President Obama signed the Food and Drug Administration Safety and Innovation Act (FDASIA) yesterday.  This new law includes major provisions of importance to the rare disease patient community and creates several changes to FDA policy including a provision to empower the FDA to accelerate approval of life saving treatments for patients with rare diseases.

The FDA Safety and Innovation Act  includes some of the most groundbreaking legislation measures for rare disease patients and their families since the Orphan Drug Act of 1983.

Rare Disease Legislative Advocates (RDLA) and EveryLife Foundation played key roles in developing this new legislation and helping get it enacted.  Global Genes Project also worked closely with RDLA and industry to make sure the voices of the rare disease community were heard.

Press Release: WASHINGTON, July 10, 2012 /PRNewswire-USNewswire/

Obama Signs FDA User Fee Legislation Bringing Hope to Rare Disease Patients

EveryLife Foundation for Rare Diseases Applauds Congress for Including Provision to Empower the FDA to Accelerate Approval of Lifesaving Treatments

July 10, 2012, Washington, DC – Yesterday President Obama signed into law The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, bipartisan legislation that will spur the development of lifesaving treatments for 30 million Americans suffering from rare diseases.

“We are thrilled the language to improve access to the FDA’s Accelerated Approval pathway for rare diseases has been included in FDASIA,” said Emil Kakkis, MD, President, EveryLife Foundation for Rare Diseases.  “We wish to thank Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY) for being champions for the rare disease community.”

Stearns and Towns first introduced Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA) to empower FDA to use all the science available for allowing surrogate endpoints in clinical trials for rare diseases to determine whether a drug is working, significantly decreasing the development time and cost.  Stearns and Towns later introduced Faster Access to Specialized Treatments (FAST) Act that improved Accelerated Approval for life-threatening diseases while maintaining high safety and efficacy standards.

FDA’s Accelerated Approval has been successful in getting treatments approved for cancer and AIDS patients, but has been essentially unavailable for rare disease treatments.  There are currently fewer than 400 FDA-approved treatments for nearly 7000 rare diseases.  Investment and interest in development will surge for rare diseases if there is access to the Accelerated Approval pathway.

“We would not have been successful if it were not for the great work of Energy and Commerce Chairman Fred Upton (R-MI), Biotechnology Industry Organization (BIO), and more than 300 patient organizations that advocated for improving the FDA’s regulatory process,” added Kakkis.

FDASIA is the culmination of more than a year of negotiations between industry and FDA and includes the reauthorization of the drug and device user fees.
The Foundation will host its fourth Rare Disease Workshop on “Developing Guidance and Policy Recommendations for Accelerated Approval in Rare Diseases” on November 15th in Washington, D.C.  FDA, NIH, industry and academic scientists are invited to participate.

The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.  We can do more with the science we already have and bring life saving treatments to millions of people suffering from rare diseases.
Contact: Julia Jenkins, Director of Public & Government Relations
Email:   Phone:  415-884-0223

9 thoughts on “Historic Legislation Signed by President Obama To Help Patients with Rare Diseases”

  1. Brad Wilson says:

    My son has recently been diagnosed with MD. Will this and can this help my family? What help can we get if not?

    Thanks so much
    Wilson Family

  2. The legislation helps speed the approval of rare disease therapies proposed to the FDA but does not guarantee that someone is working on a therapy for your specific disease. “Muscular Dystrophy” is actually an umbrella for some 4 dozen different specific diseases. Have you searched to see if someone is working on your specific muscular dystrophy and contacted them for an updated status? If you can advise us of the specific form of muscular dystrophy we can perhaps point you in the right direction.

    – Global Genes Team

  3. nicolas says:

    quiesiera saber, confirmar o ratificar, que dentro de estas enfermedades raras esta la Atrofia Muscular Espinal tipo 1, y si es asi, me pregutno, ¿esto significa que se encontrara mas rapido el remdio para la enfermedad, o se acortan plazos?, gracias.-

  4. If we understand your question correctly – are you asking to confirm whether or not Muscular Atrophy, Spinal Type 1 is one of the specific diseases which will be included for finding faster cures of the disease or shorten the deadlines for existing treatments?

    – Global Genes Team

  5. Michele says:

    Would all types of SMA be included in this research?

  6. SMA Type 1 is a RARE Disease and is covered by policy that affects RD, like PDUFA. Just as in the previous inquiry, we don’t know who might be taking advantage of this policy to develop/accelerate a cure for SMA 1. Have you searched to see if someone is working on your specific muscular dystrophy and contacted them for an updated status?

    – The Global Genes Team

  7. Kacey says:

    I have been told i may have mitochondrial myopathy,test results inconclusive, i have thynoma in my chest test results for myathenia gravis inconclusive, i have progressive degeneration of the thoracic spine and have been told i could have myotonic muscular dystrophy, i can no longer work get worse by the week and have to fight for disability i have no insurance and no way to get a definate diagnosis, is there any help with genetic testing or to help find out what disorder i have, i can’t even get treatment for pain.

  8. Kacey –

    Those of us @GlobalGenes are committed to education and awareness of Rare Disease; thus, we hope you will find the following information useful in your pursuit to find support, guidance and testing for the disorders your mentioned:

    For support and guidance: 888-MITOACTION (888-648-6228)

    For genetic testing options:

    Our best to you always,

    – The Global Genes Team

  9. Carolyn b heath says:

    I have ALS. I want to try GM604. There is no other option. I will die before the drug completes the clinical trials. There is controversy over granting FDA accelerated approval to to GM604. I thought this was the very intent of FFDA?

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