The NORD program uses a collaborative approach to educating state legislators
One of the issues raised by the Affordable Care Act is the importance of decisions made at the state level and the need to build awareness of the needs of the rare community among state officials.
“Many important healthcare decisions are being made at the state level. While NORD will maintain our focus on federal policies, we’re also becoming increasingly involved in educating state legislators about the challenges of living with a rare disease,” says Tai Spargo, of the National Organization for Rare Disorders (NORD).
Advocating at the State Level
To increase awareness, NORD has created a toolkit designed to be used by coalitions that bring pharma, patient organizations, and physician organizations into the discussion. The NORD program uses a collaborative approach to educating state legislators.
“NORD is starting to look at the need and views this as an opportunity to focus on state trends,” Spargo says. “One of our member organizations, the Shwachman-Diamond Foundation, and MassBio have organized a very successful Statehouse event for Rare Disease Day the past several years. We enlisted their help to create a template that others could replicate in other states.”
For Rare Disease Day 2013, there were 5 Statehouse events—in California, Connecticut, Massachusetts, New Jersey, and Texas. In Texas, there was a rally on the deck of the statehouse before they went in and presented the Rare Disease Day proclamation. Connecticut had a patient who was a member of the legislature and talked about his disease. Medical centers and organizations are often involved as well.
What Role Does Pharma Play?
Pharma is often involved through the state bio organization. “We believe collaboration among all stakeholders is an important part of these events. This includes patient organizations, companies developing orphan products, academic researchers, and others,” Spargo notes. “This year we are focused on general awareness topics—daily living challenges for patients, diagnosis, finding treatments, and access. In some states, there may be particular issues. But in general, the purpose is simply awareness and greater understanding.”
The NJ Rare Model
NJ Rare is a good example of a successful coalition. Julie Raskin, Executive Director of Congenital Hyperinsulinism International, was Co-Chair of the event at the New Jersey statehouse last year. “We put together a program that included various kinds of speakers, including patients, researchers, biotech companies, and advocates. Susan Anderson spoke movingly of her son who suffered from an ultra-rare disease. He passed away before he was even diagnosed. Francois Nader, CEO of NPS Pharmaceuticals, spoke about developing a treatment for a rare condition. Diana Autin, Co-Executive Director of SPAN NJ, spoke about advocacy and support opportunities for families living with rare diseases in New Jersey. In December we worked with HINJ, the Health Institute of New Jersey, and they put together a forum on the Affordable Care Act (ACA) and rare disease patients. In addition to Biotech, clinicians, and patients, there were representatives from the insurance industry and the Department of Health and Human Services. It was a great, well-attended event. I wanted to make the point that the ACA is a huge step forward because of the elimination of lifetime caps and pre-existing conditions, but there are many additional issues.”
Some of the things that rare disease patients must still grapple with are affordability of care and the need to travel to access a center of excellence to achieve the best quality of life. For some people with rare diseases, the ACA provides a new bridge to affordable healthcare. For others, affordable care is still out of reach. Even under the ACA, premiums, deductibles, and out of pocket expenses can still be extremely expensive for rare disease patients and their families. The first Rare Disease Day event and the Forum that followed were so successful that the group has decided to continue to meet and plan events on an ongoing basis.
The Future of NJ Rare
“I have goals as we move forward,” Raskin says. “We want to support rare disease patients in New Jersey by connecting them to resources they need, providing information about governmental services, creating linkages between government and non-profits, and providing information about state and non-profit service organizations.” She also hopes to have more biotechs and pharmaceutical companies working with NJ Rare.
Plans for this year’s Rare Disease Day event will include paired patient stories—one from a fortunate family and one from a family that is less fortunate. “We have a patient that had an easy diagnosis and we want to show the difference that made in the outcome, compared with someone who didn’t have a diagnosis right away. The young woman in her 20s who wasn’t diagnosed close to birth has to live in an institution and has the intellectual development of a 9-month-old. We will compare that to 2 girls who were identified through genetic testing and born at Children’s Hospital of Philadelphia in a birthing center dedicated to high-risk babies. They have a good prognosis and no brain damage.”
Raskin knows from personal experience what a difference early diagnosis can make. Congenital hyperinsulinism is a genetic disorder that is more common in some ethnic groups. “Now it’s part of a genetic testing panel for Ashkenazi Jews. We are Ashkenazi,” she says, “but when my son was born 17 years ago, it was not part of the testing at the time.”
The overall mission of the group is better lives for rare disease patients and families, according to Raskin, and they are creating a powerful partnership to make it happen. “Alone we are rare, together we are strong” is more than a motto for the rare disease community in New Jersey. They’re living it.