LentiGlobin: a potential gamechanger in thalassaemia therapy

By Jack Rawson, Analyst Email: jrawson@partners4access.com October 2018 turned out to be a good month for β-thalassemia patients seeking new and effective treatments. The European Medicine Agency (EMA) accepted US-based biotech firm bluebird bio’s marketing authorization application (MAA). The new gene therapy – LentiGlobin – could be a gamechanger for patients with transfusion-dependent β-thalassemia (TDT).…

CAR-T reimbursement: Lessons learnt from NICE assessments

By Max Rex, Analyst Email: mrex@partners4access.com It’s been difficult to keep up with the flurry of reimbursement news coming out of England relating to the recently approved CAR-T therapies Kymriah (Novartis) and Yescarta (Gilead). Despite only having gained EMA (European Medicines Agency) approval in late August this year, England’s medicines watchdog, NICE (National Institute of…

NICE balks at Spinraza price, rejects reimbursement in its draft guidance

By Christina Poschen, Consultant Email: cposchen@partners4access.com Biogen’s Spinraza, a treatment for the rare genetic disease spinal muscular atrophy (SMA) is currently making headlines for its high price and promising outcomes.  Various European countries including Spain, Italy as well as The Netherlands and Belgium just made the product available to their patients or confirmed they will…

Slow Brexit proceedings frustrates biotech industry

By Aparna Krishnan, Corporate Affairs Lead As the UK Prime Minister’s office is preparing for the cabinet meeting at Chequers this Friday, British businesses including the biotech industry are none the wiser on what lays ahead after March 29, 2019 when the UK exits the European Union. The current discussion is focussed on the Brexit…

Cancer diagnosis – a step forward for England

New rapid diagnostic and assessment centres are being piloted in ten areas across England in a focused approach to identify cancer early.  These new centres are part of the NHS plan to meet the new faster diagnosis standard, where patients with suspected cancer should receive a diagnosis or given the all clear within 28-days. The…

PRIME – two-year assessment – was it good for biopharma?

The PRIority Medicines Scheme (PRIME) of the European Medicines Agency (EMA) was started two years ago to support and optimise medicine development. The scheme provides early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. The EMA has published their two-year review and not surprisingly more than…

Irish patients continue to be denied vital new drugs

Todays’ article in the Irish Times[1] highlights again that patients in Ireland continue to be denied nine drugs which are already approved for use by the National Centre of Pharmacoeconomics (NCPE). They have been waiting on average for 2 years for the Health Service Executive (HSE) to approve them for reimbursement. The drugs concerned range…