A few members of the community have brought a social media conversation to our attention that inaccurately describes NORD’s involvement in state-based Rare Disease Advisory Councils. Because the issues of state advocacy and collaboration are so important, we would like to set the facts straight.
Rare Diseases United Foundation (RDUF) did approach NORD to support legislation to be introduced in several states establishing these councils, similar to the legislation referenced in Connecticut. We reviewed the legislation with the support of our members, at which time it was determined that without certain revisions we could not in good faith promote the legislation.
After receiving phone calls and emails from our members on this issue, we contacted RDUF in February expressing interest to support this effort and to communicate our concerns over a few of the common provisions that appeared in all of the state bills. The changes we proposed would prioritize increased patient and organizational membership on the councils, and coordination with other state and federal efforts. Those changes were declined by RDUF.
NORD was then approached by a representative at our Connecticut State House Event for support and input on H.R. 6580, which the representative had drafted and modeled after the Massachusetts and Rhode Island state bills without our consultation. We, along with several advocates, reviewed the bill and met with the representative to propose changes, which included:
1. Remove NORD from the Council and to instead include up to 3 representatives from patient-based organizations operating within the state
2. Improve representation on the Council:
a) Increase physician participation to 10 representatives, and include the following specialties: gastroenterologist, psychologist, pediatrics, metabolic specialists, and genetics
b) Increase both rare disease survivor and caregiver participation to 2 representatives, one of which would include a pediatric caregiver
c) Include a representative from the State Newborn Screening Program
d) Include a representative from the State Department of Education
The proposed changes did not include a request for government funding, but rather stated that the Advisory Council should be allowed “to apply for and accept any grant of money from the federal government, private foundations, or other sources which may be available for its operation and State programs related to rare diseases.”
We do not support naming any specific organization(s) in these bills because we believe that is inappropriate for any legislation intended to benefit the entire rare disease community. The Council should be as inclusive as possible. As well, representation is best suited for those living in and managing the day-to-day care of rare disease patients in the given state.
We are happy to share our proposed changes with any interested advocates. Additionally, since the bill is still in committee, the representative’s office has indicated that the public still has the opportunity to submit other changes that would improve the legislation. (Please Note: the current version of the bill available online does not reflect changes proposed by NORD and other rare disease advocates. The changes are currently being reviewed by the Public Health Committee and Connecticut Department of Health.)
Our team is made up of dedicated and compassionate patients, parents and advocates. We are in the fight against rare diseases alongside you fighting for all 30 million Americans – including the ones we love and have lost.
NORD is pleased to support the Patients’ Access to Treatments Act (PATA). This important bipartisan legislation introduced in the House of Representatives this week by Reps. David B. McKinley (R-WV) and Lois Capps (D-CA), will limit cost-sharing requirements for medications placed in a specialty tier and make innovative and necessary medications more accessible by reducing excessive out-of-pocket expenses.
Peter L. Saltonstall, President and CEO of NORD, said: “NORD supports legislation that would limit the cost-sharing of drugs placed on specialty tiers. Specialty tiers frequently adversely affect patients with rare diseases, as orphan products are often much more expensive than drugs that are more widely used.
“We have seen many cases where specialty tiers prices have limited patient access to medically necessary drugs and biologics. Untreated diseases not only affect patients directly and sometimes tragically, they also can be an expensive burden to the health care system. We look forward to working in support of this important legislative effort.” read more >
The final 2015 State House Event took place yesterday. Now that all of the numbers are in, take a look at (and share!) this year’s highlight reel & recap:Together, we planned and hosted 32 State House Events in capitol buildings across the U.S., up from last year’s 12 State House Events
With all of our efforts, Rare Disease Day continues to grow! Thank you to everyone who planned and attended events, joined the social media campaign, changed their profile photos, shared infographics, and got involved. As the official U.S. sponsor of Rare Disease Day, NORD thanks everyone who came together to support this truly community and group effort. Together, we are strong!
Keep the Momentum Going read more >
NORD’s Assistant Director of Public Policy, Paul Melmeyer, authored this great piece (below) in the March 2015 issue of the NCC Collaborator, the monthly publication from the National Coordinating Center for the Genetic Service Collaboratives (NCC). NCC focuses on bringing quality genetic and newborn screening services to local communities, and building bridges between public health, primary care/ Medical Home, geneticists and other specialists, and families and consumers. You can read the piece and March issue online here.
Since the passage of the Affordable Care Act in 2010 and the subsequent Supreme Court decision allowing states to decide whether to expand their Medicaid programs, decisions about healthcare coverage and quality standards for patients with rare diseases are increasingly being made at the state level. From public health initiatives, such as newborn screening programs, to insurance plan structure and coverage regulations, state governments play an integral role in facilitating the access of quality, affordable care for individuals with rare diseases.
For 31 years, the National Organization for Rare Disorders (NORD) has served as America’s leading patient advocacy organization for patients with rare diseases. Following the passage of the Orphan Drug Act in 1983, NORD has been involved in numerous Federal policy initiatives, including the Rare Diseases Act of 2002, the Affordable Care Act, and the Food and Drug Administration Safety and Innovation Act (FDASIA), among others.
Beyond our policy efforts, NORD represents over 220 organizations for individuals with rare diseases and provides education and coordination services for patients and their families. read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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