This piece was co-authored by Pierre Moïse, Engagement Leader with Quintiles' Advisory Services

Pharmaceutical companies have been investing heavily in oncology research in recent years. All of this investment has resulted in the launch of dozens of innovative treatments that have significantly improved the prognosis for cancer patients around the world. Today, two-thirds of US cancer patients live at least five years, compared to just over half of patients in 1990. As there are currently hundreds of investigational anticancer therapies in development, together accounting for approximately 30% of research pipeline, it is expected that this trend will continue.

But it has been suggested that, in some instances, drug launch prices may not be proportional to the health benefit these treatments provide. Launch prices of oncology drugs in the US have been rising steadily over time, from roughly $100 a month in the 1970s, to more than $10,000 a month today (i.e., an annual increase > 10%). While in Europe, Canada and Australia, healthcare technology assessments (HTAs) are often used to evaluate the cost-effectiveness of cancer treatments, this approach has generally not been adopted in the US. This may be one of the reasons why drug prices in the US tend to be higher than in other industrialized countries.

It is against this background that several health-related organizations have recently launched frameworks to assess the value of anticancer therapies. Some examples include the American Society of Clinical Oncology’s (ASCO) Value Framework, the National Comprehensive Cancer Network’s (NCCN) Evidence Blocks, and European Society for Medical Oncology’s (ESMO) Magnitude of Clinical Benefit Scale.

Benefits and limitations

Each of the seven frameworks we identified aims to assess the value of oncology drugs in a transparent and objective manner. Their stage of development, target audience, the value dimensions that are taken into account, and scoring methodology varies, however, which can sometimes lead to the same drug receiving quite dissimilar ratings in different frameworks.

Some of the tools mainly aim to inform the treatment decisions that physicians and their patients have to make, while others focus on the interests of payers. They all use available data and/or clinical expert opinion to evaluate a variety of criteria. Obviously, clinical efficacy and safety are key elements of every assessment tool. In general only the results from comparative randomized clinical trials (RCT), but not data from single-arm studies or real-world evidence, is used. Other criteria such as unmet medical need, burden of disease, quality of life, novelty, cost-effectiveness and economic impact are taken into account in some, but not all, frameworks. In total, we identified 13 different value elements across the frameworks we reviewed.

We also found that the majority of the frameworks have been criticized for not being sufficiently patient-centered as quality of life measurements, patient-reported outcomes, and the preferences and needs of patients are rarely given much weight if considered at all. While not necessarily designed to inform payer decision-making, there is concern that these frameworks may be used to limit treatment exposure as a way of controlling drug costs, which could ultimately limit how physicians tailor care to the individual needs of their patients.

The industry does need to do a better job of justifying drug prices in relation to the value its products bring, and the organizations that are developing these tools should be commended for their efforts to quantify and clarify this value equation. Though with several tools still in early development, it is important for all stakeholders to consider the limitations of and differences between the various frameworks when interpreting their outputs. It remains to be seen whether the different approaches used by each of these frameworks will converge in the future, but harmonisation would help limit confusion and aid stakeholders in making informed decisions in cancer patient care. We hope this poster, and other presentations at ISPOR about value frameworks will further the dialog and spur collaboration between industry stakeholders to determine the best course of action going forward.