The Important Role Patient’s Experience Play in Drug Approvals {Interview}

The FDA drug approval process can seem complex and arduous to people unfamiliar with the procedures that are in place. For people with rare diseases hoping for drugs to be approved, one of the toughest but most important virtues they need is patience.

Most drugs that undergo preclinical (animal) testing never even make it to human testing and review by the FDA. The drugs that do must undergo the agency’s rigorous evaluation process, which scrutinizes everything about the drug–from the design of clinical trials, to the severity of side effects and even the conditions under which the drug is manufactured. [1] It can take a decade or more to go through the full clinical process and be considered for approval.

This patience can truly be tested when the one waiting for the drug approval is a child with a rare disease. Children suffering from certain diseases are no different from any other group awaiting approval for promising drugs.

One such disease is Duchenne muscular dystrophy (DMD), the most common type of muscular dystrophy. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms usually begin early in childhood. Children with DMD have a hard time standing up, walking and climbing stairs. By their teens, they will need wheelchairs to get around and will lose use of their arms. They typically don’t live past their 20’s and die due to heart and lung problems.[2]

There is currently no cure for DMD, but things are looking up for the community. There have been some breakthroughs in treatment options, some of which are showing real promise to slow the relentless progression of the disease. Children with DMD at one point were only surviving until their teens, but now are living into their 20s and 30s.   But there is still more to be done. There are some policies that have modernized the review process for rare diseases to include the patient experience as part of the data that they consider when evaluating a drug.

Join me in a recent interview with Dr. Strober and Caretaker, Julie-Caregiver as they discuss:

*Why it’s so difficult for a new treatment to pass FDA approval?

*What happens to a drug that doesn’t get reviewed or receives a complete response letter? Can they go back to the drawing board?

*How hard is it to keep hope alive while waiting for a promising treatment to come to market?

*What can people do as a community to get their voices heard?


View the complete interview here:

For more information, go to

Bio for Dr. Strober:   Dr. Strober is a clinical Professor of Neurology and Pediatrics at UCSF.  He is the director of the Pediatric Neuromuscular Clinic and MDA Care Center at Benioff Children’s Hospital.  This clinic, sponsored by the Muscular Dystrophy Association, treats children with disorders of the muscle and nerve. Strober, a specialist in childhood neuromuscular disorders, is board certified in neurology and psychiatry and has special certification in child neurology. He also is trained to perform electromyography (EMG) to evaluate patients with suspected neuromuscular disorders.

Bio: Julie-Caregiver: Julie Cosgrove-Garcia lives in Sacramento, California with her husband Marcus and son Nick (25). Nick was diagnosed with Duchenne muscular dystrophy, a rare and devastating degenerative disease, in 2001. Duchenne muscular dystrophy causes muscle weakness and eventually robs patients of their ability to walk, move their arms, and care for themselves. She works full time at Pacific Gas and Electric Company.







Interview is courtesy: PTC Therapeutics





Cynthia Tait

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