Estate Planning Seminar March 11th

Good morning! We are getting ready for our joint presentation with Blake Douglas.

Estate Planning – whether you are a college student or grandparent, there are legal documents to assist you and your loved ones in stressful times of need.

Thank you Barbara Pardue for the fabulous Facebook event design.

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https://www.facebook.com/events/794520557363555/

 

See you at the office building 5600 NW Central Drive – large conference room on the 1st floor at 2 pm.

 

The FDA Should Listen to the Voice of the Patient – Perspective on benefits of use of Eteplirsen

Dominic Romito watched during testimony about Sarepta’s clinical trial of an experimental drug to treat Duchenne muscular dystrophy.Photo from The Boston Globe 4/25/2016

Families with children with Duchenne muscular dystrophy (“DMD”) from all over the United States flew to Maryland to attend a Food and Drug Administration (“FDA”) advisory committee meeting on a mission to convince committee members to recommend accelerated approval of a new drug application for eteplirsen.  Scientists argued that the clinical trials provided evidence that the drug slowed the progression of DMD.  Parents received time-limited slots to discuss their own observations of the positive effects of the drug.

The accelerated approval provisions in Section 506(c) of FD&A Act, added by Section 112 of the Food and Drug Administration Modernization Act of 1997, allows for accelerated approval to “a product for a serious or life-threatening disease or condition . . . upon a determination that the product has an effect on a surrogate endpoint that is reasonable likely to predict clinical benefit, or on a clinic endpoint that can by measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments.”

The advisory committee was asked if the applicant provided substantial evidence from adequate and well-controlled studies that Eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit.  The phrasing of the question is not applicable where the study participation is small due to the rarity of the disease, such as DMD.  Surrogate endpoints and intermediate clinical endpoints should be considered in situations for clinical studies for drugs for rare diseases.

Unfortunately, after hours of testimony, the committee recommended against approval for Sarepta’s exon-skipping drug eteplirsen.

The FDA is allowed to expedite programs for serious conditions in Section 506(b) of the FD&C Act as added by Section 112 of the Food and Drug Administration Modernization Act of 1997 and amended by Section 901 of the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA).   Receiving and acknowledging the testimony of patients is part of the drug review process under the Patient Focused Drug Developments initiative launched by FDASIA.

By utilizing the flexibility offered under FDASIA, the FDA can fast-track the approval process for Eteplirsen.

Every minute matters to families with boys who have the rare disease.  The final FDA decision is with the director of the drug evaluation center, Janet Woodcock.

Debra Miler, co-founder and CEO of CureDuchenne, a nonprofit organization, offers her words of encouragement for approval at www.cureduchenne.org.  The advancement of treatment for a child with DMD affects the quality of life.

Senator Rubio brought attention to this matter – click here for the video SenatorMarcoRubio.

 

Radio spot – Info on Duchenne

Tomorrow morning at 8:00 on KPFT 90.1 Open Journal. Lindy Fillis (mom to Ditri age 5) and Erin Bullers (mom to Nicholas age 12) will be doing an interview from 8:25 to 8:55 explaining  Duchenne MD, upcoming Cure Duchenne Cares event taking place on Friday and Saturday, and  resources for families. They will also share with the audience on how they can help the Duchenne community with the critical adcom coming up on April 25th with the FDA. Although etepliresen only helps 13% of  Duchenne boys, it will pave the way for future drug approval.
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> You can urge your Senators to Sign a letter to the FDA. The link to do that is:
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Duchenne Muscular Dystrophy

Please think about visiting this website today and get great information about Duchenne Muscular Dystrophy and how you can help.
http://cureduchenne.com/blog/giving-our-sons-with-duchenne-the-best-care-possible/

The video starting with Emily and Will about clinical trials and research had a huge impact on me and I hope you take the time to watch it at http://www.cureduchenne.org/about-cureduchenne.html