As we continue to gather each year for our conferences, we most definitely can say that we are an HPS family, that makes up quite a heartfelt, and inspired community! Our community has also been part of other community organizations that are working together to strive for a cure for HPS, and all Rare Diseases. We feel very thankful to be able to work with these brother/sister organizations. They assist us in finding out the latest information, and updates on what all of us within the Rare Disease Community need to work on, to implement the next steps to take to strive for the best future we can aspire to!
Every so often, we are all called to Action to advocate, and/or be part of signing letters to highlight specific focus on policies that will help the needs of all of us with Rare Diseases.
This week, our Call to Action for our communities, to our executive directors, has been to focus on an Accelerated Approval Pathway that is an initiative to bolster the FDA in helping to fast-track development of therapies for Rare Diseases and other associated conditions.
A letter that was sent to us, written by NORD (National Organization for Rare Disorders) states:
“The accelerated approval pathway exists to help patients where there is urgent and unmet need. This applies to most of the estimated 25-30 million Americans living with rare diseases. Fewer than 10% of the approximate 7,000 rare diseases have an FDA-approved treatment. Last summer, NORD released a white paper with several recommendations for how to strengthen and improve the accelerated approval pathway, not weaken it. As the Department of Health and Human Services (HHS) considers various proposals limiting patient access to safe and effective accelerated approval drugs, we urge Congress to act now to help rare disease patients.”
The call to Action that was proposed was for the Executive Directors of HPS, and Rare Disease Community organizations to sign two types of letters that would support the strengthening of the Accelerated Approval Pathway:
“The first letter is directed toward Congressional leadership asking them to incorporate several policy recommendations that NORD believes would strengthen accelerated approval into legislation to reauthorize the Prescription Drug User Fee Act (PDUFA).”
“The second letter is directed towards HHS Secretary Becerra, urging the Secretary to support efforts underway within Congress and the FDA to reform and strengthen the accelerated approval pathway and reject policies that could limit patient access to rare disease therapies that utilize accelerated approval.”
In learning so much more about various policies that we have been advocating for through the years (even though we can often feel perplexed and overwhelmed about our political system) there is still so much within our political system that does relate to our mission for HPS and Rare Diseases. Though it can be hard to find a sense of hope at times, whether through our journeys, or everything that happens around us, may our sights never lose faith on change that can happen, that can be positive! Stay Tuned for more information, updates, and ways to stay involved through our whole website, and our Advocacy page!
For more information on this topic, and to check out another organization that are working on policy and advocacy for Rare Diseases, check out: https://rarediseases.org/
Any other Advocacy related questions please contact Ashley Appell at: firstname.lastname@example.org