By: Janni Lehrer-Stein

​A 10-year-old girl sees her mother’s face for the first time. An 11-year-old boy races through the aisles of Target, marveling at toys he never knew existed. Both children had mere glimmers of vision, and were destined to lose even that because of an inherited eye disease with no treatment or cure. But thanks to the miracles of modern-day medicine, what was once only possible in science fiction novels is now becoming reality. 

By: Janni Lehrer-Stein
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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

I’m the mother of a wonderful girl named Shannon, who has a rare genetic condition called Phelan-McDermid syndrome. For years I steered clear of research information about her condition and focused on the daily challenges of meeting her needs. It never occurred to me that beyond being Shannon’s mom and caregiver I would play another vital role – as part of the research community helping her and others like her live a better life. 

​NAMI is proud to be part of the movement to assess value of health care treatments and interventions, and to build an infrastructure for patient engagement throughout the healthcare system, particularly in the shift to value-based payment and delivery models. However, we think it’s crucial that any efforts to measure and assess value, incorporate the patient perspective, and consistently and meaningfully engage patients along the way.

​What is the first thing that comes to your mind when you hear the word, “research?” Maybe you think of book reports and term papers, or maybe your mind goes to Hollywood depictions of people in white coats running testing laboratories. Perhaps, you think of something else entirely. 

Whatever your association with the word, research means many different things to many different people. It can sometimes be difficult to encourage people to engage as active participants in healthcare research, particularly if their associations with the concepts are less than positive.

​​College is stressful. Especially being new in college. Your routine is turned upside down, you are focused on finding new friends, involving yourself in different clubs on campus, and passing your classes. There is a new load of stress that comes with your new world. Add managing an invisible chronic illness to that laundry list and it seems next to impossible to fit into the college lifestyle like your peers.

I had 14 years of diabetes management under my belt when I was a freshman political science student at American University. I prepared and thought about my transition to college for weeks. I had registered with my school’s Academic Support and Access Center for accommodations, scoped out the dining hall offerings, and had the nerve-wracking talk with my roommate about the Emergency Glucagon Pen.

As a new president prepares to assume office in January, the continuing evolution of our health care delivery system — from a traditional fee-for-service payment system to a more “value-based” payment arrangement — shows no signs of slowing down.
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Given the emphasis being placed on the “value of care” — who defines “value,” how the “value” of individual patient care is being judged and applied and how this assessment could deny access to treatments or services — is what motivated Lung Cancer Alliance and more than 60 groups representing patients and people served by health systems, to write an open letter to state that “value” should first and foremost be considered through the eyes of patients and people with disabilities — who are the ultimate beneficiaries of health care.   

​My name is Laura Roix, and I’ve been living with idiopathic pulmonary fibrosis (IPF) since 2006. After going through multiple false diagnoses from a number of doctors, I finally found what worked for me: self-advocacy and perseverance. Until I got to that point though, I went through 6 years of difficult medical experiences that finally led me to become a self-advocate for my medical care.

Starting in 2006, I visited a pulmonary doctor after being diagnosed with pneumonia multiple times. The pulmonologist found that I had scar tissue in my lungs, and we monitored it for years. He always said that it wouldn’t spread so not to worry about it – but things quickly changed in 2012. In February of that year, my doctor let me know the scarring had spread; not only in my right lung, where it was originally occurring, but also to the left.
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