Letter to Congress (July 1, 2016)
RE: Request for your support of The Right to Try Act (S.2912/H.R. 3012); The REGROW Act (S 2698 / H.R. 4762); and The RESULT Act (S 2388)
Asking for the right to try promising treatments when death is certain is not some right wing plot dismantle or weaken the FDA. It is a call for compassion and reason – much like the decision to administer ZMAPP (prior to any human trials) to westerners who contracted Ebola. For rapidly fatal diseases like ALS and others that lack treatment options, there are promising therapies that are simply inaccessible. They are stuck in a drug approval process that dates back to 1962. Today, medical researchers recognize that diseases and patient responses to therapies are not homogenous. Yet, the rigid standard for proof of efficacy (an arbitrary value) is still based on 54-year old medical understandings. As a result, new drugs and treatments require, on average, over a decade of clinical trials costing up to $1 billion before they get approved. Due to a lack of funding, many promising therapies simply die – along with the patients that could benefit. The only way to know if a drug works is to give it to sick people.
In 1992, in response to HIV-AIDS activism on this same issue, the FDA adopted the Accelerated Approval Program (AAP), a conditional approval. The patient community demonstrated that there were treatments capable of extending their lives and demanded the right to try them. In 2012, Congress again directed the FDA (FDASIA 2012, Title IX) to utilize Accelerated Approvals for fatal diseases lacking treatments. The FDA is still resisting this directive and as a result, Right to Try laws are sweeping the country – 28 states and counting.
Expanded Access Programs (EAP) only work for individuals with considerable financial resources and only for treatments manufactured by drug companies big enough to afford the risk. The federal Right to Try bill would provide necessary legal protections for drug/biotech firms, and only applies to products in the FDA approval pipeline and in the very specific case of a terminal illness lacking life-saving treatments. The bill provides liability protection to doctors, clinics, and drug/biotech companies and shields a company’s FDA approval application from setbacks due to adverse events. Without these two protections, companies are largely unwilling to make their treatments available.
There are life-saving treatments already approved in Europe, Japan, and elsewhere that we cannot legally obtain here. A privileged few are taking up residence in foreign countries in an effort to save their own lives. The RESULT Act promotes reciprocity. It would require the Food and Drug Administration (FDA) to quickly review drug, device, and biologic applications from sponsors who have products approved and sold in developed countries with rigorous approval processes.
The REGROW Act is necessary to modernize the approval process that is impeding stem cell research. The U.S. is falling far behind other countries. In sixteen years of R&D, there is only one approved stem cell treatment in the U.S. and researchers repeatedly list FDA’s rigid adherence to outdated protocols as the biggest obstacle to development.
On May 12, 2015, a group of us (families dealing with ALS) traveled to Washington to sit down with Dr. Janet Woodcock, Director, FDA CDER, in the first of several meetings. Over the past 14 months we have heard a willingness to consider greater flexibility – still, nothing substantive has occurred. In the interim, two people in that meeting died while fighting for access to therapies in the FDA approval pipeline – and ALS claimed another 6,000 Americans lives.
We live in a country with extraordinary medical capabilities and a nation committed to the sanctity of life. It is therefore shocking to discover you are being protected to death by being denied access to your only lifeline – therapies showing benefits in clinical trials. Unquestionably we need the FDA to protect the public health, but we also deserve an FDA that is reasonable and responsive, especially in the case of fast-moving fatal diseases that lack approved treatments. When you are dying, there is no such thing as “false hope;” there is only “hope”.
Please support these three important bills. Terminally ill people have the fundamental right to try to save their own lives. “Life, Liberty, and the Pursuit of Happiness”.
Hope NOW for ALS
Board of Directors:
Bob Hebron (ALS caregiver, NJ)
Jehad Majed (ALS caregiver, MI)
Linda Clark (widowed ALS caregiver, CA)
Eric Valor (person with ALS, CA)
Dr. Stephen Finger (person with ALS, SC)
Jay Smith (person with ALS, TX)