ORPHAN DRUGS 101

Understanding how AFT Pharmaceuticals is improving access to medicines for people with rare diseases

Orphan drugs are treatments for people with rare medical conditions.

People with rare medical conditions often find that treatments are either unaffordable or simply do no exist.

Typically this is because drug development is costly, and companies can find it difficult to recoup their investment when the number of people suffering from a particular condition are relatively few.

These people effectively become treatment ‘orphans’ - hence the term. Globally, nearly 7,000 rare diseases have been identified.1 While ‘rare’ implies that few people suffer from a condition, when extrapolated out across whole populations, the number of people with rare conditions can be very large. For instance, it’s estimated that 30 million people have a rare condition in the US,2 and a further 30 million in Europe.

Nevertheless, some pharmaceutical companies do develop, and make available, treatments for people with rare conditions. These are 'orphan drugs' or ‘orphan treatments’. AFT Pharmaceuticals is one such company.

To encourage companies to develop treatments for rare conditions, the regulatory requirements for developing and licensing treatments can be lessened by a regulator. For example, clinical testing studies may be permitted to use smaller patient groups. Tax incentives or research grants can be offered and patent protections increased. These incentives exist in the US and Europe legislation, but do not in New Zealand.