Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

Rare Disease Day 2021

NICE and ‘ultra orphans’

NICE and its processes for ‘highly specialised therapies’

EU flag

J&J gets EU green light for novel TB drug

Sirturo approved to treat multidrug resistant form of the disease

National Institute for Health and Care Excellence NICE logo

NICE: Alexion must justify Soliris’ high cost

UK body unable to recommend ‘ultra-orphan’ drug

- PMLiVE

Pharma companies back Rare Disease Day

Genzyme, Shire and more hold global campaigns today

- PMLiVE

AZ gets limited approval for metreleptin in US

First FDA-backed treatment for rare disease lipodystrophy

Define the patient burden in rare diseases

Putting patients and carers at the heart of the value demonstration equation in orphan drugs is key

- PMLiVE

FDA backs BioMarin drug for rare enzyme disorder

Vimizim is first drug approved in US under rare paediatric disease priority review

- PMLiVE

EMA to expand orphan drug HTA collaboration

Will further develop the concept of parallel scientific advice in 2014

- PMLiVE

Orphan drugs: A ‘responsible’ investment

There's growing sense of responsibility in the orphan drug market, but the market remains under-exploited by companies

- PMLiVE

Supportive strategies for demonstrating value

Improved planning will support better data generation and improve understanding of rare diseases and the orphan drugs that treat them

- PMLiVE

FDA approves sleep disorder drug for blind people

Vanda Pharmaceuticals wins backing for Hetlioz to improve a person’s body clock

The thing about small numbers

Marketers working in orphan drugs need to take a narrower, deeper approach to relationship building, and look to medical centres of excellence

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links