It's Time For Some Three-Point Shots

For over a year I've been scratching my head about the MODDERN Cures Act.

http://als-advocacy.blogspot.com/2013/04/these-are-my-questions-du-jour-on.html

Recently an outstanding advocate/tweeter/patient/person @SDFatPhRMA put me in touch with the folks at the Campaign for Modern Medicines modernmedicines.com who have gone above and beyond the call of duty to help me understand MODDERN Cures and exactly what's in it for people with ALS.  I am grateful.

As we perceived years ago, the dormant therapy features of MODDERN Cures are the most interesting for the fight against ALS.

Here is a blog from the Campaign for Modern Medicines that answers most our questions --

https://modernmedicines.com/entry.php?id=486

And here is a recap of some salient points for those interested in the fight against ALS --

Are these "dormant therapies" only things that never made it to market for another indication?

That is correct.  Think about things that didn't get through FDA approval for their original intention.  Those things that are sitting on shelves in labs somewhere could have their patent "clocks" reset so that there would be incentives for drug developers to try them on ALS.  MODDERN Cures would not apply to a lithium or a ceftriaxone, but it would apply to something that never made it through FDA approval before. 

How does it work when something is already off patent?  

There are two things that are really important in this case -- patent protection and data protection. 

The MODDERN Cures Act aligns patent protection and data protection at the date of FDA approval. 

Even if there is no patent protection left, the drug receives data protection.  A generic or biosimilar manufacturer cannot generally receive FDA approval to launch a competing product during that protection period.  If a competing manufacturer started clinical development of an off-patent molecule before the first manufacturer received FDA approval, then competing products could also be approved.

Can anybody pick up a potential drug and run with it, or does it have to be the company that owned the rights when it was on patent?

If it still has any patent life, an interested scientist would have to work with the patent holder on a license agreement; otherwise, scientists, please go for it!

OK, what happens when something has just a little patent time left?

The MODDERN Cures Act allows the patent clock to be reset during the development process and run from the date of FDA approval. This is a big incentive.

We know that it's going to take a lot of shots on goal to finally find some therapies for ALS.

We need for drug developers to make smart (and long) shots.  If something is sitting on a shelf with little or no patent protection, we owe it to people with ALS to make it viable for drug developers to pick up one of those oldies and shoot.

Many thanks to the folks at modernmedicines.com for their time and help.  If I got anything wrong, I'm all ears.  

Finally I think we have some clarity on what's in it for people with ALS.

Why?

That's the best title I can come up with for a post I never expected to have to write.

Last year some of us stumbled across some large no-bid government contracts given to ALSA and then to MDA from the CDC ATSDR ALS Registry funding.  The MDA's contract appeared in September and the amount, $2,695,642, floored some of us.

On October 18 I emailed a question directly to the head of the project at ATSDR who is a frequent speaker to groups on the Registry project.

Dr. Horton, Are you able to tell me what MDA is doing for this sum, please?

 https://www.fbo.gov/index?s=opportunity&mode=form&id=c7856e6375ef7f0fcc2c4cb47ae176a0&tab=core&_cview=0  

I have a bad case of sticker shock.  

Thanks very much for any insights.

On October 21, I received a reply from Abby Martin of the CDC.

Neither Mr. Horton or I have the authority to respond to requests for public information not in the public domain.  (This includes any request for information via social media.)  All such requests should be directed to the CDC/ATSDR Freedom of Information Officer for a response 

Okeydokey.  I learned how to submit a Freedom of Information Act request.  The following two requests were submitted and accepted on October 22.

Request 1 I would like to know what the work product, deliverables, and performance standards are for the work in the MDA contract for “ALS Outreach and Education Activities to Support the National ALS Registry.”

Contract Award Number:
200-2013-57122
Contract Award Dollar Amount:
$2,695,642.00

https://www.fbo.gov/index?s=opportunity&mode=form&id=c7856e6375ef7f0fcc2c4cb47ae176a0&tab=core&_cview=0 

Request 2 I would like to know what the work product, deliverables, and performance standards are for the work in the ALS Association contract for “Education and Outreach for the National ALS Registry.”

Dollars Obligated	$319,465
Current Contract Value	$319,465
Identifying Agency ID	7523
Procurement Instrument Number	HHSD200201141609C
Modification Number	6
Transaction Number	0
Fiscal Year	2013
IDV Agency ID
IDV Procurement Instrument ID
IDV Modification Number	0
Solicitation ID	2011N13315

 http://www.usaspending.gov/explore?fiscal_year=all&comingfrom=searchresults&piid=HHSD200201141609C&modification=6&typeofview=complete 

There is a nice online status tool that they provide to track the status of your request.  The status of both requests went from and initial  "Pending Program Search" to "Pending Final Review" within a few weeks.  Great!  I honestly expected to have answers by the end of the year.  Silly me.

By January 23, I felt that surely something had gotten lost somewhere in the "Final Review."  I emailed the CDC FOIA officer who had helped me with the requests.

The online status for both of these has been “pending final review” for over two months. Is there a way to check to make sure that something didn’t get lost in the process? Thanks very much for your help.  

I immediately got a reply that floored me again.

This is in response to the email below concerning your Centers for Disease Control and Prevention and Agency for Toxic Substances and Disease Registry (CDC/ATSDR) Freedom of Information Act (FOIA) requests #14-00039 and 14-00043.
 As we advised in our acknowledgement letters dated October 22, 2013, CDC processes all FOIA requests on a first-in, first-out basis, which is the court-approved method for agencies operating under a backlog. Program staff have completed their search for the records you requested, and your case is currently in this office awaiting final review. A thorough document review will be conducted by the CDC FOIA staff, and all releasable records will be provided. Processing time is contingent upon the number of requests ahead of yours and their complexity and volume. Therefore, we are unable to give you an exact timeframe for completion of your request but estimate that it may take up to 36 months for your request to rise to the top of the complex queue based on current workloads. Please be assured, however, that a response will be sent to you as quickly as possible.
 You may continue to check on the status of your case by going to our FOIA webpage at www2a.cdc.gov/od/foiastatus and entering your request number. The fiscal year is the first two numbers and the request ID is the second set of numbers.

Hmmmmm.  Why is this so hard?  Thirty-six months?  Does someone not want to talk about this?

My response, still on January 23, was --

I appreciate the insights.With matters ALS, the clock ticks quickly.  One of the advocates who was interested in the information has died already.I’ll keep watching the status and hope that it finds the top soon.Thanks.  Have a good afternoon.

The status is still "Pending Final Review."  That must be some fancy review.

This started as a very simple question.  The answer should be pretty simple.  It just gets curiouser and curiouser when people make everything difficult.  This is information that should be very transparent, especially since the contracts required no bidding and since we advocates go to our legislators to ask for the project's funding every year.

It should be simple, but it's not.  Such is the way with matters ALS.

Why?

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.


http://www.neurology.org/content/82/10_Supplement/P5.077.short?sid=85093e2b-8d66-4dd4-9722-dfa4458c5825

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

1. D. Kevin Horton₁,₂, 
2. Vinicius Antao₁,₂ and 
3. Paul Mehta₁,₂

+SHOW AFFILIATIONS

1. Neurology April 8, 2014 vol. 82 no. 10 Supplement P5.077

ABSTRACT

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

http://www.neurology.org/content/82/10_Supplement/P2.072.short?sid=e107f57f-3dcb-4ff4-a777-7245f9f289f7

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

1. Marchelle Sanchez₁,₂, 
2. Paul Mehta₁,₂, 
3. Wendy Kaye₃, 
4. Vinicius Antao₁,₂ and 
5. D. Kevin Horton₁,₂

+SHOW AFFILIATIONS

1. Neurology April 8, 2014 vol. 82 no. 10 Supplement P2.072

ABSTRACT

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/Methods - We 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

Here's A Different Wrinkle on ALS Awareness Month

A classic from an ALS advocate on facebook yesterday --

ALS Awareness: Where every size Allen wrench is required and every screw costs $60.

Let's Raise The Bar Ever So Slightly - Step 2

This one's so simple, too.

Every year we are surprised by events when we get social media updates from the events themselves.  Every year one ALS organization schedules an event on the same dates as another organization's event.  They schedule so far in advance that who knew?

Let's be efficient.  Let's be informative about what events agencies are sponsoring or attending.  Let's raise the energy bar on all that you do.

Technically a common, shared calendar where all ALS organizations contribute content would not be difficult.  The human and organizational factors may be a little more difficult, eh?  If you expect us to believe that you can advance the science against ALS, then you should be able to do this.  It's not hard. To do it the way you always have seems nuts.

Let's Raise The Bar Ever So Slightly - Step 1

Let's raise the bar on how things are done in the fight against ALS.

My first plea is for all organizations and individuals who publish statements and documents in the fight against ALS to please date your documents.

We have web pages and documents and publications online and in print with no clue as to the publication date.

This is simple. Nobody would argue that this is a good practice.  Please take the pledge to raise the bar ever so slightly.

Thank you.

April 20, 2014
Easter Sunday

Now THIS Would Be Patient-Centric

"I feel like I've left tissue all over the country." -- Words of a man with ALS

Today there are countless opportunities for people with ALS and their healthy relatives to give blood and skin and fingernails and all kinds of tissue samples for ALS research.  Unfortunately the donors are treated like renewable sources of tissue rather than valued people.  Researchers who often brag that they work together all gather their own samples for their own needs.  It's not about the patients. It's about their immediate research needs.

Picture this.

A person diagnosed with ALS is given a unique identifier on the day of diagnosis.  Maybe even a nice card with a bar code is included.  After that, every medical record, every blood sample, every MRI, every test result includes that identifier.  A person's information and samples are suddenly connected with the person rather than with a scientist.  And shortly after diagnosis, people with ALS are offered the opportunity to opt-in to a master bio-bank.  The tissue isn't stored in one place, but the information about where the tissue samples reside is stored centrally.  It's called patient-centric.

Think about the way your tire store can find a set of tires that fit your car overnight, even if two tires come from a warehouse in Saint Louis and two from a warehouse in Cleveland.

When a researcher or a pharmaceutical company needs a random set of blood samples from people with C9 genes, they can get them.  When a researcher needs a completely random set of skin sample from people with ALS, they are accessible.  When a research needs some blood from young SOD1 male PALS with slow limb-onset, they can get them.

Fix the information problem  Fix the center of attention.  It's the patient.  Donate once, use many times.

This would be patient-centric.

Next time you hit a chuckhole and need a new tire, think about the days when Lou had to wait for the factory to make more 4.50x21s to fix the Model A.

Your Best Salespeople Understand the Features, Advantages, and Benefits

I'm still trying to learn about the MODDERN Cures Act.  Examples would finally make it come to life for me.

http://www.nationalhealthcouncil.org/pages/page-content.php?pageid=163


OK, for dormant therapies, we've often been frustrated by a lack of study of repurposing old drugs for ALS since they aren't commercially attractive to drug developers.  Some examples of how MODDERN Cures features work would be so helpful!

Is it correct that the dormant therapies must be things that have never made it to market?  Are old drugs that were on pharmacy shelves for other indications part of the MODDERN Cures Act?

1. What if there is a drug that is within a year of losing its patent and the manufacturer holding the patent decides to pursue testing of that drug for ALS?  It will take years to test the drug for ALS.  What happens?  What does it mean to waive the old patent rights?  What does the 15-year period of "data exclusivity" mean?
2. What if there is a drug that is within a year of losing its patent and another manufacturer is interested in pursuing it for ALS?  How would that work?
3. What if there is an old drug whose patent protection has long since passed.  Can it get new patent protection with this?

We will be dynamite salespeople if we understand the features, advantages, and benefits in some real-life situations that relate to people with ALS.

And does the following link mean that the companion diagnostic features of the MODDERN Cures Act have al been taken care of?  http://capwiz.com/alsa/issues/alert/?alertid=63177606

Thanks to whomever can help.

Sometimes A Single Voice IS All It Takes

This is a story of two funerals and those single voices.

1.  Last week a powerful single voice was buried at Arlington National Cemetery.  An Army colonel, retired by ALS, had become a voice that called out weakness in the VA healthcare system for veterans.  He called out ALSA, the CDC, and ATSDR for weakness in the ALS Registry.  He called out the FDA for a system that needed to get ALS patients' perspectives.  He never minced words.  He knew how to lead and how to cut through government-speak.  He caused discomfort in a system that needs to change.  Our nation paid beautiful tribute to him for his military service.  Everyone involved with the fight against ALS should pay tribute to that single voice, too.  He asked good questions and demanded answers... regardless of how uncomfortable he made you.

2.  Last week a wife/full-time caregiver/soulmate to a man with ALS died unexpectedly.  It's not supposed to work that way.  The shock, pain, and practical problems were huge.  An individual woman who helps people who fall through the considerable cracks in ALS organizations' support systems stepped up.  She is one of those single voices who does not take "no" for an answer.  There were funds to raise for a funeral.  There were short-term and long-term arrangements to be made for the care of the gentleman with ALS.  There were difficult logistics in a system that doesn't move quickly enough.  Phone calls weren't being returned and a hospital's policies needed to be challenged.  Nobody should be kept from attending his own wife's funeral by a hospital's risk-management person.  And everybody with ALS deserves to live in a respectful setting that understands ALS.  And families need to grieve.  Yes, a single voice helped make it all happen.

This weekend I hope that all who pray will say prayers for those dealing with ALS and its aftermath... and special prayers of thanks for those single voices.  We need more like them.

And The Answers Are ???

Words from one of the three patients selected to attend the ALS Registry Annual Meeting in July:

Following up from last year, I wasn’t here last year, but one of the big questions I received from patients was, "What were the actions taken from last year’s meeting? Was it productive?" I’m sure many of you knew a great man, Rob Tison, who was here and put a lot of work into trying to give feedback. Just a really good person, and a very smart person. One of the questions I received was, "What was done with that?" How were the questions that were raised last year resolved? I’m sure they all were resolved. I’m sure action was taken, but if not everyone realizes it, it doesn’t do any good.

I find no evidence in the meeting report that the questions were ever answered. I looked and I looked.  It's like the questions just rolled off the backs of everyone.  Click here for the meeting minutes.  If I've missed something, I'm all ears.  We're not so sure that Rob Tison's issues and questions were all resolved. We're not so sure any were resolved.  Time to stop the slow-pitch softball.

It's Simple

"I'm not asking for another $10 million for you until you tell me what you're going to do with it."

-Words of an ALS advocate

http://www.alsa.org/advocacy/legislative-priorities.html 

It's time for a high-level accounting of where the ALS Registry money goes.

It's called good stewardship.

Picture This

In the past week we've learned bits and pieces about various not-for-profits' positions on a number of issues that are important to those with ALS.  There does not seem to be a place where we can read the letters and statements that they have made to government agencies and other not-for-profits about various policies and legislation.

We pretty much have our friend google guiding our way as we stumble along trying to find out about governmental agendas and policy statements that have happened in the past.

Picture how helpful it would be if organizations that take positions or make statements or send letters would simply post them for us to see them as they are made.

It would add perspective and information that would make advocacy efforts much more powerful and cohesive.  Please post those documents in a visible place so we can read them and learn and understand.

It's simple.  Transparency is good.  Timely transparency is best.  Everybody wins.

Times Have Changed, Thank Heavens

Yesterday I saw a new For-Sale sign in front of a home in my neighborhood. 

A couple of decades ago I either would have written down the brokerage phone number and made a call or stopped in at an open house to find the price on the home.  You see, the real estate broker owned "the book," and "the book" had all of the listings and prices.

Real estate brokers controlled the most precious information to potential buyers and sellers.  If you were shopping for a home, you never got to take "the book" home to shop in privacy.  If you just wanted to keep track of asking prices in your neighborhood, the real estate broker controlled that information.  You had to ask.  You could usually get the price, but the rest of the story could be quite selective.

When it was time for serious buyers and sellers to make decisions, brokers had a great deal of power to control the conversation with the information that they chose to share or not share.  And "the book."

Times changed quickly, thank heavens.  Enter the internet.  Consumers demand access to information.  They want to be fully informed as they make decisions.  Real estate brokers have had to change.  They can no longer define the value they add by controlling the information or the conversations.  They have to add value in different ways or find new careers.

And think about the travel-agent industry has changed.  They no longer own sole access to flights and seats and hotel prices.  Travel agents can't make a living today with control of information and access.

And think about the Voluntary-Health-Agency industry.  Whoops.  It hasn't changed yet.  It will.  It must.

And In The "You Can't Make This Stuff Up" Department

Picture this.

• One arm of our government has built a national ALS Registry featuring online information, data entry, and risk factor surveys for people with ALS.  They have spent almost $40 million taxpayer dollars and are asking for more.
• Another arm of our government has just implemented rules for people with ALS who need Speech Generating Devices.  They will not be able to access the internet during a 13-month rental period.  

Duh.  Do these people ever talk to each other

We have just built a very expensive highway and have blocked access to the people who need it most. And those signs that were put up April 1 are going to be dreadfully expensive to take down.

This is just outrageously stupid.