pharmaphorum

NOVEMBER 19, 2021

The cost-effectiveness agency had turned down Evrysdi (risdiplam) in draft guidance published in June, saying it was too expensive, but after negotiations with Roche has reached a deal that will make the drug available to around 1,500 people in England. .

Drugs 98

Drugs Gene Therapy Genetic Disease Gene 98

pharmaphorum

APRIL 20, 2021

The cost-effectiveness agency has said that anti-CD20 antibody Kesimpta (ofatumumab) can be prescribed via the NHS in England and Wales as a treatment for adults with RMS with active disease, as either a first-line therapy or after alternative drugs have been tried. billion by 2028. billion in sales in 2028.

Sales 111

Sales Medicine Drugs Marketing 111

Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 15, 2020

The huge rise in the use of telemedicine services during lockdown has brought care directly to patients in their homes, while pharma’s communications with healthcare professionals (HCPs) has experienced a similar push to digital channels.

Branding 130

Branding Marketing Pharma Companies Life Science 130

Rethinking Clinical Trials

JULY 6, 2023

During the COVID-19 pandemic, several regulatory authorities around the world introduced guidance on DCTs. The European Medicines Agency (EMA) released a separate guidance around DCTs in December 2022. While similar to the FDA draft guidance, the EMA guidance differs slightly in its emphasis and lack of certain topics.

Trials 130

Trials Clinical Trials Clinical Trials Licensing 130

pharmaphorum

NOVEMBER 19, 2020

Because Roche has never applied for Avastin to be used in wet AMD, clinics in the US have had to choose between the peace of mind offered by the regulatory process and Lucentis’ higher price tag, or the unapproved off-label formulation of Avastin at a lower price.

FDA Approval 64

FDA Approval Marketing Drugs Clinical Development 64

pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Comments can be submitted until 23 June. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52