Dawn Rotellini: Importance of Patient Advocacy at Annual Meetings

While focusing on rare diseases sets the 2019 National Organization for Rare Diseases (NORD) and Orphan Products Breakthrough Summit apart from most other annual meetings, those in attendance also exemplify the way the summit breaks the mold of other conferences.

While most conferences and annual meetings are attended by clinicians and researchers, the proportion of patient advocacy groups present at the NORD Summit is much higher than those in other specialties. One of the patient advocacy groups present at this year’s summit was the National Hemophilia Foundation(NHF).

Founded in 1948, the NHF has been supporting the cause of advancing and creating greater access to treatments and cures for bleeding disorders. Through their efforts in public policy reform and funding research through grant programs, the foundation has made strides in their mission of advocating for patients with varying types of hemophilia.

For more on the impact of patient advocacy groups and the unique opportunity presented to them when offered the opportunity for representation at major conferences, MD Magazine® sat down with Dawn Rotellini, chief operating officer of the NHF. As the daughter of a man with hemophilia and the mother of another, Rotellini has had a first-hand look at issues like access to care and the impact of patient advocacy over years.

MD Mag: What is the importance of including patient advocacy groups in annual meetings like the NORD Summit?

Rotellini: There's so many tenants to that question. So, one of the things is I think for care providers to understand the patient perspective—and it's not captured in a clinician setting. It's about—and I hate to use the word quality of life but it is about what is the impact of that patient's life and their disease and, for example in hemophilia, it's not just your average bleed rate in a year.

It is what are your pain thresholds, how healthy are your joints, how does that impact your day-to-day life? When a physician, a nurse comes to a conference like this they get a chance to hear the patient perspective and really interact directly with the patient organizations as well. So, the other thing is I think it's extremely important because the patient advocacy groups that attend these meetings can also hear the physician perspective.

In the case of NORD they really emphasize sessions on access to care and treatment for these rare diseases. For example, the last 2 days the sessions have been filled with panels from the NIH, which are involved in the clinical trials process, and then the FDA. We've just had excellent sessions this morning from the FDA to hear the perspective of the regulators of these drugs that are coming out to the the rare populations.

The other benefit—there's so many—but the other benefit is that in the rare disease or disorder space, some of these populations are so small that it's very difficult for 50 patients across the United States to get the attention of the federal regulators or the clinical trials or the docs because obviously if you have such a small patient group it's hard to find those experts. So, when we all come together, we're all aligned in access to treatment and care and so we have a larger voice together and I think it's a bigger in advocacy impact.