President Obama is asking us to craft a health care reform bill, to decrease spiraling costs, among other reasons. The pharmaceutical industry is one place to start. It has the highest profit margin of any industry in the United States, benefits from federal funding, but is not responsive to the public interest.
I am talking about the practice of drug trials and compassionate use. In our family's case, we worked hard to get money to a drug company to find a drug to cure Duchenne muscular dystrophy. When my son was diagnosed in 1991, there was very little funding for this disease which results from a random mutation of a gene and kills nearly 100,000 boys worldwide each year.
The late Sen. Paul Wellstone was the author of the Muscular Dystrophy Care Act, which was the only health bill passed in the 107th Congress. When I first met Paul in 1999 and explained how my son Jacob and 100,000 boys with Duchenne deteriorate and die in their late teens and 20s because there was no treatment due to the sparse federal funding, he told me he would do whatever he could and he did. As a result of Paul's bill, seven muscular dystrophy centers were created.
And, as a result of the work of parents of Duchene's boys, a drug company has received substantial federal funding -- most recently, $15.4 million from one of the Wellstone centers -- for development of a drug that compensates for the mutant gene that attacks my son's muscles.
Yet, despite our work on behalf of the company and its drug, my son has been denied the opportunity to participate in the drug trial. Moreover, he's also been denied access under the compassionate use provisions that the Food and Drug Administration has established.
How can our country move forward with health care reform when the pharmaceutical companies benefit from millions of dollars of taxpayer funded research dollars, but won't recognize their obligation to provide assistance to a dying child? Parents have lobbied hard to help drug companies make advances; yet, we find ourselves shut out from drug trials, the opportunity that may bring hope to our child.
Have we lost our way and our standards? Is there any obligation when pharmaceutical companies benefit from millions of dollars of taxpayer dollars to assist the families that have been their advocates?
Drug companies claim expanded access could prevent people from entering a clinical trial. That's just not so: the FDA has to approve all expanded access or compassionate use applications. The FDA would only consider the application if the person was denied access to the trial and there would be no adverse affect on the integrity of the trial.
In fact the FDA encourages expanded access in rare diseases such as Duchenne muscular dystrophy so they could have more information on the drug in a controlled setting before the drug goes to market. This helps them evaluate the safety of the drug better.
Many companies don't have a problem with compassionate use and it and in fact helps save lives. We can't allow our medical ethics to go out the window too.
Cheri Gunvalson of Gonvick is a registered nurse and parent advocate for Duchenne muscular dystrophy, testifying before Congress. A federal appeals court in December reversed a lower court ruling that would have provided an experimental drug to Jacob Gunvalson.