FRANK SASINOWSKI:  We are going to try to take and redirect the energy because the session that we had this morning was just fantastic.  If you have been to these large meetings, you often might find by 12:00 that people experience fatigue.  There was no fatigue in that room.  Dr. Kessler and the other people who were up on the panel were fantastic.  There was great interaction.  And so it is my pleasure now, before I introduce our luncheon speaker, Mike Astrue, to do one thing that Peter Saltonstall has asked me to do, which I think is pretty exciting.   
 

       You are the first people to hear that NORD has been working for quite some time to try to organize a congressional caucus on rare diseases. (Applause)  I met with Congressman Waxman in Denver at the Democratic National Convention about this and NORD has been working on this since.  (Applause.)  The rare disease congressional caucus is going to serve as a forum for members of Congress, their staff and related organizations to discuss issues affecting the estimated 30 million American men, women and children affected by the nearly 7,000 known rare disorders.  
 

       The primary purpose of the caucus will be to  seek creative solutions to the health care-related challenges facing people with rare diseases.  Secondarily, it will function as a forum to discuss issues critical to access, research and increased innovation.  We are looking forward to this.  Another one of the agenda items – I know Steve Groft and Tim Coté will be interested, of course – will be increased funding for both of their respective offices.  Also, we would like to have the congressional caucus look at fostering increased basic translational and clinical research into rare diseases and orphan products development.  The congressional caucus will also address the regulatory issues facing the pharmaceutical, biotech, medical device and medical foods industries.  And we would like the congressional caucus to also address access to care, pricing and reimbursement issues.  
 

       This is a big agenda.  You know those who work up on the Hill know that there are a few other patient caucuses – usually for big diseases.  There is a cancer caucus.  There is a heart caucus.  There is a kidney caucus.  There are a few others.  We heard earlier from a representative from the Cystic Fibrosis Foundation and CF has its own caucus.  But there is none that now is the umbrella for all those patients who have rare disorders and now they will have a home on Capitol Hill.  (Applause.)  By that reaction, Peter, I would say that NORD has another winning idea.   
 

       Now it is with great pleasure that I get to introduce my friend and a friend of all patients with rare disease, Mike Astrue.  Mike is the current administrator of the Social Security Administration.  Dr. Hamburg probably would like to have this, too, as she steps into her role as commissioner.  He has a six-year term.  I think the last FDA commissioner that was there (that long) was probably Dr. Kessler.  But we have the great privilege of having Mike in that post for six years.  I say privileged because Mike has had a distinguished career.  He understands how the federal system works.  He was general counsel of the Department of Health and Human Services.  In that capacity, he was looking out for patients with rare diseases.  He prosecuted successfully the first discrimination case that was against a person with HIV.  Do I have that right, Michael?  So Michael has done innovative work in the federal government.   
 

       We were talking today about public-private partnerships; we heard that phrase many times.  Michael understands that in his very fiber because Michael  was general counsel of Biogen and helped develop an orphan drug, Avonex, for multiple sclerosis.  And then Mike was very successful in taking on the leadership as CEO of TKT, as it helped to develop a product for Gaucher disease.  I am going to let Mike explain to you what he has done through his vision at the Social Security Administration in terms of reaching out and doing things very creative for people with rare disorders.  
 

       Often, those of us in different positions, whether we are at NORD or we are in the federal government or in a company, we get credit because somebody else on our staff had a good idea, developed it, a team worked it up and led it to fruition and that because we were the one at the top, you get the  kudos for it.  I want to say that this has been Mike’s idea because this is just fabulous.  We know everybody who has rare diseases and we all know those people.  And the thought of trying to get a person through the Social Security disability system without having to go through an administrative law judge, without having to take a lengthy period of time, it is all because of Mike.  So Mike?  
 

       (Applause.)  
 

       MICHAEL ASTRUE:  Frank, thank you very much.  And I want to thank Peter for inviting me to this very impressive event.   I came in and caught the very end of the session today – the kind of energy that it is in the room.  And I just want to say before I start talking substance, I am just having a blast.  I have got so many friends from different parts of my life over the years who are here that it just is like a college reunion.  (Laughter.)  So I am having a very nice time being back.  
 

       I am probably guilty, as a lot of people, when thinking about orphan diseases, thinking primarily about drug development in the orphan drug process.  And, of course, I spent 14 years of my life in the industry and then a certain number of years as part of the problem in the federal government (laughter).But I worked on the first orphan drug regulations and probably was guilty  for many years of thinking about it primarily from that angle.  But,  I had the experience when I was first working at HHS in 1985, my father came down with the same form of rare brain cancer that Senator Kennedy is battling now, and so handled the Social Security disability process, formed the whole application process.  
 

       And, sometimes you almost think like God is talking to you on certain things. I allegedly was this person who knew a lot about this.  And I did in sort of an abstract sort of policy way.  But when it really came to grinding through that process, I said, geez, there has got to be a better way.  Unfortunately, I learned more and more about the disease.  One of the advantages of being at HHS is that you can talk to people who know a lot.  And Vince DeVita, who was head of the National Cancer Institute, actually sat me down and gave me the very grim prognosis, particularly in 1985, for that disease.  
 

       And so I had two thoughts.  One thought was do we really need all of this paperwork?  And, of course, that is a refrain that my staff hears all the time.  And we work on simplification all the time.  But a thought that I had, which I thought I would never have the opportunity to do anything about was, why do we need any process at all for some of these diseases.  I mean, if you have got a diagnosis that is as certain as this one and you know what the track is, why do you have to go through the five-step process?  Why do you need the 15 years of documentation?  Why do you need all of this?  There should be categories of people where  there should just be a conclusive presumption, where you confirm the diagnosis and you say we are sorry, your check is on the way.  
 

       And it is one of those things you sort of file away and you think about it in an ideal world and then you go on to other things.  And so for two decades, I went on to other things.  And then sometimes amazingly, you come back full circle.  And so in 2006, when I started talking to the Administration about this position, you also get a little bit overwhelmed by the scale of it all.  This is an agency of approximately 65,000 people.  We will take in this year almost 3 million disability applications.  
 

       And it is a little daunting.  And it has a lot of smart people. You figure, well, what can you really do that is going to be successful?  One of the advantages I have had is being somebody who was with the agency in his reckless youth and then went away and looked at things from a different context.  And I was blessed to work with some tremendous managers when I was in the biotech industry, particularly Jim Tobin, who now runs Boston Scientific.   
 

       When I came back, I could see something that all of the incredibly smart people who worked for me couldn’t see, which is that we were captive to longstanding processes that were based on paper. What they did was absolutely brilliant, thought through, state of the art for a paper process. It had been underfunded, moving slowly and very incrementally into electronic processes.  So first of all, that that has to accelerate if you are going to serve people well.  But it also changes the paradigm.  You can do things that you couldn’t do before.  
 

       So what we have been trying to do to make the thought of 1985 into reality. It wouldn’t have been possible in the paper process because just the act of going through that much paper – we are the largest repository of medical records in the world, by the way.  And just to do the triaging is so labor intensive that it ends up being counterproductive in a paper process.  But the marvelous thing is that we have really made our disability-determination process substantially electronic at this point. It now allows us to go back and say we are not captive to a lot of the restrictions and rules and things that made sense in a paper process.  And we can do things differently and not only more efficiently, which is what all the pressure is on – the year-to-year budget cycle.  But you can pull back and say, can we actually do things better?  Can we do things that weren’t possible before?  
 

       So we had a very small pilot tested up in just a few states in New England with a planned 10-year rollout that would pull out cases for triaging, but really just highly probable cases.  And that was a good thing.  And that was something where we could make that much more robust and we could roll it out much more quickly, which is what we have done.  So we have something called QDD, quick disability determination, where we take the cases that are highly likely – and we still have to go through our five-step process, but at least we put them at the head of the queue.  And we can decide those cases extremely quickly.   
 

       But we had never embraced the notion that we could essentially have categories of diseases or conditions that would essentially be presumptive, where basically our only job was to confirm that someone was eligible for Social Security and that, in fact, there was a correct diagnosis, which saves us an enormous amount of time and effort.  So we created a category called compassionate allowances.  And  it is not necessarily a restriction of the system, but I think pretty much everything in compassionate allowances is, in fact, a rare disease and pretty much everything that comes out in the QDD model is also well under the orphan drug guidelines, even though we don’t explicitly restrict it to that list.  
 

       So first we started with the QDD model and we made it more robust and expanded it.  And we have pushed that out nationwide.  And then about six months later, we got a parallel system for essentially an even faster, more presumptive category called compassionate allowances.  As we speak, we have about 4 percent of the 3 million cases that are decided this year will be decided in an average of 10 days.  That will be about 100,000 to 125,000 people.  They will basically be all the people with rare diseases and conditions.  And they will be picked out.  And they will get benefits much faster than before.  
 

       We have made our typical process faster in the last two years through a lot of the same tricks and approaches, about 4 percent each year.  But just to get a first-level decision – and Frank made a polite reference to our rather cumbersome four-step, increasingly legalistic process that we have in the statute.  Right now it is about 85 days on the first step, about the same for the second step.  And then when you get into the legal process – although we have brought it down quite substantially, on my watch, we are still averaging just under 500 days just to get a hearing.  So it is a long process.  
 

       When I came in, there was, in fact, some institutional resistance to going this route.  And I think some blindness within the agency as to whether we had a problem because it is not only the speed. I had a deputy commissioner who is not with us anymore say, we shouldn’t bother doing this, that these cases are easy.  We make the correct decisions and why are you bothering with this?  This is a distraction and we should be doing these other things instead.  And I said – rather than just exercising commissioner’s prerogative – although that is a great thing – (laughter) – I actually realized that they were just doing it to humor me.  Ultimately, to get something done well in an organization as large as ours, you have to get people to believe.  
 

       So we brought in our quality people who were viewed as the neutral brokers within the agency to do some retrospective studies to see how well did we really do in practice.  And we generally have about a 3 percent error rate in our cases.  We found for the categories that I picked out – likely compassionate allowances – our error rate was 20 to 40 percent, and that the more obscure the disease and condition, the higher the error rate was.  I mean, the 20 percent was for ALS.  How can you blow ALS in our society?  But we were.  And part of it is we have a system where the first two levels are done at the state level.  We have young, low-paid people; turnover is high.  The typical examiner has less than three years of experience.  So they are struggling with the whole huge spectrum of every disease or condition a person can have.  And how does it fit into our incredibly arcane and complex definition of disability?  
 

       So when you think about it in that way, it is not unusual that cases were being missed.  In addition to cases being decided much faster, a fairly significant percentage of people were not going through the agony of having to go through extended process because we messed it up the first time.  And so those are good things.  
 

       Right now, about 3.5 percent of the cases are QDD, 0.5 are compassionate allowance.  We have in our strategic plan a goal of getting to 6 to 9 percent by 2013.  The goal for this year is to get it up to 4.5 percent.  We have been a bit insular sometimes in taking a huge amount of medical information  and trying to translate it into standards.  So we are reaching out to groups more.  We are having more hearings.  We did four hearings on different areas last year.  We have had wonderful cooperation from other agencies.  NIH has been heroic at giving us people to sit on these panels to help us go through this process.  We had some great help from DOD when we did traumatic brain injury.  
 

       And we are in the process now where we probably have picked a lot of the low-hanging fruit.  It is going to get harder and harder, so we are looking at some ways to let some contracts to go more systematically to add as many diseases and conditions as possible.  We started with 50 on the compassionate allowance list.  And our goal is to drive that substantially higher.  
 

       Sometimes the labels that help us understand the world are limiting, too, and particularly when you look at the area of mental diseases.  And no disrespect to the medical profession, but if you look at the nomenclature of 50 or 100 years ago, we were talking about melancholy and how that differed from other categories that we don’t use anymore.  And now you look at anxiety, depression, other things because it is very sort of fluid lines.  So what we are trying to do next, rather than look at specific disease categories, is to try to look at some broad spectrums of disease, where you are really talking about clusters of disease and where we may be able to break out subcategories from a larger label, where right now all our listings say one size fits all for all purposes – see if we can break out some subcategories.  
 

       We have a hearing coming up on schizophrenia and a hearing coming up on Alzheimer’s.  What we are hoping to do is look at those categories differently than we ever have before.  So the big mission is to take this new structure, drive it through, expand it, push it as far as it can go.  And I think that there are some people who are actually pretty tired right now who think then we are going to stop.  And they have a little surprise (laughter).  
 

       A lot of handholding and referral and amazing work gets done in field offices.  We are communicating with the American public 350 million times a year, which is what we do just in writing.  That is not even the personal and telephone contacts.  And an increasing amount of this is electronic.  You actually now have opportunities in a very cost-efficient way to provide people with information so they can help themselves in ways that would not have been possible with our old paradigm of paper and personal interviews.  
 

       We are looking now for disability claimants directly and then moving down to looking at the special needs of people with rare diseases so that when they file that application, which will be increasingly electronic over time  we ask is there information we can give them in that notice?  Or is there a specially designed Web site we can send to them that will tell them things that would be helpful because unfortunately, we have all kinds of gaps in health care in our nation.  And as much as we are going to try to fix that in the next couple of years, we are probably not going to have it down perfectly.  And disability benefits are the gateway to public healthcare benefits in our country.  It is a precondition for Medicare or Medicare.  And a lot of times, that is really why people are applying.  That was true in my dad’s case.  I mean, the money was nice on the disability benefit, but what was really panicking the family was the concern that my dad’s medical bills were going to bankrupt my mom.  
 

       So giving people information and particularly, for people with rare diseases, a lot of time – as I think most of you know, the earlier you get in with therapy, the more you can impact the course of disease.  And, if we have a process making our decision and then there is a 24-month wait for Medicare, then a lot of people who are in a critical stage can go downhill. The best thing for them and the best thing for the country is for them either not to become disabled in the first place or to become as functional as possible.  So we have got some barriers that we are looking at legally in terms of where we can refer people to. This is sensitive in terms of referring people to corporations that can provide benefits.  But we are  trying to break out of the old paradigm and figure out if there are some highly efficient ways for these populations that are coming to us for help can be provided with information, with different types of assistance than we have ever been able to do before.  
 

       So that is new and exciting. And I think that it is going to take us a while to get there.  One of the problems is we have 38 million lines of COBOL code.  And for those of you – (laughter) – who are IT people – moving out of that is long and slow and expensive, although it has certain benefits.  We actually have been dumping a lot of money into the Maryland education system to train people on COBOL.  When they come to work for us, it is basically a way around the 13^th amendment.  You can’t leave Social Security once we have trained you on COBOL code – (laughter) – because there is no other place you can go to work.  (Laughter.)  But it is mindblowingly long, slow and expensive to change and has other risks as well.  So we are moving away from that as quickly as we can to a much more modern system.   
 

       So  as that platform changes, it is going to open up even more opportunities, so that when a disabled person or even a close to disabled person, even someone that we are going to reject, comes to us, we can give them a prompt decision without a nightmare’s worth of paperwork.  And in the process, whether we end up giving them benefits of not, we can supply with them with a lot of information that will make their life better and make the public health better.  So that is what we are all about these days.   
 

       I appreciate this opportunity to be back with old friends and tell you what we are doing.  And I would be happy to take any questions anybody may have.  Thanks.  
 

       (Applause.)  
 

       MR. SASINOWSKI:  (Question not recorded.)  
 

       MR. ASTRUE:  About 25 of them (diseases subject to the compassionate allowance program) are different forms of cancer and about 25 of them are independent diseases.  So on the cancer side, it is probably not a surprise that an astrocytoma is on the list.  We have got ALS on the list.  We have got some of the lysosomal storage diseases, which I think many people know are close to my heart, on the list. 

       (The SSA staff) has reached out not only to this group (NORD) and to NIH, but to a lot of the medical associations.  We can’t do this on our own.  So the help that we are getting is important. 

       One of the things that I thought was scandalous was how long it has been since some of our key regulations had been updated.  Medical advances change.  So, for instance, we have this odd metaphysics, where we divide the world into 14 body systems that it strikes me as a little bizarre, but it is probably not worth my effort to try to undo that.  So we have one that is basically GI tract, liver and colon.  And we hadn’t updated that since 1985.  My mom kept telling me when I was growing up, given that I was type A and I liked Mexican food, I was sure to have an ulcer.  
 

       My mom’s assumptions were based on our listings (Laughter.)  And the early ’90s, we knew that it was actually caused by bacteria; antibiotics could take care of it.  And we paid no attention.  And so that was a problem.  I can’t really fault people for this, frankly -- but under the old paper model, we had such high volume and it was all paper.  There was an attitude that, we just don’t see so many of these types of cases.  It is not worth the effort to do the listing.  And at some point, too, when the regulations got like this, you would overwhelm the 26-year-old examiner in the DDS who couldn’t find it anyway.   
 

       But the great thing about modern technology is they don’t even have to look it up anymore.  We are moving to a system now.  It started last year.  We got it into five states now.  We will have it around the country mid-2011.  And so it basically compensates for a lot of this.  So when a young examiner is looking at a case, the computer will have already scanned it.  It will already know it is a cystic fibrosis case combined with depression.  And it will pull out all of the regulations and our listings relevant to cystic fibrosis and depression and make the person answer the questions and fill in the document properly.  
 

       So once you move to that kind of paradigm, you just have to kind of do this once and it doesn’t matter that the rules are this long and they may be hard to go through manually because the computer will just pick it out and they can search that way.  So that is kind of also part of, again, the paradigm shift, where moving from paper to electronic just allows us to do things radically different from the way we did it before….  
 
 

       Q:  Commissioner, I want to thank you, first of all, for compassionate listings, as I had the opportunity to testify for one of your panels.  As an attorney who has worked with Social Security conditions for 15 years, I certainly see the difference in the compassionate listings and I very much look forward to expansion.  I also have seen a difference in the agency’s willingness to reach out to the organizations and create training materials.  And I sure hope that will become much more far spread.  I think that has been really great, as well as the video access for people who are unable to make it to help facilitate the hearings.  
 

       I still put out my desperate plea that we create some sort of advocacy for the unrepresented, especially with rare conditions.  My goal would still be to see something where there is a joint public-private industry panel that people could reach when they have a condition.  It might as well be yellow ear disease because it would be that little understood within Social Security and that we establish more help from the voluntary health organizations to the Social Security Administration, so that should we have a problem with the condition that they don’t understand, that doesn’t meet the listings, that there is a place that they can go.  
 

       I also want to thank you for the talent and expertise of Diane Braunstein and her staff because I certainly think that they were amazing in what they managed to accomplish.    
 

       MR. ASTRUE:  Well, thank you.  Thank you very much.  I think the issue of representation is an important one.  History maybe traps us a little bit.  A lot of the features of the system were built on the assumption that this was a non-adversarial system that was not going to be lawyer bound.  But there was an immediate tension. Congress continued to make it more and more complicated.  
 

       When I did my first disability case as a federal law clerk in 1983, we were still really in the 1957 paradigm.  It was basically a system for white males over the age of 55 in blue-collar jobs that came in with one of three or four very typical blue-collar entries.  And that is really what I think Congress thought it was doing in ’57 when it started up the DI program.  
 

       But, our notion of disability has changed, just exploded, and I think that is an extraordinarily good thing.  So we not only do disability for the full range of diseases and conditions for adults, a lot of people don’t realize that for low-income children, we also do it for children.  And, of course, that poses a whole different set of very difficult questions and sort of standard setting because a lot of diseases affect children differently than adults.  And for adults, a key part of the standard is a vocational standard.  And, you know, even very conservative Republicans don’t talk about putting eight year olds to work. (Laughter.)  We believe in the child labor laws and that kind of thing.  So we have to figure out sort of alternative ways of handling that.  
 

       So it has gone from a very simplistic model, where the non-adversarial model made some sense – maybe it was doomed, but made some sense – to something that is very different.  So now about 85 percent of the claimants are represented.  And it is a fair question that maybe we ought to be thinking about doing something a little differently, so the other 15 percent can get representation.  Right now I think a lot of times it is an economic choice because the attorneys fees, as you know, come out of the past due benefit and some claimants don’t want to take that risk.  But that is probably fair.  
 

       I also think that we are trying to get the agency to open up and communicate better, be more transparent, accept information, give information.  It is a different model than what we are used to.  And thank you for the praise for Diane Braunstein, who more than any other person drove the details of the compassionate allowance initiative and has done a great, great job and is a wonderful human being.  I think that we have got the policy-makers in that mode.  And I think that is consistent with what you are saying.   
 

       I think where we are struggling is still a little bit more on the day to day.  And again, it has also been sort of a resource issue.  We have a general problem in just accepting phone calls.  The agency in field offices just basically shut off the phones 15 years ago because it couldn’t handle the volume of work and diverted people into 800 numbers.  
 

       When you are in that kind of mode, it is going to be very hard and slow moving off of that kind of model.  Having the kind of human interaction that you are talking about is difficult, but we have got to keep trying for both actual real people and electronic, if that is the substitute.  One of the things that will help enormously is we have almost got our hearings and appeals – in addition to everything else, we run the largest system of justice in the world with the appeals.  We have about 650,000 cases pending right now.  One of the sources of frustration for the people that are advocating for people is that the people trying to deal with the huge backlogs don’t have the time to pick up the phone and tell them the status of the case.  And, of course, in the paper paradigm, can’t find the case anyway.  (Laughter.)  
 

       So we have moved to an electronic system now on hearings and appeals.  It has its flaws.  It is not perfect yet.  It is going to take a couple more years to refine.  But we are close to being able to allow direct access to the folders, so that people can actually see what is going on, see what is happening, if there is a problem. Actually, it will make it easier – it will free up time in the offices where people don’t have to deal with the routine calls.  So if someone calls, it is much more likely because it is not something routine.  It means something is off track.  And I am hoping that they will be much more willing and able to take the call in that situation.  So the first round, I think, is due in October or November.  And then the second round of those changes is in March or April.  
 

       So it will take a little while to get the bugs out.  But it should start feeling better, at least, in the hearing offices probably by the middle of next year.  
 
 

       MR. SASINOWSKI:  I want to invite you to join me in thanking Commissioner Astrue.   
 

       (Applause)  
 
 

       (END)