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9 Total results for product and free and sample content found

In Vivo

From US To EU: Young Biotechs Going It Alone

By Jo Shorthouse 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 Diseases Strategy

Scrip

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

By Mandy Jackson 27 Nov 2020

Alnylam_Seeking_Lumasiran_Approvals

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

Pink Sheet

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

By Sue Sutter 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 Approvals Rare Diseases FDA

Pink Sheet

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

By Vibha Sharma 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 Diseases

Datamonitor Healthcare

Future of Regenerative Medicine

24 Sep 2017

Future of Regenerative Medicine

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

Topic Cancer Rare Diseases

Pink Sheet

Disease Awareness Campaigns Still Must Tread Carefully, Observer Says

By Mark W. Sherman 03 Jun 2017

Disease Awareness Campaigns Still Must Tread Carefully, Observer Says

Sponsored plotline for General Hospital was careful to avoid irking US FDA; agency withdrew its guidance on disease awareness campaigns in 2015.

Topic Rare Diseases

Datamonitor Healthcare

Discover Datamonitor Healthcare's Cystic Fibrosis Infographic

07 Mar 2017

Discover Datamonitor Healthcare's Cystic Fibrosis Infographic

Over the last few decades, there have been steady improvements in the prognosis of patients suffering from cystic fibrosis.

Topic Rare Diseases

Scrip

Orphan drug pricing and the next five years

01 Mar 2017

Orphan drug pricing and the next five years

Feb 28 is Rare Disease Day. To mark the occasion, Scrip asked experts the question: Do you see orphan drug pricing coming under pressure over the next five years?

Topic Rare Diseases

A Bird’s-Eye View of the Rare Disease Landscape

By Doro Shin 27 Feb 2017

A Bird’s-Eye View of the Rare Disease Landscape

Over 350 million people worldwide are suffering from rare diseases. Which is why it is necessary to understand the rare disease landscape to date.

Topic Clinical Trials Diseases Rare Diseases

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