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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 Diseases

In Vivo: 医療用医薬品・医療機器産業の経営層向けニュース

From US To EU: Young Biotechs Going It Alone

By Jo Shorthouse 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.

Topic Market Access rare-disease strategy

Scrip: 世界の医療用医薬品&製薬産業に関するニュース

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-disease


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

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


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

19 Jan 2018

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

Scrip: 世界の医療用医薬品&製薬産業に関するニュース

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

04 Oct 2017

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

Topic rare-disease