Advocate

The United Mitochondrial Disease Foundation is committed to increasing funding for mitochondrial disease research now – to alleviate the suffering of thousands who have this debilitating and often fatal disease and to develop better understanding of and treatments for the many common illnesses and chronic conditions associated with it.

To expedite this action and ensure Congress, the medical community and the general public understand the importance of mitochondrial disease research, we must call on our the affected community, their friends and families, to advocate for all.

Liz Kennerley’s Blog – UMDF Advocate

Liz’s Latest Blog

Advocacy takes many forms; it can be done on and off the Hill.  I like to look at the big picture, while others may be more interested in advocating on the individual level.  The way I see it, awareness brings attention to a problem, while advocacy is more assertive with the goal of trying to solve the problem made clear through awareness.  They go hand in hand.  I can’t advocate for something without making others aware a problem is present. For example, telling a doctor, you have insomnia is awareness.  Turns out; it takes an exhaustive amount of advocacy to be treated.  My doctors sometimes comment about how they have little chance of changing my stubborn thoughts since I talk with US Senators and Congressmen on a regular basis.  I’m also the only child of parents who are the oldest of six.  Enough said.

Let me first give you an example of Awareness.  Last year, I helped organize, with support from UMDF, the very first disease-specific event held at BIO International Convention which is the world’s largest gathering of the biotechnology industry, along with industry leading-leading investor and partnering meetings.  I previously interned at BIO, and have connections going back over 20 years.  Given this was the first event of its kind, we had to build the program from scratch.  We had three panels.

The first panel was titled “Understanding Mitochondria and Mitochondrial Disease: A Look at Challenges and Opportunities in Research.”  There were some familiar names in the immediate Mitochondrial Disease community, UMDF’s own Phil Yeske Ph.D., as well as researchers Marni Falk MD, Mark Tarnopolsky MD Ph.D. FRCPC, and Michio Hirano MD.  neurodegenerative conditions affecting several million people.

What would you say to a room full of researchers from every angle about mitochondrial disease?  I painted the picture of daily life using my favorite analogy and a famous line from Forrest Gump, “Life is like a box of chocolates.  You never know what you’re going to get.  My final quoted comment was used in the BIO Convention’s Daily Bio Buzz Newsletter.  “This isn’t just about me.  This is about everyone I know with Mito.  All of you give me hope, and that’s the most important thing in the world when dealing with a disease you know is fatal.”  The patient perspective is crucial in many contexts from fostering the patient-provider relationship to drug development.  This cannot be understated.  Difficult to cure something without understanding.

The other panels were, “Drug Development for Mitochondrial Disease: Examining the Current Landscape and Scientific/Regulatory Gaps” and “Harnessing the Promise of Mitochondrial Research: The Potential to Unlock Answers in Other Diseases and Aging.” Both panels had speakers that overlapped with others in the program.  Dr. Marni Falk took a deeper dive into the multitude of reasons why drug development in our space is the antithesis of straight forward.  Much of it was a review after participating in joint FDA and NIH meetings that combined to form the Mitochondrial Disease drug development CPIM (Critical Path Innovation Meeting).  Sometimes this information can be hard to hear, not only technically speaking, but personal since there is nothing I can do to simplify the complexity of this disease.

After my first panel finished, my former co-worker from BIO leaned in and said to me and Phil Yeske, “There’s so much interest that we need to bring in more chairs!”  I didn’t leave the space the entire time, but when I turned my head at one point, it was standing room only!  I couldn’t help but get teary eyed.  I learned after the convention ended that there was an entire group of people banging on the door to get into the Mitochondrial Disease Forum, but were told, “They’re in the middle of a session!  You can wait!”  It’s a really powerful feeling realizing something you created may have planted the seed in Merck or Pfizer’s head causing them to think about something that may not only treat this beast of a disease but cure it as well.  It’s a feeling that isn’t easy to describe.

My positive successes on Capitol Hill have a different tone.  Mike Fitzpatrick, my former Congressman, did not support 21st Century Cures Act.  He fought for more funding FDA for over a year, going as far as standing in front of the House Rules Committee in the wee hours before full House vote, mentioned me by name and all I’ve done for rare diseases.  He had quite a few other reasons.  Among them being concerns over expedited medical device approval, a prominent issue among other constituents.  All the while acknowledging this Act would accelerate drug development.

One comment was, “I broke with tradition and voted against the bill – not for what it included, but for what it lacked.”  It took me awhile to wrap my head around this.  As bummed as I was, there is a lot to be said that he knew 21st Century Cures Act would pass, yet voted nay out of protest.  As well as demonstrate his support of the rare disease community and our desperately needed funding for cures.  Mike was asked several times by different people, including our own Chuck Mohan, to join our Caucus, but only did so after I asked, citing as I remember, “I’m joining the Mitochondrial Disease Caucus because Elizabeth Kennerley asked me to!”  He also served on the Rare Disease Caucus.  His brother, Representative Brian Fitzpatrick joined that Caucus after Mike’s retirement from politics.

On April 5th, I participated in my third Legislative Fly-in with BIO.  Among several meetings I had that day, three stand out the best.  I met with Senators Casey (Pennsylvania) and Warren (Massachusetts), as well as Rep. Lloyd Smucker from Pennsylvania’s 16th District and got him to co-sponsor the OPEN ACT within five minutes!  Among other positive things, this piece of legislation will allow the repurposing of drugs by pharmaceutical companies for off-label use when proven effective for other indications.  For example, Bezafibrate, is currently in trial overseas as a treatment possibility. This would be tremendous since the drug has already gone through the lengthy approval process and deemed safe for its primary indication to lower cholesterol.  Opposed to, creating a product from scratch, costing more compared to something that’s been on the market for longer.  (Like Nexium vs. Zantac, both treat reflux, but one is more expensive because it’s a newer drug.)

This bill was originally part of the 21st Century Cures Act, but unfortunately, Senator Elizabeth Warren pulled it at the last minute, and advocates must start from scratch.  It was reintroduced in the House during Rare Disease Week, so hopefully, momentum can get it going again…  In the meeting during the BIO Legislative Fly-in surrounded by pharmaceutical “high level” people, as the only patient in the room reliant on both Obamacare and Medicaid, I shared my stories (along with some others) as well as my dependency on both Obamacare and Medicaid.

I do this during every meeting but was especially crucial with Senator Warren after she recognized me since our last encounter two years ago.  She is also very influential given her outspoken nature.  In addition to talking about the above, I very carefully and tactfully expressed my disappointment and frustration regarding not only her opposition to both OPEN ACT and 21st Century Cures, but the importance regarding the timely passage of health policy decisions.  As I spoke, she repositioned her chair and blinked maybe eight times.  I hope I made an impact, but truly won’t know until this unique piece of legislation is reintroduced in the Senate.  One of my group members who is in the pharmaceutical industry commented, “We’re so glad you’re here.  You’re better than 100 of us.”  Among a few other points, our collective concern and “ask” was the reauthorization of PDUFA.  The same I expressed during Rare Disease Lobby Day and Digestive Disease National Consortium’s Lobby Day.

Drumroll to about three weeks ago….and Senate HELP Committee (Health Education Labor Pensions), which both Senators Casey and Warren serve on, took the very first steps towards reauthorizing PDUFA!!

Every five years since, BIO and PhRMA (Pharmaceutical Research and Manufactures of America) negotiate with FDA how high these fees will be, as well as how many new reviewers need to be hired, and any procedure changes the FDA will undertake to accelerate drug approval process.  Many of these changes will be the direct result of 21st Century Cures Act becoming law on December 13th, 2016, just ten days after my 30th Birthday.  If PDUFA isn’t reauthorized 60 days before this legislation expires on September 30th. 2017, FDA will be forced to send lay-off notices to more than 5,000 employees.

Each and every experience from the Hill to a podium raising awareness is advocacy because of the context.  Many meetings and speaking events are successful in the long run.  Yes, both my former Congressman Mike Fitzpatrick and “honorary Senator” Elizabeth Warren, didn’t support 21st Century Cures, but they did so because they wanted MORE FUNDING and made that clear.  Yes, she pulled OPEN ACT out, but this helps focus my efforts on getting it reintroduced and getting her aboard.

In the end, 21st Century Cures Act passed in both chambers with unprecedented margins, 392-26 in the House, and 94-5 in the Senate.  I was up the gallery watching both with my own eyes.  It’s something I won’t soon forget.  We also have our champions in government.  Senator Casey has been nothing but supportive of the mitochondrial disease community and drug development.  I have a selfie with him!!  He is a true light in this sometimes-dark tunnel.  We are all more than ready for more drug approvals given the mitochondrial disease statistics we know as a community, and the larger rare disease space with 1 in 10 Americans living with a rare disease, yet just 5% have FDA approved treatment.  The FDA could always use more funding.  Even if it is “just a fig leaf” as Senator Elizabeth Warren said as her primary reason for also being unsupportive of the 21st Century Cures Act.

Living in Washington, DC, just four blocks from the Capitol and truly on the “front lines” of every decision health policy related in this political climate isn’t easy.  But I have a choice.  I can either frown and mope along or take an active role by making sure our voices are considered.  Not many things happen quickly in the rare disease space.  On average, in takes seven years for an accurate diagnosis  #path2cures

ACTION ITEM FOR RESIDENTS OF PENNSYLVANIA, CALIFORNIA, LOUISANNA

Please visit the UMDF Advocacy Action Center here and send a letter requesting your Senator support our important efforts to fund research.  UMDF has asked Senator’s Bob Casey (D-PA), Diane Feinstein (D-CA) and Bill Cassidy (R-LA) to insert important appropriations language into the budgets for the Department of Defense and President Trump’s budget for 2018.  See our appropriations requests here.  We need to keep this critical research in the NIH and DoD Budgets! Make your voice heard now!

LATEST INFORMATON ON HEALTH CARE LEGISLATION

Educate yourself on the House proposal regarding Health Care Coverage.  The Senate will be taking up “Repeal and Replace” this summer!
As you know, the House has approved its version of replacing the Affordable Care Act.   This could impact you or a family member.  Make sure you understand the proposed legislation so that you can, along with the UMDF, advocate your needs in a measure that will be considered by the United States Senate.  Click here to read the brief provided by UMDF and our partner Holland+Knight.

Concerned about “Replace and Repeal” of the Affordable Care Act?

We want you to know that the current process of “repealing and replacing” the Affordable Care Act (ACA), also known as Obamacare, is of great concern to UMDF.  Click here to see our position on “Replace and Repeal”.  Many who suffer with mitochondrial disease have historically experienced difficulties obtaining affordable health care coverage.  The requirements of the ACA regarding robust coverage of those with pre-existing conditions under family, individual and small group plans has been particularly important for many patients and families.  As you know, this bill is now working its way through Congress and we are watching carefully how any legislation will impact our families.   We invite you to check the UMDF Advocacy Action Center frequently for important alerts and ways for you to participate and make your voice heard on this important issue.

UMDF OPPOSED H.R. 1313 –Preserving Employee Wellness Programs Act

H.R.  1313 is before the House Ways and Means and Energy and Commerce committees before it advances to the entire House floor. The House Committee on Education and the Workforce approved the bill out of their committee. The Committee, in its approval, noted that the legislation would allow employers to offer employee wellness plans, help them promote a healthy workforce and would lower health care costs.  However, employees who refuse to test could be subject to higher insurance premiums. Currently, employers are prohibited and restricted from asking to collect genetic information from employees under the Genetic Information Nondiscrimination Act (“GINA”), the Americans with Disabilities Act (“ADA”) and other state laws.  H.R.1313 specifically states that GINA and other protections will not apply to genetic testing conducted under a workplace wellness program or a program relating to health promotion or disease prevention.  That is why UMDF opposes it. Under GINA, group health plans (such as employer health plans) are not allowed to request or require genetic testing. Wellness plans are only allowed to disclose employee genetic information to employers in aggregate form so that an individual’s identity remains anonymous. However, employers are allowed to offer genetic testing to employees and receive the test results from employees on a voluntary basis.  Click here to see our letter of opposition and check back to see what action we need you to take should this move to the House floor for consideration.

What can you do now?

VISIT THE UMDF ADVOCACY ACTION CENTER

Want to learn more about how to advocate?

Advocacy Help
To assist you in your advocacy, we are pleased to present a toolkit which provides guidelines for working with elected officials and for engaging the media.  Included are fact sheets, talking points, sample letters and sample media materials to give you everything you need to begin your advocacy campaign.

You are our best advocate.  It is critical that we tell the UMDF story and your individual stories to a much broader audience.  Our messages must be consistent and clear in order to make mitochondrial disease more readily recognizable and to create a sense of urgency among policy makers and the medical community.

Materials have been developed for you to download and to help you convey important, easy to understand information about the disease and the need for additional research.

Thank you for your support!

Advocacy Toolkit

WANT TO LEARN MORE ABOUT HOW TO ADVOCATE? To assist you in your advocacy, we are pleased to present a toolkit which provides guidelines for working with elected officials and for engaging the media. Included are fact sheets, talking points, sample letters and sample media materials to give you everything you need to begin your advocacy campaign. These materials have been developed for you to download and to help you convey important, easy to understand information about the disease and the need for additional research.

Thank you for your support!

  • Advocacy Toolkit– (PDF)
    This booklet contains all of the documents below.
  • About UMDF– (PDF)
    This document describes the UMDF mission and goals and can be downloaded for elected officials and media who need more information about the national organization.
  • Mitochondrial Disease Fact Sheet– (PDF)
    This document describes mitochondrial disease, incidents and prevalence and links to other diseases.  This is a document that can be sent to or left with elected officials, or given to the media.
  • Links to other diseases– (PDF)
    Defects in mitochondrial function are at the core of many common diseases and conditions.  This document provides the links these diseases.  This is a document that can be sent to or left with elected officials, or given to the media.
  • Frequently Asked Questions– (PDF)
    This document provides the answers to many of the questions asked about mitochondrial disease.  This is a document that can be sent to or left with elected officials, or given to the media.
  • Talking Points– (PDF)
    This document provides the message that we all must use when speaking to elected officials and to the media.  Core messages are bolded.  Below each core message are additional points that you may add.
  • Working with Elected Officials– (PDF)
    Information about contacting and communicating with elected officials.
  • Sample Letter to Elected Officials– (Word Document)
    You can view a sample letter to an elected official, or download the information into a Word Document, so that you may copy and paste to create and personalize your own letter.  Please include personal stories.
  • Working with the Media– (PDF)
    Information on how to contact the media to promote stories and information happening in your state or community.
  • Writing a Letter to the Editor– (PDF)
    Sample Letter to the Editor– (Word Document)
    Tips on writing a letter to the editor of your local paper.  You can also copy and paste a sample letter.  Make the letter personalized with your information as well.
  • Writing an Op/Ed Piece – (PDF)
    Sample Op/Ed Piece– (Word Document)
    Tools to detail the creation of an Op/Ed piece for your local newspaper.  You can also copy a sample op/ed piece to use after you’ve added your personalized information as well.
  • Sample Media Advisory– (PDF)
    A sample of a media advisory to help you create media awareness is available.  ALL RELEASES AND ADVISORIES MUST BE SUBMITTED TO THE UMDF NATIONAL COMMUNICATIONS OFFICE BEFORE THEY ARE SENT TO THE MEDIA.
  • Sample Press Release– (PDF)
    For your use in structuring a media release.  ALL RELEASES AND ADVISORIES MUST BE SUBMITTED TO THE UMDF NATIONAL COMMUNICATIONS OFFICE BEFORE THEY ARE SENT TO THE MEDIA.
  • Additional Resources– (PDF)
    Links to other web sites that may be helpful.