The Unlocking Lifesaving Treatment Act of 2012 (HR 3737) is giving the rare disease world new hope. Across the country, people with rare diseases (roughly 10% of the population) and others are coming together at the grassroots level to show support.
I have heard some of the criticisms of the bill, namely:
- It exposes the patients to greater risk
- It completely eliminates any burden to prove efficacy.
The standards for safety would not change. Safety is established during the first phase of drug trials. As far as I can tell, I don’t see any changes to requirements for establishing safety for new drugs.
Regarding efficacy, any accelerated approval requires post-market surveillance to prove effectiveness against clinical endpoints. Initial approval is based on biomarkers. This is nothing new.
The only thing new would be a change in the rules for getting a biomarker approved. ULTRA would prevent the FDA from requiring previous clinical data to qualify a surrogate endpoint. This data just doesn’t exist for many rare diseases. For diseases where clinical data is not available, ULTRA would ask the FDA to use valid science and our understanding of biology to qualify biomarkers that are reasonably likely to predict that the drug works.
Is this the right choice? You’re damn right it is. I do not want to wait until there is rock-solid bulletproof evidence that a drug works before I can treat my kid. I think that 99% of people who have terminally ill family members feel the same way. Anyone that says that the system is working is crazy. 95% of rare diseases have NO TREATMENT. By definition, that is broken. We need game changing new laws. New laws that will transform the landscape.
The world is changing. Information technology enables people to make their own decisions. Long gone are the days when people had to rely on a single group to “be their voice”. This is a new world of ideas; A world where each of us can watch congressional hearings on Ustream and don’t have to travel to Washington. This is a world where ideas spread fast and the grassroots can make their voice heard directly. When it comes to ULTRA, the rare disease community has spoken. We want treatment and we want it now.
We thank Congressmen Towns and Stearns for their leadership. By introducing this bi-partisan bill they have shown that the giving hope to people with rare disease is not a Democratic or Republican issue. Rather, it is a human issue. Drug development for this under-served population isn’t where it needs to be. Everybody with a disease deserves treatment.
