18 Total results for product and free and sample content found
02 Feb 2022
In our rare disease trial strategies whitepaper, see which sponsors lead the way, understand the geographical location of trials, and delve into the types of therapeutic area, plus much more.
Topic rare-disease
27 Nov 2020
Europe’s patchwork of reimbursement agencies make it a daunting place for a small US biotech to do business. Traditionally large pharma partners or CMSOs have provided an experienced hand to hold, but as a new wave of gene therapies and orphan drugs get the EMA green light, smaller drug developers are applying innovation to commercialization and choosing to go solo.
By Mandy Jackson 27 Nov 2020
Phase III results support early 2020 filings for what could be the RNAi specialist’s third commercial product, but two competing drugs for primary hyperoxaluria from Dicerna and OxThera aren’t far behind.
29 Oct 2020
Updated guidance from the UK regulator, the MHRA, outlines the rules on orphan drug applications, criteria and exclusivity periods that will apply in Great Britain after the end of the Brexit transition period
Topic Pharma-Brexit rare-disease
22 Aug 2018
Accrual difficulties force changes that nullify Special Protocol Assessment; ongoing, randomized FOCUS trial in patients with ocular melanoma that has metastasized to liver will become single-arm study and target enrollment will be reduced by 66%.
19 Jan 2018
The European Commission's plans to evaluate the EU pediatric and orphan drugs regulation in tandem.
Topic rare-disease
12 Oct 2017
Biosimilars Development in Autoimmune and Inflammatory Diseases webinar
Topic biosimilars rare-disease
04 Oct 2017
With three products now approved in Europe and/or the US for Duchenne Muscular Dystrophy
Topic rare-disease
25 Sep 2017
Find out about the past, present and future of regenerative medicine.
Topic cancer rare-disease
No Records Found
Getting a demo tailored to your needs is the best way to see how our solutions will help you gain an advantage.
Request live demo now:
Our team is ready to hear from you for a particular request or area of interest. Please do not hesitate to reach out and discuss.
Contact us for product technical and account support.
Have an immediate and specific information need?
Browse and buy from 1000s of analysis and research reports now: