by Many anti-cancer drugs approved by the European Medicines Agency (EMA) between 1995 and 2020 lack evidence of additional benefit, particularly those approved under a “fast track”, according to a study published today by ‘The BMJ’. And although the pharmaceutical industry claims that high drug prices are needed to offset research and development (R&D) costs, results show that more than half of these drugs, including those with minimal added benefit or null, you will recover the investment made in three years. For César Rodríguez, president of the Spanish Society of Medical Oncology SEOM, “the study reveals that a significant proportion of cancer drugs approved by the EMA between 1995 and 2020 offer little added therapeutic value, and that this is particularly evident in those approved through accelerated or special routes. Related News standard No A mega study finds more than 30 harmful effects of ultra-processed R. Ibarra The research, carried out by a team from the University of Utrecht (Netherlands) aimed to evaluate the additional benefit and financial results of oncology drugs, while exploring different EMA approval pathways. Using data from multiple organizations and publicly available financial reports, researchers identified 458 additional benefit evaluations for 131 oncology drugs and revenue data for 109 oncology drugs. Researchers found that many of these drugs lack evidence of additional benefits. In fact, 41% of the 458 evaluations were negative, indicating that no additional benefit was (or could not be) determined. In this group, medications approved through a special relationship, called accelerated pathway, were preferably found. This system facilitates faster market inclusion of drugs intended for patients with an unmet medical need. To this end, the EMA accepts less evidence at the time of drug approval, provided that more studies are carried out to gather additional evidence of effectiveness and safety. This limited evidence may be one of the reasons why an additional benefit cannot be established. Specifically, Rodríguez highlights, when a total of 458 potential benefits of approved drugs are evaluated, “up to 41% of them are non-existent or unquantifiable.” This work, comments Rafael López, vice president of the ECO Foundation and head of the Medical Oncology Service of the Santiago University Hospital Complex (CHUS), concludes that we must “see or study if the current regulatory and pricing system promotes and facilitates the development of more effective medications for patients. The benefit of a drug, explains López, is defined as its therapeutic value, compared to the standard and is collected from what is published by technology evaluation agencies (USA, France, Germany and Italy), medical societies ( ESMO and ASCO) and the French newsletter Prescrire. However, César Rodríguez clarifies, “it is very important to keep in mind that the approval of drugs by the European Medicines Agency does not correspond to the drugs financed and, therefore, available for use, by each of the Member States.” . , and in particular for Spain. Not all EMA approvals (and especially conditional or accelerated ones) are funded and approved by our Health System. In fact, the drugs with the least evidence available in EMA approval (often with conditional approvals) are those with the lowest percentage of funding and, therefore, it is likely that the impact of the results of this study will be of much smaller magnitude in our environment. Accelerated and conditional approvals by EMA have a low representation among the drugs financed for oncology in Spain. That is to say, as the president of SEOM points out, accelerated and conditional approvals by EMA have a low representation among the drugs financed for oncology in Spain. Rising spending Global spending on oncology drugs is expected to rise from $167 billion in 2020 to $269 billion in 2025. What’s more, an increasing number of oncology drugs are approved based on less robust evidence , raising concerns about the misalignment of incentives in the pharmaceutical market with the interests of patients. The authors emphasize that approving medications through an accelerated route is not necessarily a bad thing since, says researcher Lourens Bloem, in many cases “there is a great need for new medications.” Bloem explains that accelerated approval can be a way for patients to benefit from a new drug, but, she emphasizes, “it is essential to continue monitoring the real added benefit of these types of drugs and evaluate their costs.” For Rodríguez, it is evident that the approval and financing of oncological drugs must take into account “the value provided in terms of effectiveness, quality of life, and the prioritization of approvals must take into account not only economic criteria, but also the clinical criteria that allow for selection.” those therapeutic innovations whose added value is truly relevant. Approving drugs through fast track is not necessarily a bad thing since there is a great need for new drugs. In this sense, López points out that all oncological medications are approved by the EMA. In Spain, some medicines do not have the price and financing so they cannot be used in the public sector. But the conclusions believe that they can be extrapolated to all of Europe. Regarding the projected increase in global spending on oncological drugs in the availability and accessibility of treatments for patients in Spain, this expert emphasizes that the system must be sustainable, “but it is indisputable that those drugs that provide substantial benefits should be prioritized.” Obviously, López adds, global spending on Oncology is going to increase because the incidence of cancer is increasing, and it is also the greatest cause of suffering in Western societies, which causes an urgent unmet need that we have to address. Availability and accessibility is closely linked to the economic and social development of societies. Without a doubt, more drugs will be developed that possibly increase the results in survival and/or quality of life, but that will require an economic effort from societies. However, he points out, “doing a piecemeal analysis of simple spending on cancer drugs is a mistake. Spending on oncology must be contextualized in the increase in cancer incidence, the presence of longer survivors, longer life expectancy and better quality of life. Even beyond health, spending on cancer has not increased relative to GDP in recent years. “It would be interesting to analyze the data from a country’s overall spending perspective.” Furthermore, the study reveals that more than half of anticancer drugs, including those with little or no added benefit, can recover their research and development (R&D) costs within three years. According to the researchers, this challenges the pharmaceutical industry’s claim that high drug prices are necessary to offset R&D expenses. Analysis shows that drugs with higher levels of added benefits generally generate higher revenues. “While lower levels of added profits could lead to lower revenues, these are still high enough to recover R&D expenses,” the research team writes in BMJ. This may decrease the pharmaceutical industry’s incentive to develop high-value drugs, the researchers argue, “because pharmaceutical companies may be satisfied with the revenue generated by lower-value drugs.” With global spending on oncology drugs expected to increase significantly in the coming years, the authors argue that regulatory and reimbursement processes need to be better organized. This could help encourage pharmaceutical companies to continue searching for drugs that offer significant additional benefits, and not settle for drugs that offer minimal additional benefits but still generate acceptable revenues. Regulation Regarding whether policies in the field of regulation and reimbursement of medicines should be modified to ensure equitable and sustainable access for patients to innovative treatments, Rodríguez considers that what is desirable is that “procedures are standardized.” and the way of evaluating is always based on the same criteria, in a rigorous manner, so that any innovation that provides clear therapeutic value can reach patients in a way that is not only equitable but also agile. Incorporating clinical experts into the processes is undoubtedly a necessity. López concludes that the European Union, through the European Commission and the European Parliament, is carrying out a reform of pharmaceutical legislation with the aim of achieving more accessible, incredible and innovative medicines. On the other hand, there are movements at the European level to at least homogenize the financing process and that result in fewer inequalities than the current ones, although this task will be difficult due to the different socioeconomic development of nations. “We urge policymakers to constantly re-evaluate new and ongoing initiatives aimed at ensuring fair, affordable and sustainable patient access to innovative and expensive medicines,” the authors write in an opinion piece, also published in ‘BMJ’. «In addition, we highlight the importance of researching and promoting the rational use of these medications in clinical practice. “This approach aspires to a future where drug development and limited resources are more closely aligned with real-world benefits for patients.”
