Dr.Marshall Summar, Chair of the Scientific Advisory Committee of the National Organisation for Rare Disorders (NORD), gives recommendations to the US Energy and Commerce Committee in making progress towards the 21st Century Cures initiative.

Incorporating the patients perspective: Director of NORD Gives Recommendations at Capitol Hill Hearing

In Advocacy, Regulation & Government by Cecile1 Comment

On Friday the 11th of July, the US Energy and Commerce Committee held a hearing on the 21st Century Cures initiative: Incorporating the  patients perspective.The 21st Century Cures initiative is a collaborative effort between scientists, regulatory boards,  pharmaceutical executives and patients aiming to improve the pace of cures and medical research breakthroughs in the United States. The meeting incorporated numerous patient perspectives, including Pat Furlong of the Parent Project Muscular Dystrophy (PPMD) group.

I have spent the last 29 years working in the field of rare diseases as a physician and researcher. I am the parent of a child with Down Syndrome Dr.Marshall Summar,Chair of the Scientific Advisory Committee

Als0 in attendance of the hearing was Dr.Summar from NORD, who has spent the last 29 years working in the field of rare diseases as a physician and researcher and is the “parent of a child with Down Syndrome,” has given  9 recommendations to the Committee in working towards the 21st Century Cures initiative:

1. Establish a national plan to determine priorities, resource needs and keep a consistent agenda on rare disease registries

2. Reform the Institutional Review Board (IRB) for assessing new therapies

3. Ensure current and new laws that increase patient involvement are fully implemented

4. Ensure consistent and adequate resources, direction and funding for the Food and Drug Administration (FDA) and National Institutes of Health (NIH)

5. Commission a US national plan for rare diseases

6. Enhance focus on clinical trial design and endpoint development within the NIH Division of Clinical Innovation

7. Create an orphan protected class within the medicare program

8. Establish clearer federal policies with regards to off-label use of drugs

9. Ensure reimbursement of ‘Medical Foods.’

Dr.Summar’s comprehensive list of reccomendations for the US government take considerable regard of patient views ad will hopefully lead progress towards the 21st Century Cures initiative

Read more here: Rare disease report


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