Rare Diseases

Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late. According to EvaluatePharma’s forecasts, orphan drug sales are set to grow 11 percent each year between now and 2024, significantly outpacing the overall pharma market, which is set to expand a mere 6.4 percent over the same period.

Neil Dugdale – VP & GM, UK & RoI, Sobi

Three years into his role as general manager of Sobi’s UK and Republic of Ireland operations, Neil Dugdale introduces the scope of operations of the Swedish rare disease player to our audience, highlighting the company’s unique positioning in working closely with patient groups to drive forward better understanding of unmet needs in rare diseases and life-altering conditions.

Why Invest in Rare Diseases & Orphan Drugs?

In the run-up to Rare Disease Day 2019, David H. Crean, PhD, Managing Director for Objective Capital Partners, reviews the investment and deal activity for orphan drugs in 2018 and delves into future forecasts for the rare disease space…

Top 10 Global Orphan Drug Companies Ranking 2017

The top 10 companies in the world ranked by global orphan drug sales and market share in 2017. Swiss giants Novartis and Roche take the top two places, followed by American firm, Celgene.

Rare Disease Company Acquisitions Continue Apace

Following on from Johnson & Johnson’s acquisition of Actelion in January 2017 and Takeda’s takeover of Shire two years later, pharma multinationals’ rush to snap up rare disease specialists continues with Ipsen’s proposed acquisition of Clementia…

Rethinking Clinical Trials in Rare Diseases

Drug development in rare diseases is currently in a very exciting time. While much of the focus is on the scientific advances,  access to rare disease patients and well-designed clinical trials are also essential to evaluating new therapeutics. In this article, PJ Brooks, Program Director at the Office of Rare Diseases Research of the National…

Mythbusting: Patient Access to Rare Disease Treatments

After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica founded Bridge the Gap – SYNGAP Education and Research Foundation to support families of those suffering from the illness and to accelerate the path to better therapies…


Rare Diseases

Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late.

This special report delves into the flurry of activity taking place across the rare disease and orphan drug sector – including in investment, M&A, patient advocacy, clinical trials, regulatory reform and much more.

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Amryt: A Rare Disease Startup Going Global

Formed in 2015, listed UK start-up Amryt targets rare diseases with high unmet medical need. With one commercial asset on the market, a strong pipeline of development assets and a commercial infrastructure across the EMEA region already in place, the company is aiming to expand rapidly.

Orphan Drug Accessibility & Social Medical Coverage in China

Kevin Rufang Huang is the president and founder of the Chinese Organization for Rare Disorders (CORD). A rare disease patient himself, he is responsible for bringing “International Rare Disease Day…

5 Rare Diseases You Should Know

The 28th February 2019, marks the twelfth international Rare Disease Day. On and around this day hundreds of patient organisations from countries and regions across the globe will strive to raise awareness of some of the world’s rarest disease…

A New Era in Access to Rare Disease Drugs in Canada

Durhane Wong-Rieger is the President & CEO of the Canadian Organization for Rare Disorders (CORD). In her first article for PharmaBoardroom, Durhane explains the changing landscape for access to rare disease drugs in Canada and the actors involved in…

Christophe Bourdon – SVP EMEA, Alexion Pharmaceuticals

Specializing in ultra-rare diseases, Alexion targets patient populations of less than 20 patients per million. Christophe Bourdon discusses Alexion’s progress towards developing access for patients across the EMEA region….

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