Orphan Drugs and Rare Diseases Conference 2018

SMi Group17 - 18 October, London, UK.
Patients with rare diseases cannot continue to be overlooked because of costly therapies and have the same rights to treatment as any other patient. At this year's Orphan Drugs and Rare Diseases conference, organised by SMi Group and taking place in London, industry experts will address the opportunities and challenges within the rare disease treatment field.

Keep up to date with the industry and to learn from expert speaker panel, bringing you important new case studies and reports on this year’s relevant topics.

Highlights in 2018

  • Discuss the pricing and reimbursement of orphan drugs
  • Understand the challenges of patient recruitment and patient-centric research
  • Hear the MHRA's perspective on benefit-risk assessment in rare diseases
  • Explore the clinical development of orphan drugs for rare disease
  • Gain insight into potential synergies between regulators and assessors

Speakers include

  • Carina Schey, Researcher, University of Groningen
  • Xavier Ortega, Project Manager, Rare Diseases, Minoryx
  • Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd
  • Yolanda Barbachano, Senior Statistical Assessor, MHRA
  • MichaleBouskila-Chubb, Head of Business Development, Healx

Interactive Workshop (16 October) run by JG Zebra Consulting will be looking into question: "Working together for HTA in rare diseases - a step too far or the way forward?"

Orphan Drugs and Rare Diseases conference this year is set to inspire the international rare diseases community by exploring synergies between regulators, health and technology assessors

For further details, please visit:
http://www.orphandrugs.co.uk

About SMi Group

Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world's most forward-thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at http://www.smi-online.co.uk

Most Popular Now

Cannabis extract helps reset brain function in psy…

Research from King's College London has found that a single dose of the cannabis extract cannabidiol can help reduce brain function abnormalities seen in people with psyc...

For first time in 40 years, cure for acute leukemi…

Acute myeloid leukemia is one of the most aggressive cancers. While other cancers have benefitted from new treatments, there has been no encouraging news for most leukemi...

New cancer treatment uses enzymes to boost immune …

Researchers at The University of Texas at Austin have developed a new approach to treating cancer using enzyme therapy. The enzyme, PEG-KYNase, does not directly kill can...

Bayer accelerates six new startups

Changing the experience of health: that's the focus of the six startups which the Bayer G4A team has included in the Accelerator program this year. The young companies fr...

Novartis receives European Commission approval of …

Novartis today announced that the European Commission (EC) has approved Kymriah® (tisagenlecleucel, formerly CTL019). The approved indications are for the treatment of pe...

Shire completes sale of oncology franchise

Shire plc (LSE: SHP, NASDAQ: SHPG) announces today that it has completed the sale of its Oncology franchise to Servier S.A.S. for $2.4 billion. The franchise includes the...

Antioxidant reduces risk for second heart attack, …

Doctors have long known that in the months after a heart attack or stroke, patients are more likely to have another attack or stroke. Now, a paper in the Journal of the A...

Novartis to divest the Sandoz US dermatology busin…

Novartis today announced it has agreed to sell selected portions of its Sandoz US portfolio, specifically the Sandoz US dermatology business and generic US oral solids po...

New tablet production facility in Ingelheim: Cente…

Boehringer Ingelheim held a groundbreaking ceremony for the construction of a new production facility for innovative drugs. This new Solids Launch facility will focus on ...

SOLAR-1 trial of Novartis investigational alpha-sp…

Novartis today announced the global Phase III SOLAR-1 trial evaluating the investigational alpha-specific PI3K inhibitor BYL719 (alpelisib) has met the primary endpoint s...

Tezepelumab granted Breakthrough Therapy Designati…

AstraZeneca and its partner Amgen Inc. (Amgen) today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tezepelumab...

Pfizer terminates domagrozumab (PF-06252616) clini…

Pfizer Inc. (NYSE: PFE) announced that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrop...