follow

Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

Issues Today

From our friends at EveryLife Foundation (December 20, 2017)

Capitol Hill Updates

The House and Senate have passed a conferenced version of the Tax Cuts and Jobs Act, which makes cuts to the Orphan Drug Tax Credit (ODTC), a key provision of the Orphan Drug Act which incentivizes rare disease treatment research by lowering development costs for manufacturers. This version of the bill would reduce the tax credit’s value from 50% to 25%. The bill also makes changes to the medical expense deduction, which is currently utilized by approximately eight million Americans who have medical expenses that exceed 10% of their adjusted gross income. This deduction can now be taken for medical expenses exceeding 7.5% of income. Click here to read about the final tax bill.

The final tax reform legislation repeals the Affordable Care Act’s individual mandate, which requires individuals to enroll in an insurance plan or face a financial penalty. Earlier this year, the Congressional Budget Office (CBO) estimated that repealing the mandate would raise the number of uninsured individuals to 13 million by 2027. Click here to read about what repealing the mandate could mean for insurers.

The Children’s Health Insurance Program (CHIP) expired on September 30 but is still awaiting Senate reauthorization. It provides affordable, comprehensive health insurance for children in families with too much income to qualify for Medicaid, but struggle to afford private insurance. A Continuing Resolution passed earlier this month extended current spending levels for two weeks in order to prevent government shutdown. States like Connecticut are already planning to close their healthcare program for low and middle-income children by January 31 if Congress fails to extend federal funding. Click here for more information.

Earlier this month, the Food and Drug Administration (FDA) released draft guidance entitled, “Pediatric Rare Diseases —A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry.” The guidance covers the use of age-specific endpoints, extrapolation from adult studies to demonstrate treatment efficacy, and strategies for designing multi-arm clinical trials. Click here to read more.

In response to public feedback on Patient Participation in Medical Product Discussions of the Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA created the Patient Engagement Collaborative (PEC). The PEC will focus on how to incorporate meaningful patient engagement in medical product development discussions. The Agency is seeking participants from the patient community, caregivers and individuals who represent patient groups. Click here for more information

 

Community Action Alerts & Policy Resources

First Focus is encouraging patient advocates to sign-on to a letter asking Congress to extend CHIP funding. They note that the program is critical for the 8.9 million children that depend on it. Click here to read the letter and sign-on.

Research!America is asking advocates to write to their Members of Congress urging them to oppose legislation that would provide funding for the Department of Defense while holding non-defense discretionary spending static. This would impact agencies like the National Institutes of Health (NIH) and FDA. Click here to take action.

The Epilepsy Foundation is asking patient advocates to write to their legislators to encourage them to oppose the Americans with Disabilities Act (ADA) Education and Reform Act of 2017 (HR 620). According to the Foundation, the bill would weaken protections for people living with disabilities by making it easier for businesses to avoid complying with the accessibility requirements of the ADA. Click here to take action.

The EveryLife Foundation for Rare Diseases is asking individuals to contact their Members of Congress in support of the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatments; HR 1223/ S1509) which has the potential to double the number of treatments approved by the Food and Drug Administration (FDA) for rare diseases. For more information and to take action, click here.

 

Community Events

On January 25, the NIH will hold a symposium entitled, “Global Perspectives on Standards and Common Data Elements Used in Patients Data and Biospecimens Collection.” Click here for more information.

On February 25 to March 1, RDLA will host Rare Disease Week on Capitol Hill. Events include a rare disease documentary screening and cocktail reception, Legislative Conference, Lobby Day, Rare Disease Congressional Caucus briefing, Rare Artist reception, and Rare Disease Day at the NIH. Click here for more information, including the schedule of events.  Registration will open on January 3, 2018.

On March 1, as part of Rare Disease Week on Capitol Hill, the NIH will host Rare Disease Day at NIH. This event aims to raise awareness about rare diseases, the people they affect and NIH research collaborations to advance new treatments. Click here for more information.

On March 8, the FDA will convene a public workshop on “Safety Assessment for Investigational New Drug Safety Reporting.” Click here for more information

On March 19 to 21, World Congress will host the 6th annual Patient Advocacy Summit. Industry leaders, patient advocates and policy makers join to tackle the complexities of patient advocacy and the healthcare market. Click here for more information.

Login