Uses for Recombinant Protein Drugs - Biotechnology and Your Health: Pharmaceutical Applications - page 65

THE NEED TO MAKE TREATMENTS SAFE
Treating Hemophilia
In the early 1980s, the development of HIV/AIDS in young blood
transfusion patients was a powerful incentive to find other sources
for the blood-derived proteins used to treat hemophilia , a group of
inherited disorders affecting the clotting of blood. Hemophilia has
TREATING HEMOPHILIA
The FDA does not set the price for a drug; the manufacturer
sets the price after considering the costs of developing the
drug, the relative benefit of the drug compared with alterna-
tives, and the amount insurance companies reimburse for
alternative drugs. Because of patents and FDA rules, for
several years after a drug is approved for sale, the pharma-
ceutical company usually has an exclusive right to sell the
drug before exact copies of the drug, called generics, may
be sold. When generics enter the market, competition usually
drives down a drug's price. To gain FDA approval to market
a generic small molecule drug, a manufacturer only has to
show that it can consistently manufacture the drug to high
standards of purity, and that the generic version of the drug
is taken up and eliminated from the body in the same way as
the original drug—tasks that may be more difficult to do for
biotechnology drugs. Currently, the FDA is considering what
the rules should be for a generic biotechnology drug. But
rules allowing the sale of generic biotechnology drugs will
not necessarily drive down the price. Some biotechnology
drugs treat rare diseases, and manufacturers may not invest
in producing generic biotechnology drugs unless a large number
of people need the drug. The National Organization of Rare
Disorders (NORD) works with a number of pharmaceutical and
biotechnology companies to provide financial assistance for
people who need particular medications.