Email L. Henry Platt, Jr.
Orphan Diseases is a term which here should be expanded to include all medical anomalies that affect only small numbers of people. When there are only a few people who suffer, there is much less opportunity or a drug company to turn a profit by spending huge sums of money to develop a drug or patentable therapy which has only a few potential customers.
At the beginning of the twentieth century, poliomyelitis was basically an orphan disease. Then Franklin D. Roosevelt was stricken with the disease. He fought to succeed in life as Governor of New York and President of the United States. Private charities were organized to pay for the research necessary to find a cure this crippling disease. Even public school children were encouraged to give pennies, nickels, and dimes for research and treatment of polio. The biggest medical news during President Dwight Eisenhower's first term was the FDA's approval of a vaccine to prevent polio.
Orphan diseases will probably always be with us, and research will continue with funding coming from private sources. The only suggestion I can offer here is to personally entreat pharmaceutical companies to spend a couple of percent of time/money on orphan diseases. [The reason they are called orphans is that they don't have a Big Daddy to give them Big Money!]
To read Commentary on this proposed legislation, click here.
© L. Henry Platt, Jr.