Partnership to Improve Patient Care

  • Home
  • About
    • Mission and Priorities
    • Meet the Chairman
    • Steering Committee
    • PIPC Member List
    • Contact
  • The Issues
    • Action Center
    • Value Our Health
    • International
    • Where We Stand
    • Value Assessment Frameworks
    • Engaging Patients in Value-Based Payment
    • Patient-Centeredness in Research
  • Resources
    • Advocacy
    • Letters and Comments
    • PCORI Meeting Transcripts
    • Polling
    • Roundtables
    • White Papers
  • News
    • Press Releases
    • PIPC in the News
    • PIPC Weekly Update
    • PIPC Patients' Blog
    • Chairman's Corner
    • The Data Mine
  • Events
    • Nevada AB 259
    • QALY Panel
    • QALY Briefing
    • Past Webinars >
      • MFN/IPI Webinar 2025
      • Discrimination & Health Care
      • C & GT Webinar
      • ICER COVID Webinar
      • Value Our Health Briefing
      • ICER SCD Webinar
      • VOH Sickle Cell Webinar
      • Rare Disease Webinar
      • QALY Webinar
      • PCORI Advocacy Webinar
      • APM Webinar
      • Patient Empowerment Webinar
      • Value Assessments Briefing
    • Past PIPC Forums >
      • 2023
      • 2022
      • 2021
      • 2020
      • 2019
      • 2018
      • 2017
      • 2016
      • 2015
      • 2014
      • 2013
      • 2012
      • 2011
      • 2010
  • Home
  • About
    • Mission and Priorities
    • Meet the Chairman
    • Steering Committee
    • PIPC Member List
    • Contact
  • The Issues
    • Action Center
    • Value Our Health
    • International
    • Where We Stand
    • Value Assessment Frameworks
    • Engaging Patients in Value-Based Payment
    • Patient-Centeredness in Research
  • Resources
    • Advocacy
    • Letters and Comments
    • PCORI Meeting Transcripts
    • Polling
    • Roundtables
    • White Papers
  • News
    • Press Releases
    • PIPC in the News
    • PIPC Weekly Update
    • PIPC Patients' Blog
    • Chairman's Corner
    • The Data Mine
  • Events
    • Nevada AB 259
    • QALY Panel
    • QALY Briefing
    • Past Webinars >
      • MFN/IPI Webinar 2025
      • Discrimination & Health Care
      • C & GT Webinar
      • ICER COVID Webinar
      • Value Our Health Briefing
      • ICER SCD Webinar
      • VOH Sickle Cell Webinar
      • Rare Disease Webinar
      • QALY Webinar
      • PCORI Advocacy Webinar
      • APM Webinar
      • Patient Empowerment Webinar
      • Value Assessments Briefing
    • Past PIPC Forums >
      • 2023
      • 2022
      • 2021
      • 2020
      • 2019
      • 2018
      • 2017
      • 2016
      • 2015
      • 2014
      • 2013
      • 2012
      • 2011
      • 2010

The PIPC Blog

PIPC Patient Blog: Policymakers Can't Turn a Blind Eye to Patients With Disabilities

9/5/2017

 
Picture
By: Janni Lehrer-Stein
​​
I learned recently, many weeks after its early June announcement, that the Institute for Clinical Economic Review (ICER) based in Boston will develop a report assessing the cost effectiveness of a new cure for blindness, a gene therapy for vision loss associated with a form of retinal disease. By the time I learned about it, the comment period had closed to allow stakeholders to share key information relevant to the first phase of development of the evidence report. While ICER said it would contact key patient groups and clinical experts to gain further insights on the patient perspective and clinical context of this new treatment option, I had never heard about it when I could have had a voice in the initial phase of the study.  It’s not clear how hard they worked to get the patient perspective, and I certainly have a perspective to share

When I was 26 years old, I felt like one of the luckiest women in my generation. It was 1982, I had just graduated from law school, passed the bar exam, married six months before, and was working my dream job at a law firm in Washington, D.C. I was at the top of my game, and I felt invincible!  This was the culmination of all my years of study, commitment to my loving husband (now of 36 years), and engagement in the practice of law.  I reveled in the long hours of being a first-year associate, fascinated by the legal problems I was working on, fulfilling my dream since childhood of representing people who needed to find solutions to difficult problems.

Then, one morning in January of 1982, I woke up to my first blizzard in Washington. A daughter of the true North, born and raised in Canada, I had no idea that Washington would shut down at almost the first snowflake. And to compound the situation, I realized I had a raging (don’t you only get these things when you are a kid?) case of pink-eye. 

Fortunately, there was a doctor in my building who fitted me in, as traffic slowed to a crawl and businesses and the federal government began to shut down for the duration of the storm. The doctor examined me, gave me a prescription for the pink-eye and then said the words that changed my life forever. He told me that I had a retinal degenerative disease, separate from the itchy annoying pink-eye, and that I would likely go blind, perhaps as soon as the next six months. But then he told me not to worry, because most people like me get hit by a bus before we go completely blind.  Well, almost 40 years have passed, and so far, I have been able to stay out of the way of that oncoming bus!

But being a member of the community of Americans with disabilities did change my life, presenting both its challenges and also great enrichment. Over time, I have lost my sight, and now navigate only with the assistance of my white cane, communicate with the use of assistive technology. I cannot read, drive, watch TV, engage in the modern social intercourse of Instagram and the other computer innovations that rely on visual perception. I find myself often wondering about such basics as whether my shoes match, how to turn on my oven to make dinner, and my social universe continues to shrink as I struggle to keep up to date with all the latest news and trends. And more than anything, I desperately miss being able to see the faces of my three beautiful children.

But the enrichment is there, too. My life as a lawyer transitioned to more than three decades of work as a disability rights advocate, I have raised three incredible children, and served my country as part of the Obama administration. Truthfully, while I wish I could see, my life has been so blessed by the incredible people I have met within the disability community, and those outside the community who are dedicated to ensuring inclusion and the respect for dignity for every American. I have a great life.

But I still wish I could see.

So I was amazed to learn that one of the organizations I serve as a national Board member, the Foundation Fighting Blindness, is making great headway in its search for treatments and cures for retinal degenerative disease. In an almost unbelievable development, the Foundation has sponsored research that, with non-surgical treatment by means of gene therapy, may restore and retain sight for patients who are affected by a form of retinal degeneration called LCA. This particular condition affects children very early in life, so that the gene therapy, successfully administered, may have the effect of enabling blind babies and children to see! Instead of facing their entire lives with the challenges of blindness, this treatment may enable a life filled with vision.

The treatment, if approved by the FDA, as expected within the next six months, will be the first form of gene therapy for an eye condition.

So how does that make me feel? There is only one response-and that is to say, how incredible is this? Making it possible in our lifetimes, for babies born blind to see?

Bravo to the Foundation Fighting Blindness, its dedicated scientists, physicians and staff, its hundreds of thousands of members and supporters! Congratulations to these children, already born or in future generations, yet to be part of this world. I count on them to continue the work of advocacy, science, enrichment and inclusion for all and feel so grateful to witness this amazing futuristic discovery!
​
To groups like ICER, I urge that their work does not serve as a barrier to access to this treatment. In my three decades as a disability advocate, I have often witnessed and experienced the discrimination that may result from assumptions made as to access, capability or benefits of application. I have often fought against the stigma that is attached to disability, or those who are quick to judge that a disabled person’s life is not worthy of attention, inclusion or respect. I find it impossible to believe that the value of this treatment, to restore and retain vision for children who are just beginning their life’s journey, can be discounted or minimized. I encourage ICER, and the broader community of health care insurers, providers and physicians to do everything in their power to ensure that this treatment takes hold, is made available and at reasonable cost that will do so much to benefit young lives. I am hopeful that the Foundation Fighting Blindness, together with patient advocates, physicians, and interested families, will now work together to ensure that the tremendous potential of this treatment comes to fruition.
 
Janni Lehrer-Stein is a disability rights advocate, appointed by President Obama to serve two terms on the National council on Disability, and currently serves as a member of the Board of Directors of the Foundation Fighting Blindness, Disability Rights Advocates and as a member of the National Academies on Science, Engineering and Medicine on its Forum on Aging and Disability.

Comments are closed.

    Topics

    All
    Alternative Payment Models
    Chairman's Corner
    Patient Centered Research
    PIPC In The News
    PIPC Patient Blog
    PIPC Weekly Update
    Press Releases
    The Data Mine
    Value Frameworks

    Archives

    June 2025
    May 2025
    April 2025
    March 2025
    February 2025
    January 2025
    December 2024
    November 2024
    October 2024
    September 2024
    August 2024
    July 2024
    June 2024
    May 2024
    April 2024
    March 2024
    February 2024
    January 2024
    December 2023
    November 2023
    October 2023
    September 2023
    August 2023
    July 2023
    June 2023
    May 2023
    April 2023
    March 2023
    February 2023
    January 2023
    December 2022
    November 2022
    October 2022
    September 2022
    August 2022
    July 2022
    June 2022
    May 2022
    April 2022
    March 2022
    February 2022
    January 2022
    December 2021
    November 2021
    October 2021
    September 2021
    August 2021
    July 2021
    June 2021
    May 2021
    April 2021
    March 2021
    February 2021
    January 2021
    December 2020
    November 2020
    October 2020
    September 2020
    August 2020
    July 2020
    June 2020
    May 2020
    April 2020
    March 2020
    February 2020
    January 2020
    December 2019
    November 2019
    October 2019
    September 2019
    August 2019
    July 2019
    June 2019
    May 2019
    April 2019
    March 2019
    February 2019
    January 2019
    December 2018
    November 2018
    October 2018
    September 2018
    August 2018
    July 2018
    June 2018
    May 2018
    April 2018
    March 2018
    February 2018
    January 2018
    December 2017
    November 2017
    October 2017
    September 2017
    August 2017
    July 2017
    June 2017
    May 2017
    April 2017
    March 2017
    February 2017
    January 2017
    December 2016
    November 2016
    October 2016
    September 2016
    August 2016
    July 2016
    June 2016
    May 2016
    April 2016
    March 2016
    February 2016
    January 2016
    December 2015
    November 2015
    October 2015
    September 2015
    August 2015
    July 2015
    June 2015
    May 2015
    April 2015
    March 2015
    February 2015
    January 2015
    December 2014
    November 2014
    October 2014
    September 2014
    August 2014
    July 2014
    June 2014
    May 2014
    April 2014
    March 2014
    February 2014
    January 2014
    December 2013
    November 2013
    October 2013
    September 2013
    August 2013
    July 2013
    June 2013
    April 2013
    March 2013
    December 2012
    November 2012
    February 2012
    September 2011
    August 2011
    July 2011
    May 2011
    March 2011
    November 2010
    October 2010
    September 2010
    August 2010
    April 2010
    March 2010
    February 2010
    December 2009
    September 2009
    July 2009
    June 2009
    May 2009
    February 2009
    January 2009
    December 2008

Powered by Create your own unique website with customizable templates.