CAL RARE

Navigating life with a is incredibly challenging and taking time to prioritize mental health often doesn’t get the attention it deserves.

Your mental and emotional well-being matters! As comes to a close we remain committed to providing information and resources throughout the rare disease journey. Check out tools and support opportunities from Global Genes, here: https://bit.ly/3KnjD1H

Read this important report from our friends at the California Health Report.
📍Parents who are concerned about their child’s ventilator should talk to their child’s doctor and care team.
📍California legislators could help by increasing Medi-Cal reimbursement rates for ventilators.

Ventilator Recalls Leave Few Options for Kids with Breathing Conditions. Here’s What Would Help The recalls have shaken parents whose children depend on ventilators due to health conditions that affect their breathing. Doctors and children’s advocates are asking federal officials to better tr…

Happy Mother’s Day to Rare Mamas! ❤️

The Stanford Medicine Alliance for Disability Inclusion and Equity (SMADIE) is hosting an inaugural Disability & Telehealth Conference on Saturday May 18th from 8am-4pm. This is a free, virtual conference.

The conference will feature patient voices and many providers who are treating patients with different types of disabilities via telehealth.

Register here:

Disability & Telehealth Conference Join us for the Stanford Disability and Telehealth Conference on Saturday, May 18, 2024, as we explore the challenges and solutions aimed at enhancing the quality of telehealth care for individuals with disabilities.

April 29th is Undiagnosed Day. It is a day to celebrate those living with an undiagnosed rare disease and their families as well as to remember the ones we have lost. We also want to thank everyone dedicated to working rigorously within the undiagnosed rare disease community to find a patient’s diagnosis.

Today is Undiagnosed Day! What is Undiagnosed Day? April 29th is Undiagnosed Day. It is a day to celebrate

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The Office of Health Care Affordability (OHCA) is hosting the Health Care Affordability Advisory Committee Meeting on Tuesday, March 19 at 10 am.

The Meeting Agenda and January Meeting Minutes have been posted to the HCAI public meetings page.

Please follow link to view the materials.

Additionally, please note that public comments regarding OHCA's proposed spending target must be sent to [email protected] by March 11.

March Health Care Affordability Advisory Committee Meeting - HCAI The Health Care Affordability Advisory Committee provides input on a range of topics, including: a statewide health care spending target and specific targets by health care sector and geographic region; the methodology for setting spending targets and adjustment factors to modify targets when approp...

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This International Women’s Day we salute the incredible women who continue to inspire us, lead us, and make us who we are every day.

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Cal Rare thanks our Rare Disease California Caucus Chair for his unwavering support of rare patients! 🧡🦓🧡

Today, on Rare Disease Day, let's raise awareness and support for those living with uncommon medical conditions. Every person battling a rare disease deserves understanding, empathy, and access to resources. Let's unite to spread awareness and show our solidarity.

🚨 IT'S OFFICIALLY RARE DISEASE DAY ACROSS THE GLOBE 🚨

Join us in marking Rare Disease Day 2024 now that it's finally here! Let's unite in solidarity with the millions worldwide living with rare diseases. 🌍 Spread awareness, share stories, and show support for them.

Together, we can make a lasting impact and shine a light on these often overlooked!

Let's make today count by raising awareness and advocating for those who need it most. Every voice matters in the fight for understanding and support.

Visit our website to find out more:https://www.rarediseaseday.org/

DC News: Taking place today is the first ever White House Rare Disease Forum. This event is aimed at highlighting the current policy and research opportunities to meet the urgent and unmet need of the more than 30 million Americans living with 10,000 rare diseases — a disproportionate number of whom are children.

You can watch the livestream at: https://lnkd.in/ecMZdEeK

California rare advocates take the Hill!

News from DC: House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Subcommittee on Health Chair Brett Guthrie (R-KY) will hold a legislative hearing titled “Legislative Proposals to Support Patients with Rare Diseases” on Rare Disease Day February 29, 2024. Livestream and additional info at link.

Chairs Rodgers and Guthrie Announce Health Subcommittee Legislative Hearing on Rare Disease Bills The Committee on Energy and Commerce is the oldest standing legislative committee in the U.S. House of Representatives and is vested with the broadest jurisdiction of any congressional authorizing committee.

Cal Rare Founder/President Angela Ramirez Holmes joins 800 advocates from across the country in DC for Rare Disease Week. Cal Rare is tabling at today’s Legislative Conference. 🦓🧡

A 2022 study found that the economic impact of rare disease in the US in 2019 was nearly $1 trillion, only 44% of which were attributed to direct costs. (The National Economic Burden of Rare Disease Study) Patient communities are shouldering the impact of the cost of living with rare disease. However, traditional research methods have proven to be ineffective due to the limited number of patients.

The Innovation and Value Initiative and EveryLife Foundation for Rare Diseases have partnered to identify common patient-centered outcomes and achieve a consensus on addressing unique research challenges. We hope these findings develop effective research approaches for patient engagement and recommendations for patient-centered outcomes, leading to informed outcome selection that is important to patients, caregivers, and families.

The and Foundation project aims to advance the field of patient-centered outcomes research for rare diseases. Cal Rare was honored to be a part of this effort. We thank for their partnership and support of this project.

Read the full paper:https://thevalueinitiative.org/wp-content/uploads/2024/02/2024-Rare-Disease-Project-Report-Final.pdf

Join the FDA’s Virtual Public Meeting for Rare Disease Day 2024. Registration is free.

More info including agenda and registration: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/public-meeting-fda-rare-disease-day-2024-03012024

Share to thank your people. ❤️

It is the holidays so we are a day late! 🧡

We are grateful for our RARE community!

On December 14th, 2023, the FDA in collaboration with the Duke-Robert J. Margolis, MD Center for Health Policy (Duke-Margolis) and supported by a cooperative agreement between FDA and Duke-Margolis, will host a virtual public meeting titled “Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement.” The purpose of this public meeting is to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence to support rare disease drug approvals.

Rare Disease Patient Community Engagement Meeting On December 14th, 2023, FDA and Duke Margolis will host a public meeting for advancing the development of therapeutics through rare disease patient engagement.

Join The Innovation and Value Initiative (IVI) and EveryLife Foundation for Rare Diseases for a webinar on patient-centered outcomes and HTA in rare disease therapy. Explore patient-centric outcomes, hear critical recommendations, discover real stories from patients and caregivers, and learn how to implement these recommendations into HTAs from the payer’s viewpoint.

This webinar is designed for patients, caregivers, payers, employers, policymakers, pharmaceutical professionals, and researchers.

Register now to be part of this critical discussion and help shape the future of rare disease therapy evaluation! https://ow.ly/LWe050Q3zNk

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I’m often pretty casual when I talk about them.

My daughter’s medical issues, that is.

When listening to me, some people may think I’m being flippant or minimizing serious issues like “surgery” and “hospitalizations” and “heart valve replacements”. And you know what? When I listen to myself sometimes I guess I can hear that, too. But it’s actually not the case at all.

As a parent, when you are thrown into a world of life and death and medical chaos, it changes you.

It doesn’t harden you. No. Not at all.

Maybe it doesn’t even strengthen you. I know I don’t feel strong much of the time.

But what it does do is change your perspective on life in every way.

It makes things that once seemed big now seem little, and makes the little things now seem big.

It makes you put on your grown-up pants even when you don’t want to - just because you know you have to.

It makes things like hospital stays and never ending doctor follow-ups and foreshadowed surgeries seem normal. They are not, but for parents of kids with medical issues they are OUR normal. So we need to treat them that way - talk about them that way - so that we can feel normal.

And, also, so that we don’t completely crumble into pieces every single day.

This is our life. Our not-so-normal normal.

And this is how we cope.

*Follow Mia Carella for more.

California Budget & Policy Center presents a Free Virtual Event “Improving Health Care Access for Californians” on November 9 from 10:00-11:00AM. Click link to register!

Join us as we explore the recent downward trend in Medi-Cal coverage and learn what state leaders can do to ensure that all Californians can access and maintain the health coverage they need in order to be healthy and thrive.

You'll hear from a panel featuring a researcher, an advocate, a community health worker, and a state official, each offering their unique perspective on efforts to expand health care coverage and build a robust health workforce that reflects communities throughout California.

Improving Health Care Access for Californians We'll examine California’s health care landscape and opportunities to increase coverage and build a diverse health care workforce.