Why Rare Disease Therapeutics Need Early Market Access Planning
When developing and bringing a therapeutic for a rare disease to market, sponsors should think with the end in mind and plan for payer requirements early in protocol design. The economic and clinical value evidence required by payers for market access differs from the clinical efficacy and safety evidence demanded by regulators. Securing approval but lacking market access results in unrealized revenue and, most importantly, patients left untreated. But how do we price and how do we gain access on the promise of long-term value when we don’t have the long-term data?
In this podcast, originally broadcast by The Conference Forum's PHARMA talkRADIO, Sangeeta Budhia, VP and Global Head, Pricing and Market Access, and Wyatt Gotbetter, Worldwide Head, Access Consulting, of Parexel discuss early market access planning, including inclusive data collection, payer models and the impact of new regulations and innovative trial design on data collection and ongoing evidence provision.
Related Insights
Blog
Unpacking NICE’s review of the HST routing criteria: implications for manufacturers
Oct 3, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Whitepaper
Overview of China's Market Approval Policy Med Insurance Payment System
Apr 7, 2022
Webinar
China's Market Approval Policy and Medical Insurance Payment System for Rare Disease
Jul 21, 2022
Report
New Medicines, Novel Insights: Advancing rare disease drug development
May 22, 2023
Blog
U.S. price reforms 2022: How can drug manufacturers best prepare for the Inflation Reduction Act?
Aug 17, 2022
Whitepaper
Addressing the Challenges of Artificial Intelligence used for Data Extraction in Systematic Literature Reviews
Mar 15, 2023
Whitepaper
The Chinese Pharmaceutical Market: Size, R&D, Regulations, Market Access and Innovations
Mar 9, 2023
Whitepaper
Reimbursement framework for medical devices in India
Mar 7, 2023
Playbook
Insights from the 2022 R&D Innovation Survey
Feb 17, 2023
Playbook
Are you using real-world evidence?
Feb 1, 2023
Blog
Innovative modeling method could speed patient access to critical IO therapies
Jan 4, 2023
Related Insights
Blog
Unpacking NICE’s review of the HST routing criteria: implications for manufacturers
Oct 3, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Whitepaper
Overview of China's Market Approval Policy Med Insurance Payment System
Apr 7, 2022
Webinar
China's Market Approval Policy and Medical Insurance Payment System for Rare Disease
Jul 21, 2022
Report
New Medicines, Novel Insights: Advancing rare disease drug development
May 22, 2023
Blog
U.S. price reforms 2022: How can drug manufacturers best prepare for the Inflation Reduction Act?
Aug 17, 2022
Whitepaper
Addressing the Challenges of Artificial Intelligence used for Data Extraction in Systematic Literature Reviews
Mar 15, 2023
Whitepaper
The Chinese Pharmaceutical Market: Size, R&D, Regulations, Market Access and Innovations
Mar 9, 2023
Whitepaper
Reimbursement framework for medical devices in India
Mar 7, 2023
Playbook
Insights from the 2022 R&D Innovation Survey
Feb 17, 2023
Playbook
Are you using real-world evidence?
Feb 1, 2023
Blog
Innovative modeling method could speed patient access to critical IO therapies
Jan 4, 2023