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.

Monday, May 9, 2016

It's That Time Of Year For Some #ALSadvocacyday Qs ... And We Hope, As

 Questions kept popping into my mind as I listened to the kickoff comments for ALSA's annual public policy conference yesterday.  I write them here in hopes of hearing answers today.

ALS Registry

  • What is the cumulative taxpayer investment to-date on this project, starting back with the so-called "building block" projects that we were told to pitch even prior to the passage of the ALS Registry Act in 2008?
  • How are you going to spend the next $10 million?  For example, how much is just basic administration and support?  How much is required by the new biorepository project?  How much goes to research grants?  How much is paid to project contractors individually?  What does it cost to administer research notification? etc.
  • Has anyone actually tested the ALS Service Locator?
  • How many unique people with ALS were seen by ALSA clinics in 2015?  Of those, how many have self-enrolled in the Registry?
  • How many unique new patients were seen by ALSA clinics in 2015?  Of those, how many self-enrolled in the Registry?
  • Does anyone have data on whether people with ALS choose Medicare Advantage plans at a greater rate or lesser rate than the general public?  We learned several years ago in the Registry annual meeting notes that Medicare HMO records are missing from the government files that are mined as part of the Registry algorithm. Those are people on Advantage plans, right?  According to a recent study from kff.org, the national average to opt for Advantage plans is 31% with some states being much higher.  Minnesota is 53%.  Florida and Pennsylvania are 40%.  And there are clusters in some urban areas.  The original ALS Registry pitch was that the CDC could identify 85% of ALS cases via the passive data mining of Medicare, Medicaid, VA files; however, this HMO problem indicates that you are missing some large percentages of people with ALS from the Medicare files. Is this not a problem?
  • When will the resampling study to help ascertain completeness of the Registry be published?
  • What percentage of the enrolling interventional trials for ALS at clinicialtrials.gov are included in the research notification Registry feature?
  • Are biosamples available to researchers right away, or do the data from government files have to catch up to a person's biosamples to confirm that person's case of ALS before the biosamples can be released?
  •  Volume of email sent really isn't a great measure of effectiveness. How many people have actually inquired about a trial or study as a result of having received an email from the Registry?


Drug Approval Paths

  • This is an important month for drug approvals for all fatal, unmet-need diseases as the FDA ruminates on eteplirsen.  Why is the ALS Association not standing loud and proud with arms locked with the parents and DMD kids who are awaiting the Accelerated Approval ruling from Dr. Woodcock?
  • What is the ALS Association's position on all three pieces of legislation being promoted at www.myrighttotrynow.com ?  In particular, why is the Regrow Act not one of the "asks" for this #ALSadvocacyday ?
  • Wasn't the timing of the Lance press conference at the exact same time as the MyRightToTryNow legislative briefing an unfortunate coincidence?
  • Could you please repeat the source of the new report that is to be announced at the Lance press conference tomorrow?  It was difficult to understand in the comments yesterday.

Thanks to anyone who can help with the As.




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