SBSF and NPS Pharma Partner on New Project
CenterWatch Weekly · December 9, 2013 · By Karyn Korieth
The Short Bowel Syndrome Foundation (SBSF), a nonprofit that worked with industry leaders to support NPS Pharmaceuticals in gaining FDA approval for Gattex, undertook a new project aimed at strengthening patient-to-patient connection and supporting faster, more patient-informed development of treatments for this rare disease.
SBSF Chief Executive Andrew Jablonski, who founded the organization three years earlier, was building a platform to allow SBS patients and caregivers to connect with one another on a one-to-one basis—beyond group or social media settings. NPS, which approached Jablonski about the idea, could eventually use the platform to recruit patients for clinical trials or to learn more about the needs of patients and their advocates. In the short term, the project—called either SBS Connections or SBS Match—would be an online patient-to-patient support network.
Mary Frances Harmon, then head of global advocacy for New Jersey-based NPS, noted that while social media helped patients feel connected, many wanted more personal contact—by phone or face-to-face—and often preferred connecting with people in similar circumstances, a common need in rare disease communities.
The project strengthened a partnership that began before FDA approval of NPS’s drug Gattex, the first prescription medicine for the long-term treatment of adults who depend on intravenous support for adequate fluids and nutrition. NPS had become a corporate partner supporting educational tools and programs.
“Our mission aligns closely to some of the goals of our industry partners. We are both striving for improved care and better lives for patients. We are working with partners and cultivating them as best we can, because we are in favor of innovation that brings out new, improved treatments that can improve the quality of life for SBS patients.”
— Andrew Jablonski, Founder & CEO, SBSF
Jablonski served as an expert advisor for NPS, sharing first-hand information about living with short bowel syndrome—including risks of dehydration and malnutrition—contributing to market research, advising on educational website content, and relaying what other patients hoped medications could improve and what side-effect concerns they had.
“I have been the person who is able to connect patients to the pharmaceutical industry to make their voices heard, and vice versa,” Jablonski said. “In rare disease, patients are scarce and difficult to find. We serve as a conduit between the SBS community and to some of the stakeholders the industry wants to reach.”
Harmon said NPS could expand the relationship based on patient needs—particularly pediatric patients, which Jablonski noted made up a large share of the SBS population. NPS was preparing a global pediatric registration study for Gattex, and SBSF was seen as a valuable partner in making that program successful through ongoing two-way dialogue.
The article placed the partnership in a wider movement: patient groups becoming stakeholders in drug development—helping with study design, meaningful endpoints, trial awareness, and education after approval. Natural history insights from patient communities were highlighted as especially important in rare diseases, where epidemiological data are limited. SBS was described as affecting about 20,000 patients in the U.S. at the time of the article.
SBSF—then with hundreds of members and thousands of Facebook followers—also planned to publish a book of first-hand stories from patients living with SBS to help the public and industry better understand community challenges. “We are willing to provide perspectives from our personal lives,” Jablonski said. “We want to be heard and are willing to tell our stories.”
“As an organization committed to pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, NPS is acutely aware of the importance of our relationships with the patient community, and that we must constantly be attuned to the voice of patients and their needs. They are our most important constituents, so we aim to cultivate a constant stream of two-way dialogue with patients and their advocates.”
— Mary Frances Harmon, Global Advocacy, NPS (as reported in 2013)
This page preserves a December 9, 2013 CenterWatch Weekly article by Karyn Korieth. Organization names, product status, membership figures, and project names reflect that time period. For current SBSF programs and education, use the links below.
This article is provided for historical and educational context. It is not medical advice. Therapy decisions should be made with a qualified healthcare professional. Product availability and labeling may have changed since 2013.