I am one of many who worked long and hard to get this enacted and funded. It seems essential to me that we have an excellent epidemiological inventory of cases of ALS so that scientists can better understand the disease. Over decades we had buried the clues along with thousands of people with ALS.
Our government is good at census work. It knows how to count noses. One excellent disease registry is hundreds of times more valuable than a dozen independent, less complete registries. Add the fact that most people with ALS go quickly into various government databases such as Medicare and Medicaid, for the U.S. government to be the chief nose-counter for people with ALS made so much sense.
It took years to get this passed. People with ALS and their families were willing and able to fight for funding at a "Cadillac" (a word used by a man with ALS at an advocacy meeting) level so that the CDC could do the job right. When the ALS Registry Act passed, the CDC even said that it was a well-funded project. Hindsight is 20-20, but would a lean project have been better?
Here's the most recent pitch -- http://library.constantcontact.com/download/get/file/1113068431369-11/2013+ALS+Advocacy+Day+Talking+Points.pdf
Here is the melange we think we got for our $33,000,000 so far. We think.
The Basic Data Surveillance
Data availability time lags and some kind of secrecy purported to be required by OMB keep us from knowing exactly what the government files indicate about incidence and prevalence of ALS, but we hope that something is reported later this year, $xx million dollars later.
The Patient Self-Enrollment Portal
This was added to the project to provide people with ALS a way to step up and make sure that they are counted. The portal is there. Where are the patients? What is the uptake on self-registration? What percentage of the cases detected by passive surveillance also self-enroll? What is the communication process to insure that patients are aware of and understand the value of self-enrollment? Whose feet are held to the fire to insure that patient self-enrollment happens? So many questions, so little accountability. This isn't a "build it and they will come" proposition.
Last year a man with ALS delivered this information to the advisory group - https://docs.google.com/file/d/0B5nmEImgaRfudXc4ay1zUGNQa28/preview?pli=1 Has anything changed? He died. It's up to the rest of us to ask for some accountability. If the same survey were administered today, would anything be better?
And what in the world are the details of this contract? https://www.fbo.gov/index?s=opportunity&mode=form&id=31776989bc48abbd46ddbe5dd2231b41&tab=core&tabmode=list&=
The Risk-Factor Surveys
The self-enrollment portal could also provide a route for continuing engagement from willing patients to respond to risk-factor surveys. That seemed like such a good idea to add onto the basic Registry, too. So far we have learned that smoking is a risk-factor for ALS. Does that advance the science? Do patients even know about these surveys? Do neurologists? New surveys are awaiting review by OMB. What is OMB looking for? We even learned in May that the CDC will use budget money left over to pay scientists to suggest new risk-factor surveys. Would a few of the smart scientists on the Advisory Committee not be able to rattle off risk-factor topics that would be as good as any paid government contractor? Why do more surveys if the uptake on the current surveys isn't good? What is the communication process on these?
The Booth and the The Tchotchkes
Shortly after Registry launch, we saw a big trade-show booth that would be shipped to various advocacy and neurology meetings (along with sponge baseballs). Are the booth workers trained to be on their feet and engaging with a five-second message that will stick? What's the goal? How does this fit into a larger communication plan that will have measurable results?
The Registry Medallion for Websites
Great idea, but whose job was it to contact websites to ask them to add the medallions? It seems that a few rogue people with ALS and advocates contacted many ALS-related websites that were glad to add the medallions and had not been asked to do so by the CDC or by ALSA or by the MDA. Ah, communications.
The Website www.cdc.gov/als
It is actually less cluttered than it was a year ago, but the calls to action to patients are still not clear. As one advocate said, "It looks like a website designed by a government contractor."
The Clinic Locator
Why, oh why not just put links to organizations and agencies that maintain their own resource databases? Why add a search mechanism that will require constant data maintenance? Oh, and if you're curious, try zip code 46556, a zip in Indiana that is near the Michigan border and Chicago. It's a great test zip code to see if resources in Indiana, Michigan, or Chicago show up. Pffft. Of course, if you use an iPad you'll not realize this since it doesn't work at all for you. This service locator was unnecessary and is not particularly good and will require continuing data maintenance. Cha-ching.
The Continuing Medical Education Modules
We certainly need more ALS education among healthcare professionals, but was this the right place to deliver it? And again, what is the communication process to engage those who would benefit from this training? Are we engaging healthcare professionals who need to learn about ALS, or are we providing CME to people who already work with ALS? Just because the CDC had the ample budget to let them deliver these doesn't mean that it was the best organization to do so. Are we slipping into the mode of having the CDC handle whatever projects our ALS organizations can toss their way?
The Video Tutorials on The Registry
How about fixing the underlying usability issues rather than adding expensive layers of tools to help people fight through the usability issues? Would you ever buy a book at Amazon if you felt that you needed a video to show you how?
The Android Tablets
Tablets were supplied for ALSA clinics to assist PALS in self-enrollment. Is there a process to insure that they are used appropriately? Are there measures to see how well the process is working? Did we throw money at a problem again without understanding its roots?
ALS Research Notification
Here's the scoop on this new Registry accessory -- http://wwwn.cdc.gov/ALS/ALSClinicalResearch.aspx
Again, just because you have the budget to do something doesn't mean you're the best person to do it. Doesn't clinicaltrials.gov have better, more comprehensive information than the CDC is building separately here? Do we need yet another database and administrative process? is clinicaltrials.gov not working on gui enhancements as we speak? Are we not building another data resource that is duplicative with existing resources and that will require ongoing maintenance? And is a poor precedent being established in leading people with ALS to believe that they will be informed when pertinent research opportunities are available? Rather, they may be informed when some research opportunities are available. They are still responsible to seek and find the others.
The Biorepository Feasibility Study
My a-ha moment was when a gentleman with ALS said, "I feel like I've left tissue all over the country." Surely tissue samples are critical for scientists to understand ALS. Will the huge biorepository project being proposed by the CDC (as another accessory to the basic Registry) fix the problem of a patient's tissue being left all over the country? Is anyone thinking about having the Registry have the master directory of bio-samples of people with ALS so that tissue stored in any number of existing repositories could be indexed and not duplicated by the CDC's respository? The government is good at tying together information about our bank accounts. What if the CDC built something to pull biorepositories together rather than form yet another silo of ALS tissue and information? At the recent ALS advocacy conference, we learned that the CDC was learning from the experiences existing biorepositories, but nobody said that the CDC would be working with existing biorepositories to prevent expensive duplication of efforts and tissue and data. Patient #50239483226's brain is in a biorepository in Chicago, his blood/sweat/tears are in a refrigerator at the VA, and his son's blood is at the NIH. Is anybody thinking along these lines, or will the CDC just build a bigger silo in our growing countryside of biorepositories?
The Burden-of-the-Disease Project
Again, this is a topic in dire need of quantification. Biogen Idec undertook a similar project recently. Again, is the CDC's Registry the best place to deliver it?
If only we had confidence that the basic counting of noses of those with ALS was being done accurately and completely. If only we had confidence that all of the extras were contributing to the core mission of the Registry and not distracting from it. If only we had some measurable results to tell us.
We hope for timely minutes of the next meeting of the Advisory Committee and we hope that these experts see the value of sticking to one's knitting. We hope some advisors will challenge the scope creep. Nobody gives a government agency a prize for not spending all of its budget, but maybe we should.