Rare Diseases

Novartis drug Promacta receives FDA Breakthrough Therapy designation for first-line use in severe aplastic anemia (SAA)

January 4, 2018

Data supporting designation showed over half of treatment-naïve SAA patients achieved complete response with Promacta when given with standard immunosuppressive therapy, with overall response rate of 85%[1] Promacta is the only TPO receptor agonist indicated for the treatment of patients with SAA, currently in the refractory setting Regulatory filings of the first-line indication in US […]

CRF Health’s TrialMax Touch® Selected by Leading Specialty Pharmaceutical Company for Pediatric Rare Disease Phase II Study

November 20, 2017

Plymouth Meeting, PA – November 14, 2017:  CRF Health, the leading global provider of patient-centered eSource technology and service solutions for the life sciences industry, has been selected by a leading specialty pharmaceutical company for its latest pediatric rare disease Phase II study. The trial will utilize CRF Health’s TrialStudio®, TrialMax® and TrialManager® eCOA solutions […]

Comprehend Systems Appoints New CEO in Response to Surging Demand for Clinical Intelligence Solutions; Enterprise Growth Expert to Scale Company and Expand Partner Ecosystem

November 20, 2017

Redwood City, Calif., November 13, 2017—Comprehend Systems, Inc., the leader in Clinical Intelligence solutions, has named Terry Cunningham CEO.  Cunningham will focus on scaling Comprehend in response to explosive customer demand for solutions that reduce time and risk in clinical trials. Cunningham will also drive expanded partner programs to help sponsors achieve faster, better clinical […]

Bioclinica Acquires MDDX Research & Informatics Combining Advanced Image Management Cloud Technology with Its Scientific and Medical Imaging Leadership

November 7, 2017

Best-in-class solution ensures sponsors and CROs have global access to efficient, accurate, and scalable medical imaging to drive better data and outcomes DOYLESTOWN, Pa., Nov. 6, 2017 – Bioclinica®, the world-leading provider of scientific-enabled Medical Imaging, eHealth, and patient-centric solutions supporting clinical research, today announces it has acquired Silicon Valley-based MDDX Research & Informatics, adding […]

Facilitating a drug discovery ecosystem: Genestack joins the Milner Therapeutics Institute

October 30, 2017

“As sequencing technology shrinks to the size of a key ring and emerging technologies make it possible to track changes to the genome at single cell level, then all of a sudden genomics data becomes a streaming and wearable dataset,” says Imad Yassin, Vice President of bio data management company Genestack. He is explaining why […]

Locating Patients with Rare Diseases

October 13, 2017

Why a Qualitative Lead Generation Approach Is a Must-Have Component of Any Orphan Drug Sales Strategy Finding patients with rare diseases requires both a quantitative and a qualitative approach. By taking analytics one step further and using a hands-on approach, TrialCard’s Virtual Engagement team has identified health care professionals (HCPs) currently treating patients with rare […]

Worldwide Clinical Trials Continues International Speaking Tour at OCT Nordics

October 10, 2017

Clinical Trial Patient Recruitment and Retention Expert to Present “Improving Patient Recruitment and Engagement in Orphan Disease” MORRISVILLE, NC, Oct 9, 2017 –Worldwide Clinical Trials continues its international speaking engagement tour on Tuesday, October 10, 2017 at the Outsourcing in Clinical Trials (OCT) Nordics 2017 conference in Copenhagen, Denmark. Director of Global Patient Recruitment and […]

Demand for genetic testing drives growth for award winning Elucigene diagnostics

September 18, 2017

Having the right technology at the right time has made Elucigene Diagnostics a world leading developer and distributor of prenatal, rare disease and infertility genetics diagnostics. The Manchester-based company has been selected as the winner of the Venture-backed Management Team 2017 award for the Northwest in the prestigious British Private Equity & Venture Capital Association […]

Lysogene Launches World Class Clinical Advisory Board

September 13, 2017

PARIS & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a leading biopharmaceutical company pioneering gene therapy technologies to treat central nervous system diseases, today announced the appointments of leading experts to its newly formed Clinical Advisory Board (CAB). The CAB will provide strategic advice to Lysogene as it continues to advance its clinical […]

The New Reality: Maximizing Value in Rare Disease Launches

September 7, 2017

The rare disease landscape is changing: with assets being granted expedited review, launch is no longer a fixed point in time, and research and development (R&D), medical affairs, and commercial teams need to collaborate early in the process to reach critical consensus on where to focus, when to engage key health care provider (HCP) stakeholders, […]

DDL to Exhibit at 7th edition PFS and Injectables Summit in San Diego

June 30, 2017

Recognizing the rapid developments in both technical and regulatory landscapes in injectable devices, the 7th edition PFS & Injectables Summit will return to San Diego with a growing audience and trending topics on September 25 – 27. With an increased focus on device quality and material safety, we are delighted to announce DDL’s joining as […]

Santhera Selects Veeva CRM and Veeva OpenData as Commercial Software and Data Platform

June 28, 2017

PLEASANTON, CA — June 28, 2017 — Veeva Systems (NYSE: VEEV) announced today that Santhera Pharmaceuticals selected Veeva CRM and Veeva OpenData as a commercial software and data platform for its first product launch. The rare disease pharmaceutical company is adopting Veeva Commercial Cloud solutions to establish a consistent, global approach to commercialization. Accurate customer […]

Improved Patient Outcomes and Financial Performance

May 18, 2017

Neither orphan drug manufacturers nor their patients with rare diseases are served well by the traditional multi-channel model of drug delivery and patient service. A new integrated model increases adherence and improves patient outcomes, which in turn leads to better financial performance for manufacturers.