Marcia Boyle
Special to AOL News
Jan 24, 2011
Thirty years ago my infant son was diagnosed with a rare genetic disorder that make even common cold germs potentially life threatening. His disorder — clinically termed a primary immunodeficiency disease — started me on my career as a patient activist.
Today, my son — and thousands of others suffering similar diseases — is leading a normal productive life without fear of becoming desperately ill from a minor infection, thanks in large part to a “biologic” drug called immunoglobulin therapy. It does for him what his body’s natural immune system cannot.
But this life-saving treatment is now being threatened by the potential that the Food and Drug Administration could allow cheaper, less rigorously tested, and allegedly similar, versions of the medicines into the market.
Biologics are expensive. There’s no question.
They are prescription drugs created through biological processes instead of the chemical processes used to produce conventional drugs. It’s like the difference between churning out plant food in a factory and cultivating an orchid in a greenhouse. And immunoglobulin therapy is a very complex biologic prepared from plasma pooled from thousands of donors.
To cut the cost of these medicines, the FDA is considering a plan that would approve minimally tested substitute drugs, called “biosimilars.” In theory, generic versions of simpler chemical drugs are identical to the originals, at least in terms of chemical formula. However, that is not the case with biosimilars. As the name suggests, they are similar but not identical to the original biologics.
Why does this matter?
Compared to the chemical manufacturing process that produces the prescription medicines that most Americans use, the sophistication and uniqueness of the production process for biologics makes it difficult to produce a biosimilar.
And compared to the process for producing chemical drugs, the complicated process of creating a biologic can be affected by very subtle changes in conditions.
The problem is that even small variations in the creation process can make a given biologic ineffective or even dangerous for certain patients. For immunoglobulins, what may appear as minor differences in manufacturing can result in serious, life-threatening adverse events in some patients.
Of course, the additional care and investment involved in producing biologics makes these drugs very expensive. But biologics’ success in treating primary immunodeficiencies and other diseases has made them the fastest growing segment of the pharmaceutical market.
As a result, there is a huge potential market for lower cost biosimilars. As a patient advocate and a mother, I recognize the desire to have additional biological products available with the goal of lower patient cost.
As a mother and a patient advocate, I would love to see the price of biologic drugs reduced but not at the cost of endangering the patients, including my son, who depend on them.
This is the message that I and other patient advocates brought to the FDA during a recent hearing on biosimilars. We urged the FDA not to cave in to industry pressure and short-circuit the screening and testing process or “pathway” required for biosimilars.
The FDA has protected America from ineffective and dangerous drugs for a century. This is no time to compromise that tradition.
Marcia Boyle is the president and founder of the Immune Deficiency Foundation, the national patient advocacy organization devoted to improving the diagnosis, treatment and quality of life for the nearly 250,000 diagnosed Americans living with primary immunodeficiency diseases.
