Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Monday, October 29, 2012

Empowered Patients Hold The Key

People dealing with ALS often feel like passengers on an out-of-control train.  That is changing.  The train is still out-of-control, but patients are starting to find their way to the locomotive.

It's Q4 -- that time of year when people wait for publications and announcements of ALS clinical studies.  The big global ALS MND conference in December is a target date for researchers. In the past, patients and families have been forced to wait patiently on the out-of-control train for news.  Clinical trial data are top secret.  People die waiting for information.  Valuable data are in a secret folder marked, "Don't Open 'Til December ... (Maybe This Year, Maybe Next, We'll See)."

The following was posted by Jamie Heywood in the ALS forum at --

Dear ALS Community, 
PLM has been working to refine the tools we used in 2010 to show lithium was ineffective. We are continuously refining our ability to predict progression in ALS and to improve our understanding of the disease.  The purpose is to give you better ways of measuring your health outcomes and eventually show, in real-time, what is working and what’s not.  We have recently prepared a publication to talk about what it means for the collective ALS community to share this kind of data, specifically on the development process for therapies like NP001 and KNS-760704 (Dexpramipexole) as well as off-label or experimental use of treatments like lithium and sodium chlorite. 
Our goal in issuing this publication is to encourage a discussion and debate as to the best way to address the complex medical, ethical, and scientific issues surrounding the collection and sharing of such data. We would appreciate your feedback and thoughts.  This is still not peer-reviewed work, although we are submitting it to one of the major scientific journals.  There is still time for your voice as patients to be added. 
Below is a link to the paper, but here is a summary of our results:
These are based on the PLM members that reported using these treatments, which are detailed in the paper.  Note for reference ALS patients on PLM decline at an average of roughly 1 FRS point per month:
  • Lithium:
  •  as we reported before, has no effect on ALS progression.
  • Sodium Chlorite:
  •   we have an 80% confidence that it is worsening ALS-FRS progression in ALS patients and potentially by quite a bit.  We estimate the most likely result to be an worsening of the average FRS points/month of +0.69 after correcting for a placebo effect.
  • NP001:
  •  we have a 75% confidence that it is slowing the rate of decline of the ALS-FRS progression by more than 20% over the 4-month trial period.  We estimate the most likely result to be a slowing of the average FRS points/month of 0.41 for the combined treatment arms in the study.
  • KNS-760704 (Dexpramipexole):
  •  we have a 64% confidence that it is slowing the rate of decline of the ALS-FRS progression by more than 20% over the 12-month analysis period.  We estimate the most likely result to be a slowing of the average FRS points/month of  0.27 FRS point per month for the treatment arm of the study. 
Please note that the error bars on our data are large (which is why we keep asking you for more data).  Therefore, it’s possible that even if our PLM population represents what actually happens in the trial, the effect could be much larger or smaller.  It’s also possible that our group is different than the rest of the patients in the study. We do not encourage anyone to make any treatment decisions based on these preliminary scientific findings.  As always, you should consult your doctor whenever considering changing treatments.
The full paper can be downloaded here:
It’s been six years since we launched our ALS community and every person who has stopped here along the way has contributed to where we are on this journey.  We thank you for that.  Please tell us what you think and advise us on how to use this approach to serve you.
Thank You

People with ALS don't have time to wait until some December to make their decisions.  They are forced to play their lousy cards the best way they can every day.  Information and facts are the best antidote to those lousy cards.  Information like Jamie describes above is simply empowering.

For those dealing with ALS, please post your data at ... regardless of what you are or aren't trying.  It will enlighten you and other patients.  It will get you all closer to getting control of that train.

Please unlock your data from those secret clinical trial and medical files.  Please share. You own your data.  Please google "patient-reported outcomes" to learn more about the difference you can make.

You hold the key.

Friday, October 26, 2012

Runners Left on Base. We Need To Drive Them Home!

Yesterday at the FDA meeting for Patient-Focused Drug Development disease areas, three gentlemen with ALS had solid base hits.  Now it's up to the rest of us to take some swings to bring them home.

There were a number of people and organizations who signed up to make public comments.  Because of the number of people asking to comment, each was limited to two minutes.  It was mostly organizations.  On the speaker list there were four individuals who would speak to ALS (three of whom were patients) plus two organizations -- MDA and ALSA.  All were interspersed throughout the long list of commenters. That was good.

Batter Up

After some brief FDA presentations the leadoff hitter was the veteran with ALS who has taken this cause to the patients.  He is not able to travel and the FDA read his comment.  It was a powerful statement.  His closing words were --
I provide that short summary of my ALS story to show the FDA that they may still have something to learn directly from ALS patients. ALS is obviously a complex disease with the potential for many secondary issues like the one I described. The only way the FDA is going really understand the entire ALS disease process, and the unmet medical need is by talking to ALS patients and caregivers directly. The only way the FDA will really understand the risks ALS patients are willing to accept in drug development is by talking directly to ALS patients. I encourage the FDA to change its position, add ALS to the disease list, and make ALS the stand alone topic of the first PDUFA V patient meeting.
Solid base hit.  One man on base.

After a few other speakers my name came up on the list.  I hope that I did no harm and set things up a little for the next ALS patients to speak. My words are in the prior blog post.

A few more speakers talked of their diseases and then came another man with ALS.  He made another strong showing.  The patients weren't working from an organization's agenda. Each spoke independently and uniquely.  The didn't always agree on details, but that is actually a good sign that they'll be productive participants in patient-focused drug development discussions.  He gave a strong message that this is a moment that must be seized.

Another solid base hit.  Runners on first and second.

The third patient speaker made quite an impression.  He told us that he would give anything to have some of the diseases being discussed rather than ALS.  He was motivated and reminded the FDA that nobody was paying him to be there (in contrast to the many paid advocates there).  He came across as a man who could add much clarity and common sense to patient-focused drug development discussions.

Another hit.  Bases are loaded.

It was time for the two organizations on the agenda to hit their runners home. The ALS speakers had made a strong showing.  Unfortunately neither organization adapted its comments to advance what ALS patients had just accomplished.

MDA suggested that all neuromuscular diseases be grouped into one "disease area" for the patient-focused discussions.  It was like a parent who doesn't want to pick a favorite child, perhaps.  It really didn't serve any of the diseases well in my opinion.  It certainly set back the concept that our ALS patients had set up so well.

Strike out.  Bases are still loaded.

It felt like ALSA came to a baseball game with a golf club.  They mentioned that they have been working with the FDA for two years.  They mentioned that ALS incidence is higher in veterans.  They mentioned that the FDA's announcement of disease areas was not clear to patients and has caused some "disease wars."  They asked for a Part 15 Hearing for ALS for 2013. That's an existing priority for ALSA and a noble cause indeed.  Unfortunately that hearing has nothing to do with the Patient-Focused Drug Development meetings and disease areas.  A 200 yard drive down the middle, but it was the wrong game.  Runners were not advanced.

Two outs.  Bases are still loaded.

It's up to us all to bring the three patients on base home.  Please continue to submit comments at!documentDetail;D=FDA-2012-N-0967-0001

There are other posts on this blog to help give you background.  We must make a good case for people with ALS to contribute as a disease area in the Patient-Focused Drug Development meetings.  We need more solid base hits to bring our base runners home.

Thanks to those who have submitted. Please take another at-bat and submit again.  If you've not submitted comments yet, take a few practice swings, and batter up.  Please don't leave three all-stars stranded on base.  We have until November 1. 

This Is What The FDA Heard Yesterday

Below are my comments delivered at yesterday's FDA meeting on their Patient-Focused Drug Development disease areas --

Thank you for the opportunity to comment today. 
We lost Mom to ALS over 15 years ago.
I know that the FDA doesn't need an ALS impact statement. You get it. We are grateful for that.
You also know about the ticking clock that ALS patients hear. It is fast and relentless. Those patients with their terrible ticking clocks can be a special asset to the FDA in patient-focused drug development discussions.
Whether you discuss risks or endpoints or measures directly with people with ALS, the ticking clock is always on the table. That can raise your game, just as speed training strengthens world-class athletes.

People with ALS ask good questions. They challenge us all (to borrow an expression from Apple) to "think different."
  • Some may talk of the frustrations of inconsistent FVC measurement.
  • Some may speak of those "last times" dates that they use to index progression - The last time one could turn the key in a lock, button a button, blow out the birthday candles.
  • Some may question today's gold-standard -- a functional rating scale that buries possible good outcomes among the bad.
  • Some may ask if you are sitting on top of an untapped gold mine of control data… if only they were gathered at clinic visits.
Just the FDA, ALS patients, and the ticking clock in the room, without the background noise of all of the rest of us stakeholders. Please consider the value that they will add to your insights. Please include their "speed training" as just five percent of your disease area discussions.
I leave you with a quote from Mom -  
"If you can't cure me, then at least learn from me."
Thank you very much.

Friday, October 19, 2012

This Has Gr8 Content!

If you read this on Friday afternoon, just click on the link and listen... and watching the #eadv twitter stream is interesting, too.

Later, the archives will be the next best thing to being there.

Thanks @eadvocacy !

Friday, October 12, 2012

We Need To Apply For A Job

The FDA has a help-wanted sign out in its window.

The FDA seeks 20 disease areas that will serve them well in a set of meetings on patient-focused disease development. There is a list of 39 candidate diseases; however, more are welcome to apply. You may submit your "disease resume" in advance. You may attend a live interview either remotely or in person on October 25.

If you don't apply, you'll never get the job. If you don't apply with a focus on what the FDA seeks, you'll never get the job. It's going to be tough. This is a very tight job market.

Do you wish you had some inside scoop on how to pitch ALS for this job? I think we got some by listening to the FDA's process meeting that was held Wednesday. Here's the scoop as I heard it:

There were a number of patient advocacy organizations at the process meeting. It was difficult to hear them all on the remote connection, but I would estimate 20 were there. There were also two individuals with ALS in the room!  In addition there were around a dozen people on the remote phone line, including at least three individuals with a direct interest in ALS.

There will be 20 meetings over the next five years, each of which will focus on one disease area.  The meetings will be an opportunity for the FDA to learn from patients.  The FDA wants hear what it needs to know about a disease from the patients themselves.  The FDA wants to hear the patients' thoughts on the benefits and limits of therapies.  The FDA wants the patient perspectives on benefits and risks.  Should they approve a drug despite risks?  What outcomes do the patients want?  How do patients think success should be measured? Are investigators today measuring the right things when they see if a drug works (or not)?   The FDA wants to enhance its reviewers' ability to find benefits in therapies.

Here's the rub.  We heard that the FDA knows that ALS is the worst of the worst.  The FDA has all process ready for a quick approval of the first thing that comes along with even a small amount of efficacy.  Clearly the FDA has all hands on deck to move on ALS as quickly as a therapy candidate comes along.  As a result, some folks there may not perceive that ALS has anything to add to the patient-focused disease discussions.  They feel that they have you covered when it comes to patient-focus, ALS patients.

We need to make sure that they know what's in it for the FDA by having ALS be one of the 20 patient-focused meeting diseases.  We are fortunate that in early 2013 there will be a completely separate FDA meeting to talk about ALS issues. That is great and all kinds of stakeholders can get things off their chests and learn from one another; however, that's not a patient-focused drug development discussion.  Asking for both is not overindulgence and there is a lot in it for the FDA.

In the last year we have seen people with ALS shake up some traditional thinking about clinical trials and measures and endpoints.  We have seen patient perceptions about measures and the way trials are trying to evaluate efficacy.  Below are the words of a man with ALS
“I wish clinical evaluations were more quantitative and less subjective… Our disease is not well understood, and it seems that every regular clinic visit offers a great opportunity to learn much more about how the disease progresses, including average and variations of measurable changes in strength measurements related to arms, legs, neck and even tongue. I suspect this data could then be used to add more precision to evaluations between treatment and placebo (control) groups during clinical studies, rather than rely on much less precise and much less stratified data from the use of (functional rating) scores and survival.”
Those comments fit the goals of the patient-focused drug development meetings like a glove. 

Do you remember last year when a man with ALS purchased a bunch of strength-grip testers on ebay for his friends with ALS? He thought that was a pertinent measure.  People with ALS have ideas about measures and endpoints and the risks that they are more than willing to take in order to have a shot at life.  People with ALS are motivated to a higher purpose for their limited days and surely their voices would make the FDA reviewers better.

We need to apply for this job.  People with ALS can deliver value for the FDA.  We need to make a good case.  These meetings are about making the FDA better.  We need to show them a disease population that can help.

Nobody ever earned a job offer with a me-oriented application.  Please apply for this job by showing the FDA what's in it for them.  People with ALS will add value to their processes, and perhaps in doing so will spark some new ideas that will result in better therapies for a lot of diseases.

Please submit comments at --

Please spread the word.  Persuasive grass-roots comments will make a difference.
We all need to apply for this job on behalf of all with ALS.  Please do it now.

Then plan to attend and participate in the meeting on October 25, either in person or remotely via the internet.

Thank you.

Monday, October 8, 2012

Mark Your Calendars

This morning while googling to defeat ALS, we found this --

Click here for document.

It contains important information about the October 25 FDA meeting on the 20 diesase areas that has been on our radar.


There is a link regarding an October 10 FDA "consultation meeting" to address "considerations and challenges in establishing and conducting a process that will be useful to both the patient community and FDA."  There is a link to the Federal Regsitry information on this meeting and instructions to contact for information to participate.  I believe that remote web access is possible. 

Saturday, October 6, 2012

Call to Action - WOOO - We're On Our Own!

The story of the FDA's list of 20 disease areas for patient-focused drug development continues. The plot thickens.

The fact that ALS was missing from the preliminary 39 candidates was found by an individual with ALS.  The comments being submitted to the FDA so far have been generated by grass-roots individuals interested in ALS.  We're on our own on this one, and we need to get ALS on the list as one of the 20 diseases areas for the FDA's patient-focused drug development initiative.  No big ALS advocacy organizations have picked up the ball on this one.

Other disease advocacy organizations for diseases that were already among the 39 candidates are on their cases, though.  Take a look at an example from the American Lung Association -- 
They obviously think that it's important to be on that final list of 20.

We're on our own, and we need to rally everyone interested in ALS to make a comment to the FDA so that people with ALS will be a formal part of the patient-focused drug development process!

Since we're not on the preliminary list, we need to submit thoughtful, cogent cases for ALS.  We need to stand out amid a flood of emails and comments as a compelling disease whose patients' perspectives count.

Here's your link to make a comment to the FDA --!documentDetail;D=FDA-2012-N-0967-0001

We're on our own on this one, and we must get ALS on this list!  It is important.

Blenders Are A Mixed Blessing

Beautiful dinners like this are meant to be savored, one bite at a time.

The whole is greater than any one ingredient.  There may be parts you don't like, but the adult palate tries everything and may leave a few things on the plate that don't hit the spot.

It would be a taste waste to put that entire dinner in the blender before eating.  Certain flavors would overcome all the others and some of the subtle, yet delightful, nuances would be lost in the puree. The textures and colors would be missed.  You might not even be aware of the foods that you dislike.

People with ALS are often forced to having the courses of a beautiful dinner smooshed into a blender.

That doesn't mean that it's right to smoosh their voices and ideas in a blender, too.  They have delightful insights (along with a few that may not be your cup of tea).  Some ideas from people with ALS may not be drug developers' or regulators' favorites, and there may be some subtle ones that are truly enlightening.  It would be wrong to force them to be a part of some kind of "idea puree" rather than having their individual voices heard.

As anyone with ALS will tell you, never puree the food before you absolutely have to.  The same holds  true for their ideas for drug development and approval.

Thursday, October 4, 2012

Please Speak Up

Please see the previous post here if you've not read it yet.

We have a public comments period.  It seems like a crime not to use it.  Individual voices make a difference.

The FDA is assembling a process to design more patient-focused drug development.  They are using a set of disease areas to attack the project systematically.  They realize risk and reward decisions should no longer be made without patients at the table.

Organizations and institutions can represent constituencies well, but people with ALS should be allowed to represent themselves first-hand, too. They are bright and full of ideas and deal with life in spite of that ticking clock.  They are not of one mind.  That's a good thing.  If the FDA gets to work with some of them on an up-close and personal basis, perhaps they'll start hearing the clock tick, too.

It would be a classic win-win for the FDA and for people with ALS if ALS were one of those represented disease areas.  Unfortunately ALS wasn't mentioned on their preliminary list.  It's time for the public to speak up during the public comments period, folks!

What are they looking for in the disease areas that they will use to develop the patient-focused processes?  Here's what it takes to make the cut --
  • 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
  • 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.
ALS fits these criteria like a glove. Please click here and read the materials and make some comments to the FDA on the importance of FDA patients in patient-focused drug development. It simply fits their criteria.

The letter that I submitted can be viewed by clicking here.

Thanks very much for exercising your free speech to help reform FDA processes to take advantage of what people with ALS have to offer. Those who follow them will benefit.

Tuesday, October 2, 2012

Action Item -- The FDA Needs To Hear From You Now!

Yesterday a man with ALS posted this in his facebook group:!documentDetail;D=FDA-2012-N-0967-0001

Please take a few minutes and read the document.  It is extremely important.  I have taken the liberty of posting most of the document below with some color highlights.

It is great that the FDA seeks patient perspectives on the drug development process.  It knows that it needs to enhance "benefit-risk assessment in regulatory decisionmaking."  That is welcome news for people with ALS.

There is no more perfect disease to get some fresh patient perspective on benefits and risks than ALS.  ALS is a stress test for drug development and approval.  So far the process has not passed that test.  I tried to find a picture of empty pharmacy shelves for this blog post, but I couldn't find one.  That speaks well for the positive impact that available drugs have on healthcare, but for people with ALS, those shelves are simply bare.

Please take a look at the criteria for disease areas to be selected for being the FDA's sounding-board for patients.  Please write your comments to the FDA to make sure that they get the valuable the benefit of hearing from people with ALS.

This listening process will happen over five years.  Most people with ALS won't make it to see the end of the process.  Perhaps that fact in itself will shock some people into designing some new risk-reward concepts for those who don't want the FDA to protect them to death and who want to make a contribution.

Please write.  If you are able, please show up to the public meeting.  Thank you.  Special thanks to Veterans With ALS on facebook for finding this.

[Federal Register Volume 77, Number 185 (Monday, September 24, 2012)][Notices][Pages 58849-58851]From the Federal Register Online via the Government Printing Office [][FR Doc No: 2012-23454] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2012-N-0967] Prescription Drug User Fee Act Patient-Focused Drug Development; Public Meeting and Request for Comments AGENCY: Food and Drug Administration, HHS. ACTION: Notice of public meeting; request for comments. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA) is announcing a public meeting and an opportunity for public comment related to FDA's patient-focused drug development initiative. This initiative is being conducteto 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 currentlyavailable treatments for a set of disease areas. FDA is publishing a preliminary list of nominated disease areas for the patient-focused drug development initiative and the criteria used for nomination. The public is invited to comment on this preliminary list through a public docket and at a public meeting where FDA will provide an overview of the patient-focused drug development initiative with discussion of the nominated disease areas. DATES: Submit either electronic or written comments by November 1, 2012. The public meeting will be held on October 25, 2012, from 9 a.m. to 12:30 p.m. Registration to attend the meeting must be received by October 18, 2012. See the SUPPLEMENTARY INFORMATION [[Page 58850]] section for information on how to register for the meeting. ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD 20993-0002. Entrance for the public meeting participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to
Submit electronic comments to Submit written comments to the Division of Dockets Management (HFA-305), Food and Dru Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. All comments should be identified with the docket number found in brackets in the heading of this document. 
FOR FURTHER INFORMATION CONTACT:Andrea Tan, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1168, Silver Spring, MD 20993, 301-796-7641, FAX: 301-847-8443,,or Robert Yetter, Center for Biologics Evaluation and Research (HFM-25), 1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448, 301-827-0373. SUPPLEMENTARY INFORMATION: I. Background On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA). Title I of FDASIA reauthorizes the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary user fee resources to maintain an efficient review process for human drug and biologic products. The reauthorization of PDUFA includes performance goals and procedures tha represent FDA's commitments during fiscal years (FY) 2013-2017. These commitments are referred to in section 101 of FDASIA and are available on the FDA Web site at Section X of these commitments relates to enhancing benefit-risk assessment in regulatory decisionmaking. A key part of regulatory decisionmaking is establishing the context in which the particular decision is made. In drug regulation, this context includes a thorough understanding of the severity of the treated condition and the adequacyof the existing treatment options. Patients who live with a disease have a direct stake in the outcome of the review process and are in a unique position to contribute to weighing benefit-risk considerations that can occur throughout the medical product development process. Though several programs exist to facilitate patient representation, there are currently few venues in which the patient perspective is discussed outside of a specific product's marketing application review. The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition. FDA is committed to obtaining input from patients and, as set out in the commitment letter, will conduct public meetings to consider 20 different disease areas over the 5-year authorization of the program. For each disease area, FDA will conduct a public meeting to discuss such topics as the impact of the disease on patients, the spectrum of severity for those who have the disease, the measures of benefit that matter most to patients, and the adequacy of the existing treatment options for patients. These meetings will include participation of FDA review divisions, the relevant patient advocacy community, and other interested stakeholders. FDA seeks public comment on the set of diseaseareas that will be discussed at these meetings throughout PDUFA V. A preliminary list of possible disease areas and the criteria used to identify these disease areas are published in this notice for public comment. FDA recognizes that there is significant interest in patient-focused drug development within the patient community. To ensure that patient stakeholders have an opportunity to contribute as this initiative moves forward in PDUFA V, FDA also is convening an additional series of patient consultation meetings with patient stakeholders to discuss key process questions for patient-focused drug development. These consultation meetings will be separate from the disease-specific meetings that are part of FDA's commitments in PDUFA V. FDA has published a separate notice elsewhere in this issue of the Federal Register requesting that patient stakeholders notify FDA if they intend to participate in the patient consultation meetings. II. Disease Area Nomination FDA is nominating the following disease areas as potential candidates for the focus of one of the 20 future public meetings and invites public comment on this preliminary list. In your comments, please identify the disease areas that you consider to be of greatest priority and explain the rationale for your recommendation. 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. Patients who have experienced an organ transplant. Sickle cell disease. Chronic graft versus host disease. Amyloidosis. Aplastic anemia. Melanoma. 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. FDA is also interested in public comment on disease areas that are not represented on this preliminary list. 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 [[Page 58851]] 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. FDA will consider the public comments received at the public meeting and through the docket and post the set of disease areas for FY 2013-2015 on the FDA Web site. By the end of FY 2015, FDA will initiate a public process for determining the list of disease areas for FY 2016- 2017.