An orphan disease may be a rare disease presenting within a small population or can also include conditions that are not receiving adequate attention from the pharmaceutical industry, perhaps due to the geographical spread, such as in developing nations. Orphan diseases have a strict legal definition in many countries in order to encourage research in these areas. The US Food and Drug Administration (FDA) defines orphan drugs as those intended to treat rare diseases/ disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Many of the people living with rare diseases are desperate to do something to influence their illness. Thier eagerness to influence their illness can potentially lead them to take greater risks surrounding their treatment. This may also include a lack of proper consideration as to whether as to whether participation in a particular clinical trial is really suitable.
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