ALS Advocacy

ALS Advocacy
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.

Friday, May 2, 2014

Help! Someone, Please Help!

Eight years of advocating for a national ALS Registry.

I felt and still feel strongly that we need a census of cases of ALS.

$40 million taxpayer dollars spent.

This is the spring when we finally expect to see some numbers.

And so far we see a flock of poster sessions from this week's AAN meeting.  Here are a couple.

I feel like I've been spun around a few times and the blindfold has been partially removed.  Now I certainly need some help with some questions.  My comments and questions are in hot pink below.  These are things that we need to understand.  I think we've earned that right.

Using the National Amyotrophic Lateral Sclerosis Registry as a Subject Recruitment Tool for Research (P5.077)

  1. Paul Mehta1,2
  1. Neurologyvol. 82 no. 10 Supplement P5.077
Objective: To describe how the National ALS Registry links persons with ALS (PALS) to scientists who are conducting ALS research.Background: Subject recruitment for research is critical for understanding inherently fatal diseases like ALS, however, linking patients with researchers is not so straight-forward. Researchers often face challenges in timely recruitment, obtaining sufficient sample sizes, and/or in enlisting patients that meet certain eligibility requirements. Similarly, patients can face difficulties in finding specific research studies for which they are eligible.Design/Methods: The federal Agency for Toxic Substances and Disease Registry’s (ATSDR) National ALSRegistry now links Registry-enrolled PALS with external scientists who are conducting ALS research. TheRegistry’s new online Research Notification Mechanism allows ALS researchers to upload an application that briefly describes their research study and objectives, while PALS can elect to be notified about these opportunities.If the application is approved, ATSDR then queries the Registry for PALS meeting the study’s specific eligibility criteria (e.g., age, sex), Does the registry have onset date? and then distributes the researcher’s study material and contact information to PALS via email. PALS have to directly contact the researcher to take part in any research.Results: Since the Research Notification Mechanism’s May 2012 deployment, approximately 96% of Registry enrollees have elected to be notified about ALSresearch opportunities. Additionally, ATSDR has approved a number Do we know what the number is? of institutions to use the Registry for research recruitment, sending out thousands Do we know how many thousands? of e-mail notifications to PALS Do we know how many PALS have been targeted? on behalf of the recruiting And do we know what percentage of the open recruiting clinical trial seats were represented? institutions. And do we know the most important metric of all, how many clinical trial seats have been filled because of this mechanism? Conclusions: The National ALS Registry’s Research Notification Mechanism is an effective ??? tool for linking PALS with ALS researchers who are conducting epidemiologic studies or clinical trials. The Mechanism benefits PALS by conveniently delivering timely and tailored research opportunities via email. It also benefits researchers by helping to speed-up the recruitment process, increasing the study sample size, and efficiently identifying PALS meeting specific eligibility requirements.  Do we know it actually sped or increased sample size in any studies?
Disclosure: Dr. Horton has nothing to disclose. Dr. Antao has nothing to disclose. Dr. Mehta has nothing to disclose.
Wednesday, April 30 2014, 3:00 pm-6:30 pm

Amyotrophic Lateral Sclerosis (ALS) Estimates from National Databases in the United States - 2001-2010 (P2.072)

  1. D. Kevin Horton1,2
  1. Neurologyvol. 82 no. 10 Supplement P2.072
Objective - To present preliminary ALS estimates from national databases in the United States.Background - The uncertainty about the incidence and prevalence of amyotrophic lateral sclerosis (ALS) in the United States, as well as lack of knowledge about the role of environmental exposures in the etiology of ALS, have created a need for data collection through a national ALS registry. In 2008, the Agency for Toxic Substances and Disease Registry (ATSDR) conducted four pilot projects to determine the feasibility of creating a national registry. It showed that approximately 80% of ALS patients can be found through national databases. The first approach utilizes existing national administrative databases to identify prevalent cases. The second approach uses a secure web portal to identify cases not included in the national databases. This latter approach allows patients to self-identify and enroll in the ALSregistry and take risk factor surveys. Design/MethodsWe sought to identify U.S. residents with ALS in Medicare, Medicaid, Veterans Health Administration (VHA), and Veterans Benefits Administration (VBA) databases for the years 2001-2010. Records were searched and identified in these databases for ALS and Motor Neuron Disease (MND) codes.Results - A total of 147,889 individuals, with any MND code Are those distinct individuals? So over a period of nine years, 147,889 distinct MND individuals were identified?, were identified across the four national databases. Medicare contributed the largest number of individuals. The total number of individuals identified having ALS was 36,547, which is 24.7% of the total number of individuals identified with any MND. I always thought that ALS was by far the largest subset of Motor Neuroon Diseases.  Am I wrong?  Do they really think that over 75% of the MND cases are not ALS... or did they just fail to identify a lot of MND cases as ALS?  Conclusions - This is the first effort to identify ALS cases for a national registry. The number of individuals identified via the national administrative databases is not a prevalence estimate Why not?; however, it does indicate that a large portion Based on what assumption? of ALS individuals can be identified for the National ALS Registry by using the national databases selected. How do you arrive at that conclusion?  It sure seems like a jump to me!  
Disclosure: Dr. Sanchez has nothing to disclose. Dr. Mehta has nothing to disclose. Dr. Kayehas received personal compensation for activities with McKing Consulting Corporation as an employee. Dr. Antao has nothing to disclose. Dr. Horton has nothing to disclose.
Tuesday, April 29 2014, 7:30 am-11:00 am

1 comment:

  1. Is the problem one with ICD codes?... where some records are recorded at the summary MND level and others are recorded at the detailed ALS level? Such was perceived early on in the Registry because I believe that death certs are encoded at the MND level.