How many times have we felt that those in positions of power don't understand what it's like to live with Migraine disease, cluster headaches, or other headache disorders? Too many times to count. This is an issue that increases the burden of living with these disorders. It increases our feelings of frustration, isolation, and more.
Today, I want to share an opportunity with all of you and encourage each of you living in the United States to take advantage of it...
The Food and Drug Administration (FDA) is holding a public meeting next week related to FDA's patient-focused drug development initiative. The initiative is being conducted to fulfill FDA performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). This effort provides for a more systematic approach under PDUFA V for obtaining patient perspective on the disease severity and the currently available treatments. FDA is publishing a preliminary list of nominated disease areas for the patient-focused drug development initiative and the criteria used for nomination.
The list FDA has published in the Federal Register is a preliminary list for the program, not the final list. A final list of 20 disease areas will be selected for inclusion in this five-year pilot program. Then, quarterly, over the next five years, FDA will meet with stakeholders — patients, caregivers, physicians, and others — with an interest in one of the 20 disease areas for an in-depth review of the needs related to treatment, the current state of treatments, disease burden, the human side of living with the disease, and other issues of possible relevance to reviewing treatments that may come before the FDA for review. It's truly an interesting and potentially very valuable program, and I hope you want to know more about it. There's an excellent summary of the background in Volume 77, Number 185 of the Federal Register.
Those disease areas initially nominated as a starting point to develop the list of 20 for this program are:
- Pulmonary arterial hypertension.
- Heart failure.
- Primary glomerular diseases.
- Huntington's Disease.
- Peripheral neuropathy.
- 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.
- Patients who have experienced an organ transplant.
- Sickle cell disease.
- Chronic graft versus host disease.
- Aplastic anemia.
- Lung cancer.
- Cancer and young patients.
- Cancer treatment in pregnancy.
- Cancer and sexual dysfunction.
- Cancer and depression.
- Clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).
- Thrombotic disorders (e.g., antithrombin deficiency and protein C deficiency).
- Primary humoral immune deficiencies (e.g., common variable immune deficiency).
- Neurologic disorders treated with immune globulins (e.g., chronic inflammatory demyelinating polyneuropathy).
- Hereditary angioedema.
- Alpha-1 antitrypsin deficiency.
As you can see, neither Migraine nor any other headache disorder is included in this preliminary list of nominated disease areas.
The public is invited to comment on this preliminary list online through a public docket and at a public meeting on October 25 where FDA will provide an overview of the patient-focused drug development initiative with discussion of the nominated disease areas.
Last week, I attended a preliminary meeting on this subject at the FDA headquarters in Silver Spring, Maryland. At that meeting, I learned:
- Some diseases such as ALS and Alzheimer's are not on this list because FDA staff members who compiled the list felt it obvious that they are of high importance.
- The above list is indeed a list of preliminary nominations, not the final list.
- Not everyone who has registered for and been confirmed for attendance will have an opportunity to speak. Those who do will have three minutes to state their case.
- Written comments submitted by the public are important.
The Alliance for Headache Disorders Advocacy has asked me to attend the meeting next week on their behalf, and the American Headache Society has asked me to represent their new patient organization, the American Headache and Migraine Association. I will be there representing all of us, but don't know at this point if I will have the opportunity to speak. Whether I have the opportunity to speak or not, it IS important that we are represented.
What I can tell you is that it absolutely is helpful for every single U.S. citizen to submit written comments for this. Comments are submitted through a web form for which I'll give you the address. I strongly suggest that you write your comments in your word processing or email program where you can work on them, edit them, check the spelling, and be sure to say exactly what you want to say. Then copy your comments into the web form.
The Agency used several criteria to develop the preliminary list of potential disease areas. FDA requests that when proposing additional disease areas for consideration, please describe how you applied the identified criteria in making recommendations for additional disease areas to consider. FDA also welcomes public comment on the criteria for disease area selection. These criteria include the following:
- Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
- Disease areas that reflect a range of severity;
- Disease areas for which aspects of the disease are not formally captured in clinical trials;
- Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
- Disease areas that represent a broad range in terms of size of the affected population; or
- Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.
I fully realize how difficult it can be not to get very emotional when preparing comments such as these, but please — address the criteria. Do share your experience, but please try to keep your comments relatively short, and don't get too emotional. We need to come across as determined. Short, concise, and succinct is best.
Are you ready to comment? Please go to this page for the Prescription Drug User Fee Act Patient-Focused Drug Development Meeting and click the blue "Comment Now!" button near the top right corner of the page!
This is a magnificent opportunity to tell FDA what it's like to live with Migraine or other headache disorders. Please, let's not let it pass without making ourselves heard. Comments must be posted by November 1, 2012.
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