Please support us through AmazonSmile

This is the season of giving! The holidays are approaching and we know our supporters are busy shopping for gifts, decorations, and more for family and friends.

Did you know that your purchases can make a difference?

When you purchase on Amazon Smile, specifically smile.amazon.com/ch/93-1036770, they donate to United States Adult Cystic Fibrosis Association (CF Roundtable) when you do your holiday shopping!

AmazonSmile is a website operated by Amazon that lets you enjoy the same wide selection of products, low prices, and convenient shopping features as on Amazon.com. The difference is that when you shop on AmazonSmile (smile.amazon.com), the AmazonSmile Foundation will donate 0.5% of the purchase price to the eligible charitable organization of your choice.

Please support us through this simple and automatic way every time you shop, at no cost to you. United States Adult Cystic Fibrosis Association (CF Roundtable) sincerely thanks you!

Happy Holidays!

Vote Like Your Life Depends On It! It Does.

By Beth Sufian, JD

Vote like your life depends on it Tuesday because if you have cystic fibrosis it does!

I did not think I needed to write an article about how important voting on November 6th is to people with CF, but last week I realized I was wrong.  I had discussions with some people with CF who did not understand the importance of the election to the CF community. I started to explain that if there are no changes in who is serving in Congress, the Affordable Care Act will be repealed in 2019.

We saw how close Congress came to repealing the Affordable Care Act (“ACA”) last year. Senator John McCain was our hero when he voted against the repeal of the ACA. Senator McCain lost his own battle with cancer this year and he will not be able to save us again. To be safe we need 3-4 new Senators and 23-26 new members of the House who will protect the ACA.

Everyone with CF and everyone who loves a person with CF should be spending the next two days getting out the vote for candidates who will protect our ability to access the care and the medication we need to fight CF.

If the Affordable Care Act is repealed it will become impossible for large numbers of people with CF to access the health insurance policies or government programs we need to pay for our care and the expensive medications that are improving the lives of children and adults with CF.  Last week we heard from the Republican majority leader Senator Mitch McConnell who said that in 2019 he pledged to repeal the Affordable Care Act, to both reduce the number of people who are eligible, and to restrict coverage under Medicaid and Medicare.

Many in the CF community mistakenly believe that the ACA does not apply to them because they have a private health insurance policy. The protections of the ACA apply to all private insurance policies. Here are just some protections the ACA provides to people with CF.

  1. The right to keep a parent’s health insurance coverage until a young adult turns 26.
  2. No lifetime caps on coverage.
  3. A maximum out of pocket amount.
  4. No pre-existing condition exclusions.
  5. 45 services that must be covered.
  6. The option for a state to expand Medicaid to low-income adults.

Without the ACA all of these provisions and many more protections go away.

Recent data show that at any given time at least 50 percent of children and adults with CF are enrolled in Medicaid.  Even if you have private health insurance, the loss of Medicaid by people with CF will still affect you. If 50 percent of our population does not have coverage for medication or treatment it will be impossible for companies and health care providers to deliver medications and treatment to the rest of us.  There will not be enough people who will be able to pay for care and medication. All people with CF will be negatively affected if the ACA is repealed.

If you have made other plans on November 6th I urge you to reschedule. There is no other event that should take precedence over the fate of health care for those with CF. We should all be coming together to make sure when we wake up on November 7th we know we did everything we could to protect the rights of people CF.

We ALL need to be getting out the vote on Tuesday. What does that mean? We need to be reaching out to friends and family members and explain why this election is so important to the CF community.

We need to reach out to campaigns who need our help block walking, phone banking and texting to get voters to the polls. We need to make sure everyone we know has a way to get to the polls. Many important races will be won by only a few votes. Every hour you spend getting out the vote now counts, there is no going back after the election is over.

This is the most important election of our lifetime.

Please join me in assuring that we have done everything we can for everyone in the CF community to continue to have the legal right to access insurance coverage. So many worked so hard to develop the excellent CF Care Center system. So many have spent their lives researching and discovering medications to help ease the burden CF places on us. Let us honor the memories of those who have lost their battle with CF by making sure we explain to friends and relatives why their vote on Tuesday is so important.

On Tuesday, I will be available all day to answer questions about the Affordable Care Act, Medicaid or Medicare coverage or to connect people to the information they may need to vote. Please call me at 1-800-622-0385 if I can help. I am acting as a private citizen on Tuesday and not as a member of any organization. I hope my next post will be of me dancing in the street late Tuesday night celebrating all of our efforts to make sure that everyone with CF has a bright future.

Be Involved in a Meeting with FDA on CF!

On October 29th, individuals with cystic fibrosis and their families will have a unique and pivotal opportunity to share their experiences with representatives of the FDA during a live-streamed interactive meeting.

CFRI is very honored to host an Externally-Led Patient-Focused Drug Development Meeting on Cystic Fibrosis with the FDA on Monday, October 29th. This is an amazing and singular opportunity to share the patient experience with FDA representatives. They want and need to know the impacts and burden of the disease, your hopes for new therapies, and what you are willing to go through to find these new drugs.

PLEASE register to participate in this free live-streamed meeting! We need your input and participation You will have the opportunity to participate in live polling, and to email and call in to share your experiences. Those who do not have CF/a family member with CF should also feel free to join us.

You can register and log in for any or all of the day’s presentations and discussions. The day begins at 9:45 am. Please note: all times listed are East Coast time, as the meeting will be held at the College Park Marriott Hotel and Conference Center in Hyattsville, Maryland.

Here is the link:  http://cfri.org/advocacy/advocacy-events/

Speakers/Panelists
Jen Caruso
Lise-Courtney D’Amico
Boomer Esiason
Gunnar Esiason
Joseph Klausing, JD
Emily Kramer-Golinkoff
Robert Lim, MD
Jane Mitchell
Anna Payne
Kat Quinn Porco, MS
Tejashri Purohit-Sheth, MD
Arek Puzia, CPA, MBA
Emily Schaller
Isa Stenzel Byrnes, LCSW, MPH
Ahmet Uluer, DO, MPH
James Valentine, JD, MHS

Thank you for having an impact upon those who are assessing the safety and efficacy of new CF therapies, and making recommendations for their movement to market. 

Your voices matter!

The Cystic Fibrosis Reproductive & Sexual Health Collaborative (CFReSHC) Invitation to Participate

The Cystic Fibrosis Reproductive & Sexual Health Collaborative (CFReSHC) is a patient engagement project that provides women with CF the opportunity to be equal partners with healthcare professionals to shape the future of CF research.  CFReSHC offers women with CF the opportunity to participate on the Governance Board, Research Advisory Panel and Patient Task Force as Patient Partners to develop research projects that directly respond to the questions faced by women with CF.  CFReSHC holds monthly virtual meetings on topics that impact the lives of women with CF like contraception, family building, hormone influences on CF, incontinence and menopause.  As we enter our third year, CFReSHC is looking for women with CF who are passionate about sexual and reproductive health research to join our collaborative who can commit 4-6 hours a month and we provide  a small honorarium for your time commitment.

CFReSCH is looking for a:

  Social Media Strategist who has connections in the CF community or who is willing to engage with the CF community on our behalf.  CFReSHC currently has a social media presence on Facebook, Twitter and Instagram and the applicant would need to be able to post 2-3 times per week as well as check the platforms regularly and make responses as needed.  The applicant would need to attend 3 paid monthly meetings often held during work hours.

Grant writer and Coordinator who has connections in the CF community or is willing to connect with the CF community and engage it to seek out funding opportunities for potential grants.  The applicant would need to maintain a database of potential grant opportunities.  An applicant who has experience writing applications is preferred.  The applicant would need to attend 3 paid monthly meetings often held during work hours.

Women with CF are welcome to send a letter of interest to: cfreprohealth@gmail.com.  Together, we will respond to the health needs of the CF community by providing data for healthcare professionals to pave the way for improved sexual and reproductive health resources, healthcare, and knowledge.

I’m Drowning – A researcher-patient’s plea for broader inclusion in cystic fibrosis trials

By: Ella Balasa

I’ve always known cystic fibrosis (CF) is a progressive disease; it destroys lung cells, tightens the small airways in the bottom of my chest, and each day takes me closer to the time when it will have ravaged my lungs. I had never really questioned if there was some way this process could be altered. I accepted that it couldn’t.

Recently, however, this has changed. The epicenter of new CF research is the development of medications that will slow, stop, and hopefully even reverse the effects and damage that CF inflicts on the body. The possibility of the cells in my lungs functioning to their full potential — with CF transmembrane conductance regulator protein function restored and working correctly, expelling chloride out of my cells, hydrating the surface of my lungs, and halting the thick sticky mucus that has caused my airways to be enveloped in a suffocating cloak for all these years — is like a feeling of being rescued when you are drowning.

Unfortunately, I am still drowning.

“I’m very sorry, Ms. Balasa, but you will not be able to be a participant in this clinical trial.” This was the response I received during one of my searches for these drug trials. Excited by the possibility of participating, finding one recruiting at my local adult clinic, I reached out to study coordinators and was informed that I met all but one criterion to participate in the studies. This specific criterion has prevented me from prior trial participation involving other investigational medications treating the symptoms of CF, including anti-infectives and anti-inflammatories.

Most CF studies, including phase I, II, and III trials, require a lung function minimum of at least 40% FEV1 (forced expiratory volume in one second). My FEV1 is 25%, so I am excluded from these trials. Many patients face a similar situation. The 40% threshold biases samples toward a young patient population, as this degenerative condition causes steadily decreasing lung function with time. Furthermore, as CF treatment has rapidly progressed and increased patients’ life expectancies, there are now more adults with CF in the U.S. than children, according to the CF Foundation Patient Registry.

As a patient who works in the science field, I started to ask myself: Where does that number come from? Should this one variable be such a deciding factor? Are we getting comprehensive results from these studies if a subset of patients is omitted? Are investigators using eligibility criteria from a prior study without determining whether the exclusions are scientifically justifiable?

To continue reading, please visit MedPage Today.

CF Foundation asking for Public Comment on Lung Transplant Referral Guidelines

Dear CF Community,

The CF Foundation seeks your input on the draft of the Lung Transplant Referral for Individuals with Cystic Fibrosis: Cystic Fibrosis Foundation Consensus Guidelines. This guideline is one in a series of guidelines for advanced stage lung disease; the other guidelines in progress on this topic include: Advanced Lung Disease Consensus Guidelines and Post Lung Transplant Consensus Guidelines.

A committee of CF health care providers, transplant healthcare providers, and an adult with CF researched and developed the consensus recommendation statements presented in the draft. It is our hope that you will add your input and feedback to ensure that our community issues actionable practice recommendations for lung transplant referral for individuals with cystic fibrosis.

To systematically collect feedback from the CF community, we have created an on-line tool which is linked below. We recommend that you review the attached document, Lung Transplant Referral for Individuals with Cystic Fibrosis: Cystic Fibrosis Foundation Consensus Guidelines PDF, and use the link below to enter your comments.

Submit comments here: https://www.surveymonkey.com/r/LTxReferralPublicComment

The deadline for comments is Wednesday, October 10th, 2018, 5:00 PM, Eastern.

We value your input. Thank you in advance for taking time to review and provide us with feedback on this very important aspect of CF care.

If you have any questions, please contact shempstead@cff.org.

Sincerely,
Albert Faro

Albert Faro, M.D.
Senior Director of Clinical Affairs
Cystic Fibrosis Foundation | National Office

Join in BreatheCon 2018!!

Calling all adults with CF!

Join me at #BreatheCon 2018! Step back from your day-to-day routine and connect with other adults with #cysticfibrosis to discuss topics that are unique to #lifewithCF. Created by and for people with CF, BreatheCon is a two-day virtual event with keynote panels, group chats, fun activities, and small-group video breakouts.
https://cff.swoogo.com/BreatheCon2018

CF Foundation | BreatheCon 2018

Registration is open for BreatheCon! Step away from your day-to-day routine and connect with other adults living with cystic fibrosis to discuss topics that are unique to life with CF. Created by and for people with CF, BreatheCon is a two-day virtual event with keynote panels, group chats, fun activities, and small-group video breakouts.Register today: https://on.cff.org/BreatheCon2018

Posted by Cystic Fibrosis Foundation on Tuesday, August 28, 2018

Patient-reported outcomes: Time for a new approach?

By Janice Abbott

Patient-reported outcome (PRO) measurement (e.g. health-related quality of life questionnaires, symptom diaries) can provide a standardized, valid and reliable way of gaining the patients’ perspective on ‘how they are’ or the benefits and limitations of a specific intervention. The insights that patients have concerning their health are important given that aspects of patient-reported quality of life are independent predictors of survival in cystic fibrosis (CF) [1]. Regulatory authorities require the inclusion of PROs in clinical trials as an additional outcome parameter and PRO information is becoming important in labelling claims. It is noteworthy that the top 10 research questions, reached by global consensus of patient and healthcare providers, all require the inclusion of CF-specific PROs to achieve meaningful answers [2]. This represents a significant paradigm shift but capturing data that matters to patients, families and clinicians is challenging. Two of the persistent challenges in CF PRO measurement are a) the development and use of technologies to enable efficient administration, accurate scoring, and the correct interpretation of data and b) being able to accurately measure PROs (or parental proxy assessment) across the entire CF lifespan. These important issues are considered by two papers in this issue of the Journal of Cystic Fibrosis [34].

PRO measurement largely remains a research endeavour with little uptake in clinical practice. Administering, scoring and interpreting PROs in a busy clinic is difficult. It requires staff time and expertise and the results are not instantly accessible to steer a discussion with the patient or to aid clinical decision making. Paper-based data collection suffers from missing, unreadable data that is prone to scoring/mathematical error. The development of electronic PRO (ePRO) technologies is immensely important in clinical practice and for endpoint assessment in clinical trials. It is a cost-saving, patient-friendly approach to PRO assessment: data collection can occur in clinic, the patient’s home, workplace or school. Results can be added to a patient’s electronic medical file, alerts triggered by problematic scores and clinicians can track patient/parent-reported symptom/event data over time. Importantly, electronic data capture enhances the integrity and accuracy of the data, makes it logistically easier to collect repeated assessments (daily or at several points over a trial), and is preferred over paper-based data collection by the US Food and Drug Administration (FDA).

There is growing evidence that paper and electronic versions of PROs typically provide comparable data but this requires psychometric evaluation if transferring an original paper-based questionnaire to an electronic mode of administration. Solé and colleagues have demonstrated measurement equivalence with paper and electronic administrations of the Cystic Fibrosis Questionnaire-Revised (CFQ-R teen/adult version) [3]. The e-CFQ-R web version is linked to an online database that can be adapted for any electronic devise (smartphone, tablet, computer). Immediately the patient completes the questionnaire, results are sent to the healthcare team and the data are saved in a centralized, protected database. Real-time patient-reported data are available to the clinician as an adjunct to clinical data. Access to the English and Spanish versions are by independent web addresses provided in the paper. Ultimately, the integration of PRO data within electronic care records as developed by Peckham et al. [5], or in CF patient registries would enable efficient patient care and longitudinal research endeavours.

There is a lack of PROs that can be used as endpoints in early intervention studies in CF. Such instruments are time-consuming and painstakingly difficult to develop so the research of Edwards et al. reporting on the initial development of a CF-specific, parent-reported instrument for children 0–11 years is welcome [4]. The need for an effective way of data collection is also considered. The instrument takes the form of an electronic (web-based data capture), observational sign/symptom diary containing 17 respiratory and activity signs that parents report the presence or absence of. Results suggest that children aged 7 to 11 years are best reporting for themselves, therefore observational reporting by parents should focus on young children aged 0 to 6 years. Considerable evaluation has yet to determine the final instrument but the development of the scale follows FDA guidance enabling its acceptance as a clinical trial endpoint in infants and young children with CF.

Over the last twenty years we have learned a great deal about measuring patient-reported outcomes in CF, and there are many pitfalls when employing PROs in CF trials [6]. They are typically secondary endpoints and the trial is not powered on them, often making it difficult to draw valid inferences about treatments. However, there are trials that have collected patient-reported respiratory symptom data as the primary endpoint [78], employing the only CFQ-R subscale that has been approved by the FDA for use as an endpoint. Scientific, regulatory and pragmatic factors are driving the shift towards ePRO data collection. The development of ePROs is not trivial, yet they are fast becoming the ‘gold standard’ for PRO data capture in clinical trials. The challenge now is to develop CF-specific, lifespan PROs, utilising new technologies that can deliver real-time, high-quality PRO information. They also need to be acceptable to the regulatory bodies to aid their decisions on cost-effectiveness and ensure the appropriate commissioning of new medicines to improve the lives of people with CF and their families.

Original article with references here.

Cystic Fibrosis Podcast 192 Emily’s Entourage

In the latest Cystic Fibrosis Podcast, Jerry speaks with Emily Kramer-Golinkoff about the role of a patient advocate organization in driving drug development in rare disease.
Emily, a 33-year-old who has a nonsense mutation of CF, is a co-founder of Emily’s Entourage, a 501 3(c) that’s goal is to accelerate research for new treatments and a cure for CF. She is an internationally recognized patient advocate and speaker, has a Master’s degree in Bioethics and is certified in Clinical Ethics Mediation, was named “Champion of Change” by President Obama’s Precision Medicine Initiative, and has been featured by CNN.com, Time.com, AOL.com, People.com, and more for her work with her charity.

Continue reading Cystic Fibrosis Podcast 192 Emily’s Entourage

Cystic Fibrosis Podcast 191 ‘Why do you run?’ with Ben James

In his latest podcast, Jerry Cahill sat down with Ben James, a 19-year-old college student who was diagnosed with cystic fibrosis at 4 years old. Originally from Chester, VA, Ben now attends Mount Vernon Nazarene University in Ohio where he studies pre-medicine. When he isn’t focusing on school subjects like anatomy and physiology, Ben likes to play basketball and soccer, and also likes to run long distance.
Ben loves the challenge of running long distance for a number of reasons, including building character, facing challenges as an individual, and learning discipline, which helps with his overall medical compliance.
Click here to listen:
This podcast was made possible through an unrestricted educational grant from Genentech to the Boomer Esiason Foundation.