On October 25, 2012, the FDA will provide an overview of Patient-Focused Drug Development and the public is invited to comment and share their voice. This will be the first of many public meetings held by the FDA over the next five years with a focus on 20 different disease areas. Registration for the October 25, 2012, public meeting ends on October 18.  Written comments into the public docket (FDA-2012-N-0967) are due by November 1, 2012.

The focus of the meetings include considerations in FDA’s regulatory decision-making process — ranging from the severity of the disease and the current available treatment options to the benefit risk framework surrounding PDUFA V.

Examples of potential process topics include the following:

  • Given the limits of FDA staff resources and time available, how to prioritize and balance different disease areas identified by different patient stakeholders.
  • How to approach issues when patient stakeholders for the same disease area have different and potentially conflicting views.
  • How to balance access to FDA for patient stakeholders who are local to FDA headquarters versus those in other locations who have less physical access.
  • How to support engagement of patients in disease areas for which no formal advocacy organizations exist. What role, if any, might organized groups already play?

FDA is currently nominating disease areas as potential candidates for the focus of one of the 20 future public meetings and invites public comment on this preliminary list.  Here are some examples of the diseases currently nominated.  Few are rare diseases.

  • Pulmonary arterial hypertension, Heart failure, Primary glomerular diseases, Narcolepsy, Huntington’s Disease, Depression, Autism, Peripheral neuropathy, Fibromyalgia, Obesity, Nocturia, Chronic fatigue syndrome, Irritable bowel syndrome, Inflammatory bowel disease, Alopecia areata, Diabetic ulcers, Female sexual dysfunction, Interstitial cystitis/painful bladder syndrome, Fracture healing, Diabetic foot infections, Hepatitis C, HIV, Sickle cell disease. Chronic graft versus host disease, Amyloidosis, Aplastic anemia, Melanoma, Lung cancer, Cancer and young patients, Clotting disorders (e.g., hemophilia), von Willebrand disease, Alpha-1 antitrypsin deficiency.

A list of criteria, patients/advocates would like to propose for consideration, can be found here. For more information and to view the preliminary list of the disease areas that will be addressed in these meetings, the FDA has published a Federal Registry Notice.

FDA is also interested in public comment on disease areas that are not represented on the preliminary list.  In order to have a disease make the final cut, patients/advocates will need to unite in their push for their respective disease to be included in the final list of 20 diseases.

As part of the PDUFA V performance commitment, the FDA will be holding consultation meetings with patient stakeholders to address the challenges and considerations required to establish processes both useful to the patient community and the FDA.

Participation at the consultation meetings require a notification of intent no later than October 3, 2012, as the first meeting will be held on October 10, 2012. See the Federal Registration Notice for further information.



23 thoughts on “FDA To Hold Series of Public Meetings To Pick 20 Diseases To Focus on Over Next Five Years; Few Rare Diseases On Preliminary List”

  1. KIMBERLY says:

    To whom this may concern,
    I do believe that SCLERODERMA is a disease that NEEDS to be on the Preliminary list. It is important that people are aware of this devastating disease that touch so many, many lives. This disease can be fatal and take women from their families at a very young age. Wont you please, please, PLEASE..consider SCLERODERMA on the preliminary list of Rare diseases.
    Thank you so very very much!!
    Mrs Kimberly Radomski,

  2. diane begley says:

    pLEASE CHOOSE sCLERODERMA….there are so many of us suffering and dieing!

  3. Sally Connolly says:

    Scleroderma should absolutely be on this list. Because of the wide variety of symptoms resulting from “hard skin”, progress on this disease could impact many other diseases as well: pulmonary hypertension, inflammatory bowel, chronic fatigue, cardiac issues, and many others. Finding a cure for scleroderma would bring relief to hundreds of thousands of people.

  4. Yvonne Johnson says:

    Please include Scleroderma in your 20 rare disease research.

  5. Thank you all for sharing your voice! Written comments to the FDA are due by November 1, 2012. 🙂

    For any of those who have not had a chance to share your story, raising awareness about Scleroderma is a great first step! Look for the heart, and submit your story today!

    – The Global Genes Team

  6. I’m very surprised and disappointed that Scleroderma is not on the preliminary list. Scleroderma is a much rarer disease than many that are on your current list. As a Scleroderma patient of 17 years, I would urge you to consider it as one of your focus diseases. There are between 300,000-500,000 people in this country suffering with this debilitating and life-threatening disease. Please consider Scleroderma as one of your 20 diseases. I would be happy to help you in any way I can.

  7. Christine Christou says:

    Please consider adding scleroderma to the list of rare diseases that receive special consideration.
    So many are suffering and so little is understood about this often fatal disease.
    Thank you for your consideration.

  8. Liz Leadbetter says:

    i live with Scleroderma. although i am not as badly off as some people, it has still totally changed my life, this disease is so unpredictable, sometimes it progresses slowly as with is with me, but others by the time they are getting dianosed they die soon after, i have known a young girl of 17yrs to pass away because she had scleroderma, we need to be heard, we are losing to many good people to this fatal disease, it can affect every part of the body inside and out including the skin ant the connective tissue, so little is known about scleroderma, it needs more funds for research, having scleroderma makes you feel you are living with a ticking bomb, and you are waiting for it to go off, you never know what is going to happen next,,, so pleas help us out here, we are not asking but begging you to put us on the list

  9. SuS Longiaru says:

    Please include Scleroderma in your rare disease research. Scleroderma is so difficult to diagnose, because not much is understood. While we are waiting for the Drs. to figure itout we are living in limbo, feeling so very sick, with no explanation. Please there are so many who need help, including our Drs. to understand in order to help us.

  10. Maria S.Leija says:

    When reading your list of rare diseases, it’s like looking at all that ails us with SCLERODERMA. Scleroderma Is so complex, deadly if we don’t take a multitude of prescriptions just to keep alive. This disease has affected many of us from the top to the bottom, no organ is spared. Our hair falls out, our vision dimmed/painful, teeth fall out, dysfunctional thyroid, gastro, reproduction organs, congestive heart failure pulmonary arterial hypertension, liver toxicity, blood disorders, mental/brain fogs, dermatology issues etc. The list of ailments is longer than you can imagine. I myself have many of what I listed but I’m not even mentioning what I have to take in prescriptions to keep this insidious disease stable. Please reconsider your list and include SCLERODERMA as primary, we 300,00 sufferers of this rare disease would be grateful. Give us the chance to live, please.


    I do believe that SCLERODERMA is a disease that NEEDS to be on the Preliminary list. It is important that people are aware of this devastating disease that touch so many, many lives. This disease can be fatal and take women from their CAREERS AS WELL AS THEIR families at a very young age. Wont you please, please, PLEASE..consider SCLERODERMA on the preliminary list of Rare diseases.

  12. dominique toland says:

    I think Scleroderma should be on that list. I suffer with this illness and i have many friends who ive met on facebook who also suffer and ive had a few die with it also. Enough of us have died with this how many more before we get our chance to be noticed.
    Thank u

  13. Lucie Mathis says:

    Please add Scleroderma to the Preliminary list. This disease has made my life very difficult to live. I was missed diagnose for years because not even the doctors know enough about this disease to properly diagnose it. Please help!! Many are dying from this disease. Thank you in advance for your consideration!
    Respetfully Submitted!
    Lucie Mathis

  14. susan savino says:

    because of it’s rare nature scleroderma should be a challenging disease for you to add to your preliminary list. There are many, people that live and die with this disease. There are few individuals that are even aware of it’s exsistence. Wouldn’t you want to be part of an organization that could be part of a cure?? This would be part of my answer to a prayer and wish I have had for a very long time as I have lived with various symptoms for many years. Please consider these requests in making your decision. Thank you , susan savino

  15. Cindy says:

    More information is needed on this disease.

  16. Crystal Bradley says:

    I believe that SCLERODERMA is a diease tht need to be on the preliminary list. It so many people that dont know about this disease and how it affect people life. People even die from this disease. i have it myself and it has affected my life in many ways im only 29 and had to stop college because of this disease because the pain hurt so bad and im not able to use a pencil to right cause of my hand. There is no cure for it just meds to try and slow it down and that dont really work. Could u please please please find it in your heart to add this on the list for rare diease

    Thank so much Crystal Bradley

  17. Kim Trevathan Turner says:

    Please add Scleroderma (Systemic Sclerosis) to the list of 20 diseases that you will focus on for the next five years. A potential life changing discovery was made during Scleroderma research this past May at The University of Pittsburgh. A peptide was found to be successful in reducing fibrosis in human skin and in mice. See: https://stm.sciencemag.org/content/4/136/136ra71 and https://www.scleroderma.org/site/News2?page=NewsArticle&id=6127. I don’t believe anything of this magnitude has been discovered in the potential treatment of Scleroderma in several decades. The progressive fibrosis (scar tissue due to overproduction of collagen) caused by Scleroderma ravages our lungs, heart, blood vessels, kidneys, skin, tendons and gastrointestinal tract. Scleroderma patients like me can only hope that whatever organ Scleroderma manifests itself in has a treatment that will lessen the sypmtoms of this horrid disease. For you to include Scleroderma in the list of 20 diseases could help to save the lives of many young women, as most Scleroderma patients are diagnosed as young women in the prime of their lives with young children. (I was diagnosed when I was 41 and my child was only 7 years old.) I also suffer from several of the diseases that have already been nominated….two of them, Pulmonary Arterial Hypertension and heart failure, were caused by my Scleroderma. I beg you to add Scleroderma to the focus list, not just to the nominated list. Thank you.

  18. There is significance in advocating for a rare disease in the final 20 REGARDLESS of whether the disease is listed as one of the 38 or not! Everyone should consider advocating for THEIR rare disease! Great suggestions by ALL!

  19. Monica says:

    To whom this may conser,
    I do believe it must urgently be accepted < my Son Died Last Year and 17 others in 3years 24 have lost their lives …..i cant do anything for my Son but believe me there are thousands suffering this BOB SAGET Sistor was one of them and a Movie was made about her life called (FOR HOPE) which should be released to TV once again
    Thank you so Much
    Monica Dugdale Jacquin….

  20. Patricia Castelhano says:

    Will you please put Scleroderma on your focus list. I have scleroderma for the past 16 years. During my duration of Scleroderma I was sick and in excruciating pain. I was in and out of the hospital for four years where I almost died. My doctor told my husband to prepare my family for I wasn’t going to survive. By a miracle I did survive. The recovery took about two years. I couldn’t walk and I had many complications. I was in a nursing home for six months. Scleroderma still affects my life. I’m in pain most of the time. I can’t walk long distances. It has put many restrictions on my life. So many people are suffering from this horrid disease….people are dying every day! So please please consider putting scleroderma on your focus list for there is no cure for this devasting disease,

    Thank you
    Patricia Castelhano

  21. Scleroderma has many people in its grip. It is not well known and not diagnosed in time for Specialists to help as so much damage has occured before the correct medication can be prescribed.
    Dr. Fredrick Wigley of Johns Hopkins Scleroderma Center in Baltimore has treated me for the past five years. I do not ache as before and through many medications have achieved a good quality of life to have this disease. It has not gone away and still is affecting my internal organs. The unknown is the worst part of having a rare disease. Please put this on your five year focus. Early detection is critical to a patient’s health.
    Sallie Carmichael

  22. Bailey Van Druff says:

    Scleroderma NEEDS to be on this list. People are are unaware of this and doctors do not know how to treat it. I am watching my good friend die that has this disease. She is 24 and is living in hell. Please PLEASE choose scleroderma.

  23. Sharon Snyder says:

    Scleroderma needs to be on the preliminary list. This is a disease that needs awareness and a cure. Please for all that are suffering and have lost to this terrible disease. In memory of my amazing Mother.

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