Industry News: Soliris, a Shaky Cardiovascular Treatment, and How Pharma Is Changing Due to a Demand for RWE

Keeping up with what’s going on in the industry can be a bit of a headache; there are new studies coming out every day and new initiatives launching all the time. Don’t stress, we’ve neatly chunked together 3 of the most interesting and useful stories from the last month – so you don’t have to! This month we hear about how the pharma world is changing due to a demand for Real-World Evidence. As well as that, we look at two examples of real-world data providing a grounding (or not) for treatments currently in use.

Real-World Data on left atrial appendage closure does not reassure
By John Mandrola for Medscape

A major downside for percutaneous left atrial appendage occlusion (LAAC) is that it leaves a foreign body within the left atrium of the heart. Foreign bodies in the arterial circulation raise concern over thrombus.

Two abstracts presented during the late-breaking clinical-trials sessions at the European Heart Rhythm Association (EHRA) EUROPACE-CARDIOSTIM 2017 meeting addressed this issue. Their results did not deliver reassurance.

Real-World Evidence and pharma
By Dr Nicola Davies for The Pharma Letter

Randomized control trials have long been the gold standard for assessing the efficacy and safety of new drugs, but there is an increasing desire among stakeholders for evidence of how drugs work in the real-world, not just within the confines of a highly controlled experimental environment. No longer is Food and Drug Administration (FDA) approval the ultimate goalpost. As drug prices rise, so resistance becomes more entrenched from government, payer organizations and the patients themselves. Stakeholders now want real world evidence of a drug’s true performance and effectiveness before they make final decisions about pricing and payment.

At the 22nd Congress of the European Hematology Association in Madrid, Alexion Pharmaceuticals, Inc. presented new data from the International Paroxysmal Nocturnal Haemoglobinuria (PNH) Registry that displayed benefits of Soliris (eculizumab) treatment for patients with PNH, regardless of high disease activity status, and for patients with haemolytic PNH and those with PNH with concurrent aplastic anemia.

PNH is an ultra-rare, auto-immune blood disorder that can be diagnosed at any age, and often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years. It has been identified more typically in patients with bone marrow disorders, including aplastic anemia and myelodysplastic syndromes.