There is no question that the incentives contained in the Orphan Drug Act (ODA) have stimulated development of products to treat rare disorders. Emerging treatments for rare and ultra-rare disorders, particularly those without a potential non-orphan use, are a unique subset of orphan products. They require a focused, unified advocacy strategy to ensure continued viability. In today’s environment, rare disease treatments that “deserve” incentives must be differentiated from those for which orphan designation is a savvy step in a broader commercialization strategy.


The ODA did not include the reimbursement and market access incentives so critical in today’s environment of aggressive utilization management, which is steadily creeping into the orphan and ultra orphan universe.


The creation of a coalition dedicated to promote the unique standing of rare and ultra-orphan products will provide the focused, amplified voice necessary to ensure that lawmakers consider “payer side” incentives that were never envisioned in 1983, and that should be added in light of the changes our healthcare system has seen since the early 80’s. Started in mid-2016, the coalition that is today known as the Haystack Project, has been bringing together patient organizations representing extremely rare diseases to learn from each other and systematically speak out about ICER, the QPP, Medicaid and Medicare (including inpatient, outpatient, pharmacy) and 340b.


Connect 4 Strategies, LLC is uniquely positioned to engage key stakeholders in the rare and ultra-rare community to establish a unified voice on the Hill, in regulatory policy decisions, and at the grassroots level. Led by Saira Sultan, JD., Connect 4’s team has a strong track record of effectively bringing to bear patient, provider, and researcher perspectives to the cross-roads of Capitol Hill and FDA and CMS regulations.

Saira has over nearly 25 years of experience representing industry, providers, and patients.  She brings significant expertise building cross-functional teams that lead to successful outcomes for her clients. To learn more about our staffing, please go to