Briefs
Rare diseases present unique challenges to companies looking to commercialize treatments.
Although there is vast potential in rare disease, each space is different and unique.
Companies must effectively address challenges around identifying and treating patients—as well as providing long-term support. It is critical that companies understand the nuances in each disease.
Keeping a patient-first mentality, advancing patients’ needs and providing excellent support services are mission-critical for life sciences companies touching rare disease. However, lack of disease awareness and unclear referral…
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Blog
Published July 21, 2023
For life sciences companies focused on rare diseases, accurate patient finding is a worthy challenge—one deserving dedicated time and resource to tackling and solving. The benefit of enrolling even one new patient is large, both for the lives of patients in need and the commercial success of the therapies.
Why is patient finding in rare disease such a challenge?
The hallmarks of a rare disease work against traditional targeting methods: small patient population sizes, complex disease recognition, lengthy roads to…
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Blog
Published March 18, 2022
Key Takeaways
An analysis was conducted of all the orphan therapies registered by the FDA and ANVISA before and after 2018 to effectively evaluate approval times once the RDC 205 pathway was implemented. The main takeaways from the analysis can be found below:
The difference between FDA and ANVISA time to approval for orphan drugs is four times shorter after the implementation of the RDC 205 / 2017 pathway
The RDC 2015 pathway regulated the deadlines for the regulatory and…
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Blog
Published August 19, 2021
Executive Summary
While 7 additional rare disease drugs were included on the 2020 NRDL in China, this development comes amid a backdrop of rare disease policy development signaling a move towards multi-channel funding mechanism to support more widespread patient access and maintain sustainability of the NRDL
As such, manufacturers cannot solely rely on NRDL inclusion for their rare disease product market access strategy in China and should consider alternative funding opportunities outside of the NRDL (e.g., provincial critical illness budgets,…
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