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18 Total results for product and free and sample content found

Rare Disease Trial Strategies

02 Feb 2022

Rare Disease Trial Strategies

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

From US To EU: Young Biotechs Going It Alone

27 Nov 2020

Analysis

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.

Topic Market Access rare-disease strategy

Alnylam Seeking Lumasiran Approvals Soon, But Has Two Rivals On Its Heels

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. 

Topic Clinical Trials Launches Rare Diseases

How To Get Orphan Drug Status In Great Britain From 2021

29 Oct 2020

Pink Sheet Brexit Orphan Drug Status

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

US FDA's Regulatory Flexibility To Be Tested As Delcath Overhauls Melphalan Trial Design

22 Aug 2018

US FDA's Regulatory Flexibility To Be Tested As Delcath Overhauls Melphalan Trial Design

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%.

Topic clinical-trials drug-approval rare-disease fda

Pink Sheet

Pharma Wants Clarity On Joint Evaluation Of EU Paediatric And Orphan Regulations

19 Jan 2018

Pharma Wants Clarity On Joint Evaluation Of EU Paediatric And Orphan Regulations

The European Commission's plans to evaluate the EU pediatric and orphan drugs regulation in tandem.

Topic rare-disease

Biosimilars in Inflammatory and Autoimmune Disease

12 Oct 2017

Biosimilars Development in Autoimmune and Inflammatory Diseases webinar

Topic biosimilars rare-disease

Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity

04 Oct 2017

Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity

With three products now approved in Europe and/or the US for Duchenne Muscular Dystrophy

Topic rare-disease

Future of Regenerative Medicine

25 Sep 2017

Future of Regenerative Medicine

Find out about the past, present and future of regenerative medicine. 

Topic cancer rare-disease

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