Ambrose Healthcare Ltd (Ambrose Healthcare), publishes new white paper summarizing regulatory mechanisms to stimulate development of new rare disease treatments. Insights show that leveraging rare disease mechanisms can benefit both businesses and patients – by reducing development costs and accelerating approvals.
Since the US’ FDA first introduced the Orphan Drug Act in the early 1980s to stimulate the development of new drugs for rare diseases, many other countries around the world have followed suit. Now, in the past decade, new treatments for rare diseases have started outgrowing those for non-rare diseases, with one recent report estimating that by 2028 orphan drugs will comprise one in five non-generic prescription sales with an estimated total value of $300 bn.
Even with the increased focus, only around 5-10% of known rare diseases currently have an approved drug treatment.
“As rare disease specialists, we are often met with incredulity about our ambitious plans to expedite change in rare disease”, explains Toby Wilson Waterworth, Co-founder and CEO of Ambrose. “Regulators around the world have recognized that special incentives are crucial to bring much needed rare disease treatments to market, but many professionals are still unaware of all the available support mechanisms. That’s why we commissioned this whitepaper to help spread the information and lead the way for a new generation of companies taking full advantage of the regulatory and financial support available.”
The white paper, titled “Leveraging Regulatory Stimuli in Rare Disease Drug Development” focuses on:
Regulatory incentives
Clinical advantages
Best practices and case studies.
Download the white paper below to explore this topic and access exclusive insights from our interviewed experts: Michael Eging (Acuitiy Strategic Partners), Robert Donnel (Smartway Pharmaceuticals) and Tamsyn Frost (IDEA Regulatory).
With thanks to the authors, Jordan Taylor and Julie Walters.
About Ambrose
Ambrose Healthcare is an award-winning pharmaceutical start-up addressing the global need for rare disease treatments.
With only 5-10% of rare diseases having an FDA-approved treatment, Ambrose is on a mission to get safe and effective therapies onto the market and into the hands of patients quickly and cost-effectively.
For more information, please contact:
Juliet Burns, Corporate Communications