Posted on Oct 25, 2018
With debate raging about the European Commission’s plans for health technology assessment (HTA), the ESMO Congress in Munich was told that some European countries take more than twice as long as others to reach HTA decisions in respect of reimbursements for new cancer drugs in the wake of European Medicines Agency (EMA) approval.
The average decision time is in excess of a year in certain countries, Congress heard.
According to a study unveiled in Munich, once the EMA has approved a new treatment, many countries evaluate its benefit and cost-effectiveness through a HTA process as part of making a decision on whether to reimburse use of the treatment for routine patient care.
Looking at all of the new cancer drugs approved for solid tumours by the EMA between January 2007 and December 2016, researchers tracked the time between EMA approval for each of the drugs and HTA decisions being taken by health authorities in four European countries.
The countries were England, France, Germany and Scotland. The median time from EMA approval to HTA decisions was two-to-three times longer in England (405 days) and Scotland (384 days) compared to Germany (209 days) and France (118 days).
Study co-author Dr Kerstin Vokinger, senior research scientist at the University Hospital of Zurich, Switzerland, and affiliated researcher at Harvard Medical School, Boston, in the US said that the differing amount of resources invested in such assessments and different national regulations may lead to variation in the time from EMA approval to HTA decisions in different countries.
The study found that health authorities generally made decisions much more quickly for drugs ranked as being of highest benefit on ESMO’s scale, compared to those with less clinical benefit. But the variation in time from EMA approval to HTA decisions remained between different countries for even the highest benefit drugs.
Analysis also showed that virtually every cancer drug ranked as being of highest were approved for reimbursement by all four countries: Germany (100%), Scotland (95%), England (92%) and France (90%).
EAPM is, of course, heavily engaged in the debate on HTA and is taking the discussion to Member State representatives next month.
Congress heard that securing access to optimal cancer care for all patients can only be achieved through integrated, sustainable translation of today’s scientific advances into tomorrow’s treatments. This must be reinforced by a clear understanding of the magnitude of clinical effects and accurate identification of patients most likely to benefit.
ESMO’s Scientific Chair, Professor Solange Peters, stressed the importance of ensuring that the innovations reach the right patients at the right time – a phrase often used by EAPM and other proponents of personalised medicine.
In Munich, data was presented from over 2000 submitted abstracts, representing nearly 116,000 patients who have taken part in clinical trials.
“ESMO is a driver of change affecting the entire field of oncology,” said its President, Josep Taberno, adding that the organisation is “a major contribution to securing access to optimal cancer care for all patients, wherever they live.”
Another study released in Germany during the ESMO event highlighted barriers to the inclusion of 12 to 25-year-olds in both adult and paediatric early-phase clinical trials. This suggests a need for more tailored approaches to give these patients better access to therapeutic innovation.
In Europe, the legal minimum age to participate in adult clinical trials is usually 18 years. Study author Aurore Vozy, from Gustave Roussy Institut de Cancérologie in Villejuif, France told attendees: “We know…that certain girls will develop genetically driven breast cancers very early in life: there are no paediatric trials for this disease, yet these patients are systematically barred from participating in the relevant adult trials.”
She added: “The situation is similar for some adolescents with lymphomas or sarcomas, whose tumours often resemble those of adults much more closely than those found in children.” Congress heard that, in rare cases, adults in their early 20s are also diagnosed with tumours most commonly seen in children. Paediatric clinical trials, meanwhile, typically set an upper age limit of 18 or 21 years.
The research found that, among the 389 trials not open to adolescents, 55% could have been relevant for underage patients. “This means that patients have been denied access to innovative medicines which were available at the very centre where they were being treated, and to which they may have had a better response than to conventional therapy,” said Vozy.
The European Alliance for Personalised Medicine has at its core solid campaigning for better access to clinical trials as well as the many innovative treatments that are now available in theory.
Said EAPM executive director Denis Horgan, who was in Munich for the Congress: “This is yet another concrete example of situations in which patients are being denied access to potentially excellent treatments. In the era of personalised medicine this is clearly unacceptable. Young people being denied the chance of a better life? It can’t be allowed to happen in the 21st century.”
A new scale for tumour DNA mutations which will simplify and standardise choices for targeted cancer treatment has been agreed by leading cancer specialists in Europe and North America. The scale, called ESCAT (ESMO Scale for Clinical Actionability of molecular Targets), has been published in the Annals of Oncology and aims to optimise patient care by making it easier to identify those cancer patients who are likely to respond to precision or personalised medicines, and help make treatment more cost effective.
Professor Fabrice André, Chair of the ESMO Translational Research and Precision Medicine Working Group who initiated the project, said: “Doctors receive a growing amount of information about the genetic make-up of each patient’s cancer, but this can be difficult to interpret for making optimal treatment choices,”
“The new scale will help us distinguish between alterations in tumour DNA that are important for decisions about targeted medicines or access to clinical trials, and those which aren’t relevant,” he added.
The paper’s lead author, Dr Joaquin Mateo, Principal Investigator of the Prostate Cancer Translational Research Group from the Vall d’Hebron Institute of Oncology, Barcelona, Spain, said: “For the first time, ESMO has created the tools to make it clear what data are needed for a mutation to be considered actionable and how this may change in response to new clinical data.”
EAPM’s work in the area of clinical data has, of course, included the launch of its MEGA initiative ADD LINK and the Alliance is now working with a coalition of willing Member States in order to share vital genomic data across borders for the benefit of patients in all parts of the EU and beyond.
An upcoming ESMO workshop entitled ‘Gender medicine meets oncology’, to be held in Lausanne, Switzerland, on 30 November and 1 December, will see a multidisciplinary faculty of experts discuss concepts and methods of gender medicine and their implications for clinical practice and research in oncology. Dr. Anna Dorothea Wagner from Lausanne University Hospital, who initiated the workshop and co-wrote a recent paper on the subject, “the need for further research to understand gender impacts in oncology is significant”.
Wagner added: “With efforts to include sex aspects in biomedical research abounding in other areas, like cardiovascular medicine, it was high time for us in the oncology field to take notice.”
Her paper had already pointed to various areas in which gender differences have been known to exist for many years but are still poorly understood. The meeting will focus on the differences in body composition, hormones, genetic makeup and metabolism. As one example, women experience higher toxicity with certain types of drugs, which is likely a result of their metabolising these differently from men, due to factors that are thought to range from higher body fat levels to differences in the activity of drug-metabolising enzymes. “Toxicity is a problem in itself, not least because it can cause cancer patients to discontinue treatment,” Wagner said.
While gender differences in efficacy are reported more often, those relating to toxicity are only rarely analysed and systematically reported.
In the era of personalised medicine, differentiating between the sexes where applicable is clearly a forward step, in oncology and elsewhere.
A European Commission briefing paper recently outlined five research missions and 10 industry partnerships to receive funding from Horizon Europe, the EU’s 2021-2027 research programme.
These proposed would account for 40-50 per cent of the €94.1 billion programme.
Two of the five missions suggested cover digitization and health, with health innovation, for the rapid development, deployment and safe use of medical treatments, devices and technologies to be enhanced by digital technologies.
Meanwhile, the plan sees global health, including links to national health research systems and philanthropic funding being boosted alongside key digital technologies, including novel technologies such as AI and linking to downstream sectors.
As it stands, missions should gain around 10 % of the budget in the first years of Horizon Europe, with around €1-2 billion going to each.
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