Question

What are examples of controversial (but legal) usage of the orphan drug law (as it can result in huge profits for the drug companies)?

drugs already mass marketed for certain diseases still qualified and been approved for orphan drug law patent as they are used to treat rare diseases also

a drug can receive multiple orphan drug patents if manufacturer proves that the given drug can be used to treat or cure each and everyone of these diseases

certain drugs receive only one (1) orphan drug patent even if manufacturer proves that the given drug can be used to treat or cure several rare diseases

a and b

none of the above

Answer 1

Orphan drug law was passed in US to facilitate development of orphan drugs which affect small number of people(<200,000) residing in US. Thus more companies are willing to invest money on drugs that have a small market.

To date, 7000 rare diseases have been identified. In aggregate, these rare diseases end up being common and affect 25 million people in the US, but each condition is unique.

Many ethicists and scientists have argued that some pharmaceutical companies have exploited the law. A key provision of the law is that each medicine gets approval for 7 years of market exclusivity. Kaiser Health News, revealed in 2015 that pharma companies sell these drugs at a very high price using:

1) repurposing commonly used drugs

2)getting approval to use one product for multiple orphan diseases.

example : AbbVie's Humira was approved by FDA in 2013 to treat rheumatoid arthritis which treats 1 million adults in US alone. This later got approval to treat several orphan diseases including juvenile rheumatoid arthritis and pediatric crohn's disease.

Peter Saltonstall, president of National Organization for Rare Disease told KHN on 2015 that Humira is not a "true orphan drug".

So, a and b are the correct options for this question.