Some truly bold words have been first written, then published last month in the Value & Outcomes Spotlight. Allan M. Korn, their author, emphasizes in his short ‘Value of Observational Data in Health Care Decisions’ that the risk of staying too long in a status quo ‘safety’-zone might very well backfire.
Almost as if he had his own text on the right and Michael Rawling’s Oration on the left, he diminishes the everlasting value of RCTs by saying that ‘at the conclusion of a typical RCT we only know that, for a group of 200 ideal patients with no comorbidities, concurrent medication or behavioral problems, FDA/EMA requirements were met.’
Nothing new, indeed, but it does seem it needs to be stressed from time to time. However, Mr. Korn doesn’t stop there. Invoking the robust area of oncology he claims that ‘observational studies must inevitably replace RCTs in many clinical settings’, what can be considered as a step further in comparison to widely acknowledged Rawling’s legacy. He also implies that rules and guidelines concerning observational research methodology are ‘fairly well defined now’. This brings me to link it together with another quite recent publication of the ISPOR Task Force, that developed ‘’A Questionnaire to Assess the Relevance and Credibility of Observational Studies to Inform Health Care Decision Making’’. It is a truly interesting lecture, describing step by step what one needs to be asking themselves in the context of a well-designed study protocol. A must-read to those wishing to evaluate the pros and cons of their non-interventional way of reasoning.
Furthermore, Mr. Korn also touches upon the much sought-after new way of thinking, logic, ethics and business sense, which he all describes with two words only, adaptive licensing.
Moreover, he also attempts to redefine value, while urging to use the same methodology in assessing the value of a product as the industry has learnt to do with human life. The PAPY (Patient-Adjusted Product Year) was born, still closely related to its cousin QALY. From this point of view, the value of a product should evaluated against the lack of other therapeutic options, enhancement of survivability, lowered morbidity, similar outcomes for less money, or, better outcomes for same money. Mr. Korn also points out that such an evaluation would not stifle the R&D, on the contrary, it can become a testimony of a real, accurate, monetary value (even seemingly high at first sight) of numerous drugs, such as Gleevic in CMML patients (chronic myelomonocytic leukemia).
He concludes with a memorable phrase, "Health care is dangerous. Transparency and an understanding of impact of innovations and discoveries on real patients in their own real worlds will make it safer. Much safer".
All in favour?
Read the article here.