Orphan & Rare Diseases
PPi HC helps companies developing technologies for rare diseases to optimize patient access and to maximize revenues despite challenging pricing environments and sometimes low levels of clinical evidence frequently achievable for such drugs.
Orphan drug clinical development is often challenging due to the burden of access to the targeted orphan patient population. This clinical development remains however frequently associated with (necessarily) high and challenging pricing as investment need to be recouped over relatively small patient populations where there is a high degree of unmet clinical need. PPi HC has provided support on many orphan are rare disease technologies, working with the complex issues specific to such drug development and commercialization. We provide the required expertise to optimize not only orphan disease launch adaptations but commercial and market access strategies through a combination of:
- Primary research intelligence and market insights for orphan drugs,
- Benchmarking of orphan drug HTA reports and price corridors,
- In-licensing reviews,
- Biomarker analysis,
- Launch planning support.
Services
TESTIMONIALS
“With their depth of market access experience across the EU, Adam Barak and his team at PPi Healthcare are well-positioned to provide timely, insightful guidance on pricing and reimbursement. ProMetic’s global strategic launch plans involving ultra-orphan therapies, have been greatly enhanced by our collaboration with PPi.”
Kristine Dorward, Director, Marketing & Business Development, ProMetic Life Sciences Inc.
“I want to let you know that the feedback from my colleagues after your presentation was that they are impressed by the amount of information that you have gathered within the research within such a short period.”
Ramona Schmid, Global Market Access, Pricing and Reimbursement Manager, HRA Pharma
“I have worked with PPi on several projects and I will work with them again. The first project was a European pricing study for an orphan product. Having already completed one study with a competitor, I found the PPi team to be particularly insightful, having taken the time to completely understand this complex project.”
Thomas W. MacAllister JD, PhD, Vice President of R&D and General Counsel, Remedy Pharmaceuticals
“Recently I completed an assessment to inform the collection of pharmacoeconomic data in a clinical study in the US and Europe. The team again were very insightful and we had unexpected value-added when we found a member of the PPi had been involved in the pricing and reimbursement activities relating to one of our comparables. Great team, great results!”
Thomas W. MacAllister JD, PhD, Vice President of R&D and General Counsel, Remedy Pharmaceuticals
