Total BioPharma

Seven top independent companies forge alliance to tackle Orphan Drug access across Asia Pacific

jolene — Wed, 19 Jun 2013 02:00:00 +0000

18th June marks the day of the formation of the Asia Pacific Orphan Drug Alliance (APODA). It was formally announced during World Orphan Drug Congress Asia 2013.

Currently, it has been noted that not many orphan drug reach Asia Pacific patients due to various challenges such as regulatory complexity, low perceived sales opportunity and high commercial barriers. APODA aims to bring together the different Biotechs in the orphan drug industry to provide solutions for 10 markets and 1.8 billion orphan drug patients.

“APODA is the ideal solution for Biotech companies wishing to remain independent from big Pharma but still gain access to Asia Pacific” said Giles Moss, Director of AFT Pharma.

Click here to download the press release to find out more!

Video: World Orphan Drug Congress USA 2013 event highlights

Caroline Hornby — Tue, 18 Jun 2013 22:00:59 +0000

World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.

Watch the event highlights now for insights on who was at the event this past April, what they think some of the main challenges and opportunities are ahead for the orphan drug sector, and why you should be there in 2014.

For more information, go to www.terrapinn.com/orphandrug.

Video: Building a rare disease patient database as a commercial framework

Caroline Hornby — Tue, 18 Jun 2013 21:58:34 +0000

There was an interesting panel discussion at World Orphan Drug Congress USA 2013 on the topic, ‘Small populations, big value: building a patient database can set the commercial framework for pre and post launch success.’

• Engage the patient community to best understand and communicate the true value proposition for patients and caregivers in the US and globally
• Translate value to regulators, payors and investors
• Identify the right programs, services ways to deliver them that ensures therapy uptake and success
• Measure the success of all programs

Panelists included:

• Peter Nalen, President and Chief Executive Officer, Compass Healthcare Marketers
• John Siemienski, Director, Interactive Services, Compass Healthcare Marketers
• David Stefanoni, Head, Early Stage Commercialization, Upsher-Smith Laboratories
• Ramsey Johnson, Senior Director, Clinical and Regulatory Operations, Edimer Pharmaceuticals

World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development. For more information on the 2014 event go to www.terrapinn.com/orphandrug.

Video: Denise Vondohren of Omnicare on critical access services and rare disease patients

Caroline Hornby — Tue, 18 Jun 2013 21:55:23 +0000

Denise Vondohren, VP of Access Solutions at Omnicare Speciality Care Group, presented at World Orphan Drug Congress USA 2013 on the topic, ‘How to integrate critical access services from manufacturer to patient.’

• How to design the “right fit” channel strategy and what is the role of specialty pharmacy
• Determining what patient services are required based on your product and the characteristics of the disease it treats
• How to design financial services necessary to provide optimal patient access
• Third party logistics providing new opportunities for orphan brands — traditional and non-traditional, including direct to physician, direct to patient

Video: Brazilian orphan drug market access panel

Caroline Hornby — Tue, 18 Jun 2013 21:52:54 +0000

World Orphan Drug Congress USA 2013 included an exciting panel on market access in Brazil: ‘How will industry and government working groups in Brazil collaborate towards structuring an Orphan Drug Act?’

• Understanding the 2 proposals from the MOH lead working group and the industry working group
• Where Brazil is headed with this and when can we expect an Orphan Drug Act
• Pricing, reimbursement and co-pay

Participants were:

• Roberto Luiz D’Ávila, Presidente, Conselho Federal de Medicina (Federal Medicine Council)
• Eduardo Matarazzo Suplicy, Senator, Brazil
• Jean Wyllys de Matos Santos, Congressman, Brazil
• Octávio Nunes, Director, Institutional Communications, Interfarma

Video: Scott Gottlieb of American Enterprise Institute on stimulating orphan drug R&D through policy opportunities

Caroline Hornby — Tue, 18 Jun 2013 21:48:20 +0000

Scott Gottlieb, Resident Fellow at the American Enterprise Institute, presented at World Orphan Drug Congress USA 2013 on the topic, ‘How to stimulate orphan drugs R&D through policy opportunities.’

Video: Martine Zimmerman of Alexion on Europe’s regulatory process

Caroline Hornby — Tue, 18 Jun 2013 21:45:31 +0000

Martine Zimmermann, Executive Director Global Regulatory Affairs at Alexion Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Where industry stumbles in Europe’s orphan drug regulatory process.’

Video: Gayatri Rao of FDA on FDASIA provisions and implementation

Caroline Hornby — Tue, 18 Jun 2013 21:42:05 +0000

Gayatri Rao, Acting Director of the Office of Orphan Product Development at the FDA, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Orphan drugs at FDA highlights and next steps: FDASIA provisions and implementation.’

Video: Charles Shasky of Aetna on payers’ financial planning

Caroline Hornby — Tue, 18 Jun 2013 21:39:19 +0000

Charles Shasky, Head of Pharmacoeconomic Comparative Effectiveness Research at Aetna, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Optimizing payers’ financial planning by reducing the level of uncertainty for orphan drugs approval.’

• Initiatives in the marketplace to predict the value of a potentially approved orphan drug
• Reciprocal benefits from orphan drugs coverage among commercial insurances plans in the US
• What is the impact of payers’ deteriorated financial condition on orphan drug developers and patients

Video: John Maddox of Infusion Pharma Consulting on pricing and reimbursement

Caroline Hornby — Tue, 18 Jun 2013 21:36:03 +0000

John Maddox, Managing Director of Infusion Pharma Consulting, presented at World Orphan Drug Congress USA 2013 on the topic, ‘The impact of payers financial reality in shifting the orphan drugs pricing environment.’

• What happens if the pricing latitude for orphan drugs is discontinued?
• How difficult it is to get pricing in a potential low value market?
• Competitiveness in the orphan drug space to benefit payers. What are payers’ ability to pay?

World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.

For more information on the 2014 event go to www.terrapinn.com/orphandrug.