Insights from ISPOR DC

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RTI Health Solutions had over 45 researchers attending and moderating sessions, conducting short courses, and presenting their research this year at the ISPOR 21st Annual International Meeting in Washington, DC. Here are some of their takeaways.

From Josephine Mauskopf, PhD
Vice President, Health Economics

There is currently great interest in “Value Analysis Frameworks,” with different organizations proposing different frameworks that include a variety of cost and benefit endpoints as well as input from a variety of stakeholders including patients, providers, payers, manufacturers, and government on behalf of society. The ISPOR Vaccines Task Force Value Framework presented at a Forum in DC also points out that different vaccine decision makers have different contexts in which they optimize value (vaccines only, health care only, all government funded activities) as well as with or without a constrained budget. Therefore, they need different types of analyses to assess vaccine value (e.g. traditional cost-effectiveness analysis, extended cost-effectiveness analysis, budget optimization modeling, and fiscal services modeling).

from Kati Copley-Merriman, MS, MBA
Vice President, Outcomes

The Issue Panel, “What's Next for Value Frameworks for Perscription Drugs?” focused on value frameworks for prescription drugs and provided good insight into the various approaches to demonstrate value – for example NCCN includes clinical efficacy, safety/toxicity, and affordability while ASCO considers survival and toxicity. Ultimately, the panelists agreed upon the importance of understanding that these frameworks are intended to help committees – not individuals – make decisions. Multiple stakeholders (e.g., pharma, payers, physicians, patients and policy makers) will need to engage in the evaluation and application of existing and future value frameworks.  Are these value frameworks the start of a US HTA or HTA-like shift in our healthcare system?

from Ari Gnanasakthy, MBA, MSc
Head, Patient-Reported Outcomes

Interesting question from an issue panel related to the challenges of assessing symptoms in rare diseases: Researchers often report findings of treatment benefit using various metrics (example: % reduction from baseline, absolute reduction from baseline, % of patients archiving a certain threshold). However, treatment benefit as assessed by different metrics may lead to different conclusions. Should we ask physicians the metric most useful to them to make an appropriate decision? 

The Prescription Drug User Fee Act (PDUFA) led to the availability of many new drug to patients sooner without compromising FDA’s high standards for safety, efficacy, and quality. Since the first user fee law was passed in 1992, PDUFA has been reauthorized four times. The current legislation, PDUFA V, is set to expire in September 2017. Under PDUFA V The Patient-Focused Drug Development program has been successful in systematically obtaining patient perspectives on certain diseases and related treatments, and patient-reported outcomes took prominence in drug development. An important aspect of PDUFA VI, to be implemented from October 2017, will be the use of ‘real world evidence’ for the purpose of drug approval. However, appropriate methodology needs to be developed and validated.

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