ALS-ETF

Remember when I asked you to sign an online petition in support of expanded access (aka compassionate use) to promising treatments for ALS? Well, in a bit of cruel irony, the writers of the original petition (dubbed ALS Treat Us Now) have sadly succumbed to the disease. However, their spirit lives on…In 2012, with your Read more about ALS-ETF[…]

We Need the FDA to be Faster

Last Monday, leaders from nearly every major ALS organization in the United States, in addition to patients, caregivers, physicians, scientists and other concerned individuals, addressed a panel of representatives from the U.S. Food and Drug Administration (FDA) as the agency conducted its first-ever public hearing specific to amyotrophic lateral sclerosis (ALS).The day’s agenda can be Read more about We Need the FDA to be Faster[…]

FDA ALS Public Hearing (cont’d)

Pat Wildman, Director of Public Policy for the ALS Association, plans to call for expanded access and accelerated development and approval of ALS therapies at next week’s FDA ALS Public Hearing. He also will advocate for the use of biomarkers and surrogate endpoints “to speed development and let us know sooner whether something is working or not working,” Read more about FDA ALS Public Hearing (cont’d)[…]

Compassionate Use (cont’d)

Are you confused and/or overwhelmed by all of this?  Well I am, what a cluster!  Thankfully, most of us aren’t required to be experts in FDA law.An easier and more understandable way to help the cause, is to sign an online petition to advocate for expanded access to promising treatments for ALS.  Like the Saltzman family below, “I love my wife Read more about Compassionate Use (cont’d)[…]

Compassionate Use (cont’d)

The ALS foundation ‘Treat Us Now’ does not promote or recommend any particular investigational drug.  Instead they work to provide access in cases where patients and their doctors have choose to pursue a particular drug.  The foundation directs its access campaigns to drugs that have performed favorably in human safety studies and have already shown Read more about Compassionate Use (cont’d)[…]

Compassionate Use (cont’d)

Expanded access programs require a significant amount paperwork by the sponsoring physician (including the Investigational New Drug Application) and by an Institutional Review Board; who first must be both aware of the drug and inclined to pursue it.  Furthermore, for the prospective pharmaceutical company who has agreed to provide the drug, there has been little perceived assurance that Read more about Compassionate Use (cont’d)[…]

Compassionate Use (cont’d)

The 2009 ammendment also defined three categories of patient populations to whom investigational drugs could be made available for the purpose of treatment outside of a clinical trial through expanded access.  They are:Source: www.fda.govindividual patients,groups of patients smaller than that typical of a treatment investigational new drug (IND) or treatment protocol, andtreatment IND – larger populations Read more about Compassionate Use (cont’d)[…]

Compassionate Use (cont’d)

The sponsor (typically a physician) requesting expanded access to an investigational drug will most likely want to have it shipped to clinical investigators in many states.  However, current FDA law requires that a drug be the subject of an approved marketing application before it is transported or distributed across state lines.  Therfore, the sponsor must seek an Read more about Compassionate Use (cont’d)[…]

Compassionate Use (cont’d)

In 2009, the FDA amended the expanded access program rules in order to ensure broad and equitable access to investigational drugs for treatment.  The regulations included the following:Source: www.fda.govCriteria that must be met in order to authorize the expanded access useRequirements for expanded access submissionsSafeguards to protect patients and the clinical trial processThe regulations also included general criteria for Read more about Compassionate Use (cont’d)[…]