Ensuring payer support, delivering stakeholder benefit and generating commercial returns: Pharma's growing orphan drug challenge
Rising competition and closer scrutiny by hard-pressed payers are just two of the trends that are challenging pharma's orphan drug ambitions. Success hinges on ever closer relationships with patients, HCPs and KOLs to create therapy awareness, conduct research and build evidence of value. But what are the patient engagement, market access and pricing strategies that will deliver success in this changing landscape?
In Orphan Drug Commercial Models: Strategies for Success, orphan drug experts from leading pharma companies speak out on the current commercial imperatives for companies working in the orphan drug space and the strategic refinements necessary to meet the challenges of today.
Orphan drug experts answer tough questions
What orphan drug experts say…
"Aligning with your market access team is crucial. Building a pharma economic model together with the reimbursement and pricing process is key for small population drugs like orphan drugs. The support of the medical affairs team is also crucial when you want to make a strong value story of what the treatment means for patients. As orphan drugs are not commonly seen by HCPs, the medical affairs team can help you raise awareness regarding the disease."
Richard Van Leeuwen
Merck
"With regards to the price, the starting point is understanding the value and the innovation of the product; the real benefit that this new drug is bringing to the patients. Beside this, we usually consider many different factors, which we believe are equally important. However, it varies from product to product and country to country. The factors and methods that we take into account can usually be divided in two different groups: a) external factors like benchmarks set by similar products (particularly those that target the same indication or are in the same drug class), health system budget impact and payer willingness; and b) internal factors like our assessment of the drug's potential and its running and maintenance costs or the expected return on investment to take into due consideration the costs for development."
Luigi Longinotti
Recordati
"We have learned that to keep patients at the centre of drug development, and to determine how best to support them when the product enters the market, you need to actually talk to patients, their caregivers, and the advocacy societies that work with them every day. In addition, we need to talk to providers who have a genuine interest in helping patients and listen to their feedback. It's all about spending time listening to those stakeholders and acting on their feedback."
Parth R Shah
Amgen
What to expect
Expert contributors
The report harnesses critical insights from front-line experts who completely understand the commercial and market access challenges of bringing orphan drugs to market. To allow for candid responses, we have agreed to keep some of their names anonymous.
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