The Washington Post recently carried an article regarding an investigative compound being studied for the treatment of Duchenne muscular dystrophy which affects young boys – a condition for which there is no current treatment. The course of the condition means that that those affected will face physical decline that robs one of one’s ability to use muscles – to walk and eventually – to live.
The piece highlights a clinical trial for an investigative compound to treat the disease and the grassroots effort that was organized to call attention to the need for a treatment and to remember the plight of patients who did not have time to wait for the full process of investigation and approval.
The story captures so very well the tug of war that exists between the societal need for demonstrating safety and efficacy of a compound and the need of individuals who desperately need something – anything – that can save them. The families of the boys used several means to spread the word, including social media tools.
This made me think back to the earliest years of the AIDS epidemic. The approval of new drugs took a much longer time than it generally takes today. Not only were there no treatments for the underlying cause of the disease – HIV – many of the opportunistic infections that one got as a result of having a compromised immune system had been somewhat rare diseases where treatments were somewhat undeveloped. I made the rounds of hospitals in Manhattan doing deathbed wills for people who shouldn’t have had to face their own mortality for decades to come – a painful memory that is never far from the surface for me. The death toll for a subset of the population was enormous. It wasn’t just the fact that people were going to die, it was the way they were going to die that added so much exponential suffering. And the excruciating slowness with which new treatments were brought to market had to change.
As a result of a good deal of activism by a lot of AIDS activists, things did change. Activism forged the circumstance by which there was dialog and reconsideration and new standards. Today, decisions on drugs are made within a specific time frame – one that can sometimes be accelerated. And from the grim circumstances of the AIDS epidemic came accelerated approval and priority review.
For the boys with Duchenne’s, according to the article, FDA has provided a path forward for the drug being studied that may include accelerated approval. While not guaranteeing approval by any means, it is a move that holds out some degree of hope.
Naturally, science and judgment play the essential role in developing new treatments, but from time to time, so does activism that can eventually forge a new and better framework. The mind and the heart don’t work well without one another.
For a really thorough overview providing a history of accelerated approval – “A History of Accelerated Approval: Overcoming the FDA’s Bureaucratic Barriers in Order to Expedite Desperately Needed Drugs to Critically Ill Patients“.