Ahead of the launch of proposals to amend the European Union’s (EU) legislation on pharmaceuticals, Prof Jean-Yves Blay, ESMO’s Director of Public Policy, recently gave an interview to POLITICO - a major news outlet covering developments in EU affairs and a must-read for policy makers. Speaking, in addition to his role at ESMO, as a rare cancer specialist at the Léon Bérard Comprehensive Cancer Center in Lyon, Prof Blay highlighted the challenges presented by rare cancers and opportunities offered by this revision.
In the interview, Prof Blay explained that two categories of rare cancers can be distinguished.
The first category concerns subtypes of common cancers - such as for example lung, breast, and colorectal cancer - that are identifiable with a certain mutation in a given gene. Prof Blay highlighted that it is estimated that between 0.5 percent to 5 percent of cancer patients may carry such a mutation, which is sufficient to conduct randomized clinical trials.
The second category are those rare histological subtypes of cancers with an incidence of one in a million or less per year, where it is difficult to set up clinical trials with the goal of finding new treatments. Prof Blay highlighted that “As academic researchers, or even together with pharma, we will not be able to have a series of one thousand patients and this is where we have the challenge of proving the utility (of a drug).” For this category, and noting the important role that ESMO’s recently launched Real Word Data and Digital Health Working Group can play here, he highlighted the importance of real world evidence in determining the benefits of new drugs for treating certain rare cancers.
Prof Blay also underlined the value of personalized treatments for the second, more difficult, category of rare cancers: “We have very long term survivors - and when I say very long term survivors, the experience is sometimes years or decades with a complete return to normal life.”
The European Commission is set to launch legislative proposals for revising the EU’s legislation on pharmaceuticals in April 2023, which will also cover EU rules governing medicines for rare diseases (this legislation has not seen substantial revision in the last 20 years). ESMO will engage with the EU institutions throughout this revision to ensure that the needs of rare cancer patients and oncology professionals are addressed fully in the updated legislation.