAP) or Named patient programs (NPP) that exist in different markets.