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BIO 2019: Advocacy Requires Data

Collecting and analyzing data is critical, says one leading U.S. advocate for rare disease patients.

NORD President and CEO Peter Saltonstall with CSL Behring's Dennis Jackman at BIO 2019 in Philadelphia
Peter Saltonstall, President and CEO of the National Organization for Rare Disorders (left), speaks during a fireside chat with CSL Behring Senior Vice President Dennis Jackman during the BIO 2019 International Convention in Philadelphia. (Photo Credit: BIO International Convention)

Compelling data could be a key that helps open the door to access to medical treatment for people who have rare diseases, the leader of the National Organization for Rare Disorders (NORD) said this week at BIO 2019 in Philadelphia.

“We’ve made a major investment in empirical data to support what we do from an advocacy perspective,”  Peter Saltonstall, President and CEO of NORD, told CSL Behring’s Dennis Jackman at a fireside chat during the global biotech conference.

NORD advocates for patients by engaging with lawmakers, policymakers and key players, such as insurance companies. When talking to a member of Congress, it always has an emotional impact if you bring along a person or family affected by a rare disease, Saltonstall said. But it’s even more compelling if you can add data to the discussion and tell a lawmaker: “This is how many people in your district are living with rare diseases,” he said. NORD also has sponsored independent studies, carried out by IQVIA, to look at rare disease therapies and establish facts that can be shared with lawmakers.

Companies, like biotechnology leader CSL Behring, which are developing treatments for patients, also recognize their responsibility to provide data to stakeholders so they can make educated decisions, said Jackman, CSL Behring’s Senior Vice President of Global Healthcare Policy and External Affairs. An estimated 350 million people have rare diseases, including 30 million in the United States, about half of whom are children, he said. Patients face hurdles at almost every point. First, they must get an accurate diagnosis, which can take years. Then they often must fight for access to treatment.

And for over 90 percent of most rare diseases, no treatment is available, Jackman said. Researchers and industry continue to push ahead by launching new medicines and new “indications,” meaning that an existing treatment has been approved for use in treating a new disease.

In 2018, the FDA’s Center for Biologics Evaluation and Research (CBER) approved 34 new treatments for rare diseases, Jackman said. As of August 2018, there were 503 approved orphan therapies with 731 designated and approved orphan indications, which shows important progress since the passage of the Orphan Drug Act (ODA), he said. Jackman mentioned the importance of a positive public policy environment, such as the ODA to continue the progress.

They went on to address patient access, which Mr. Saltonstall indicated is getting increasingly difficult for rare disease patients.

“It is tragic if patients can’t access those therapies,” Jackman said.

For talks with insurance companies, which authorize and pay for medical care in the United States, Saltonstall wants to use data to show that treating a rare disease is a good investment. Someone with an untreated or undertreated disease still has medical problems, needs care and might end up in the emergency room, he said. It’s more difficult to quantify, but the patient’s lost quality of life cannot be overlooked when determining whether to provide treatment, he said. Saltonstall added that insurance companies are interested in mining their own data to better understand the healthcare journey of patients living with rare diseases. 

The topic of data was also on the minds of audience members who asked about patient registries, natural histories and standardized tools for collecting information from patients. NORD supports patient registries, which allow people to record information about their health which can be compared and studied. Saltonstall said his organization is also searching for tools that will standardize and combine patient data so it can be best utilized.

“The more information you have the better decisions you make,” he said.