Improving access to healthcare is a significant challenge generally and is particularly challenging in the area of rare diseases, where treatments are often complex and greater understanding of the conditions and their treatments is needed. Generally, the best access to treatment would be through treatments approved by the FDA and through clinical trials for experimental treatments. However, RRD understands that there are situations where there is no commercially available treatment and no clinical trial in progress, or the patient is not a candidate for a clinical trial.
RRD recognizes that maintaining and expanding access to care is a shared responsibility requiring the involvement of legislators, regulators, payors and others to address the many challenges of providing effective healthcare to their populations. But we also recognize that as a developer and manufacturer of innovative therapies, we too have a part to play in improving access. Therefore, to the extent RRD does have any treatment in development, we may, on a case-by-case basis, consider requests by treating physicians for expanded access for patients in need.
There is no guarantee of expanded access to an investigational product. RRD reserves the right to review and amend this policy statement in the future.