Orphan Drugs Summit 2012

Highest categories of drugs in development in the US are cancer, blood cancer, skin cancer, genetic disorders, neurological disorders, infection diseases. Find out more, click here to read the 2011 Report Orphan Drugs in Development for Rare Diseases, presented by Pharmaceutical Research and Manufacturers of America.

The Orphanet report of January 2012 lists all orphan drugs that have received a European marketing authorisation. As well stated at the report, “these medicinal products may now be accessible in some, though not necessarily all, European countries. In reality, the availability of a certain orphan drug in a certain country depends on the strategy of the laboratory and the decision taken by national health authorities concerning reimbursement.”

« Lists of Orphan Drugs in Europe », Orphanet Report Series, Orphan Drugs collection, January 2012, http://www.orpha.net/orphacom/cahiers/docs/GB/list_of_orphan_drugs_in_europe.pdf

A recent article by Putnam Associates describes the reasons behind the rise of investments in orphan drugs. These are divided between the uniqueness in the development process and the economics of commercialising orphan produts.

Smaller trial sizes with lower overall development costs, 26% less clinical development time on average and financial incentives, such as 50% tax credit on clinical development costs are amongst the unique features of OD development process.

Amongst other factors, the ability to generate a larger than average share of revenue outside the US and the the first-mover advantage support the economics of commercialising orphan drugs.

Read the short article here:  http://www.putassoc.com/company/news_raising_orphans.html

After reviewing our successful launch event, which joined over 115 participants, we have expanded opportunities to bring our platform for experience sharing to a next level: by combining well-designed topics, with key stakeholders at our speaking panels, with even more extensive networking and business opportunities.

The second Orphan Drugs Summit 2012 is the platform for experience sharing amongst Orphan Drugs professionals like you, looking at alternative ways to raise funding, to overcome development challenges and to partner with stakeholders to ensure orphan drugs reach the market of rare diseases patients.

The 2012 agenda truly reflects the current challenges faced by the orphan drugs community. Don’t miss the latest information on:

• Pricing & Reimbursement across European Member states: value-based pricing, affordability and “significant benefit” are a few of the issues to be discussed.

• Global regulatory issues: OD designation, collaboration initiatives, patient registries and regulatory issues in the US, Europe and Japan

• Grant, incentives, large pharma partnerships, funding & financing: sustaining resources to build your orphan drug pipeline. NEW

• Scientific case studies with dynamic ways to overcome OD clinical trials´ challenges: ensure quality and safety by designing  effective clinical trials. NEW

• Ultra Orphan Drug & Paediatric Drug development issues, as part of our informal round table discussions NEW

If you are looking at having serious business critical conversations to take this industry forward, to raise investments to your organization or to overcome practical challenges such as pricing & reimbursement, then join us in Denmark in September 2012!

Beatriz Viellas
Senior Project Manager
Pharma Division

For delegate enquiries:

E-mail: info@midfieldmedia.com
Tel: +46 (0) 8 650 02 70

Listen to Paul Tunnah, Managing Director at pharmaphorum  interview with Wills Hughes-Wilson, Vice President Health Policy Europe at Genzyme: Inside view: rising to the challenges of orphan drug development

Ahead of her presentation at the upcoming OrphanDrugsSummit.com 2011, pharmaphorum spoke with Wills Hughes-Wilson, Vice President for Policy in Europe at Genzyme, to understand more about the challenges and opportunities presented by developing drugs for the rare disease space. During our discussion, we explored the regulatory landscape around orphan drugs, the collaborative nature of working in this space and what success looks like for pharma, healthcare providers and the patient.

You can also read a summary of the interview directly on pharmaphorum.com.

Linking product indication and orphan drug designation as a broader business strategy is a key issue for pharmaceutical companies looking at driving the business value of their orphan drug development. This is the focus area of Harald Fricke’s presentation being held at the Orphan Drugs Summit 2011 in Copenhagen.

Harald Fricke, the Chief Operating Officer and Chief Medical Officer for Apogenix has an extensive experience within pharmaceutical clinical development and with international management positions, including at Fresenius Biotech GmbH, Baxter Oncology’s clinical development department, SmithKline Beecham and GlaxoSmithKline.

Radisson Blu Royal Hotel Copenhagen: 

Hammerichsgade 1
DK-1611 Copenhagen
Denmark
Phone: +45 33 426000

For more information please visit:

http://www.radissonblu.com/royalhotel-copenhagen

Join the Orphan Drugs Summit September 14-16th in Copenhagen!
www.orphandrugssummit.com

The International Exchange of Knowledge for Treating Rare Diseases

Over 55 million people are estimated to suffer from a rare disease in Europe and in the US. Global estimates are between 5000 to 7000 rare diseases. New rare diseases are discovered every week and many have no treatments available. * In the past 10 years, the European Union (EU) Regulation on Orphan Medicinal Products has yield over 500 orphan designation and 69 new medicinal orphan products have been approved for the European market.  At the same time, during the past 25 years, the US FDA has granted orphan drug designation to about 2,100 drugs, of which 344 have are now approved products in the US.**

Are you ready for the 2011 orphan drugs market challenges and leveraging opportunities?

Dear Colleague,

The market earned revenues for orphan drugs is estimated to reach USD 27.09 billion in 2015*. It is no surprise that this niche market is now attracting an even higher interest from not only small and medium enterprises, but from large pharmaceutical companies looking at adding orphan drugs products to their blockbuster portfolio and capture new revenue streams.

At the Orphan Drugs Summit 2011, key market players, regulators, HTAs/payers and patient organizations will meet to discuss how to support the development of orphan drugs and the real challenges to the pharmaceutical industry and patient organizations, including pricing, reimbursement and market access.

The 2011 market dynamics demands new approaches and collaboration amongst stakeholders to support orphan drug development and offer ensure patients gain access to new therapies and drugs.

Attend the Orphan Drugs Summit 2011: be part of the orphan drugs business market and gain insight into the policies of tomorrow.

I look forward to welcoming you to Copenhagen on 14 – 16th September 2011!

Beatriz Viellas

Senior Project Manager
Pharma Division

• • Approval processes, pricing and value demonstration to support reimbursement and commercialization of your orphan drugs in the most favorable European markets.
• • Market access, marketing approval and partnerships to safeguard patient access to orphan drugs globally
• • Orphan drugs market dynamics: big pharma alliance, partnership and business models to gain new markets and the impact to the existing orphan drugs market praxis.
• • Improved clinical trials, diagnosis and assessment to ensure safety and speed up patient access to orphan drugs.
• • EU & US orphan drugs regulations, collaboration initiatives and processes for designation and approval of orphan drugs.