More than half of the new drugs entering the German healthcare system have not shown to add benefits, researchers said in a statement. BMJ today.
Beate Viseuer and colleagues from the German Health Technology Assessment Agency IQWiG (Institute for Quality and Efficiency in Healthcare) say that international processes and policies for drug development are responsible and need to be reformed.
Between 2011 and 2017, IQWiG evaluated 216 medicines that enter the German market after approval by regulators, they explain. Almost all of these medicines are approved by the European Medicines Agency for use across Europe.
However, only 54 (25%) were judged to have significant or significant additional benefit. In 35 (16%), the added benefit was either insignificant or could not be quantified. And for 125 drugs (58%), the available evidence does not show any additional benefit from standard care in the approved patient population.
The situation is particularly shocking in some majors, they add. For example, in psychiatry / neurology and diabetes, additional benefit is shown only at 6% (1/18) and 17% (4/24) of the evaluations, respectively.
Some people argue that limited information during regulatory approval (and thus widespread use by patients) is the price to be paid for early access to innovative medicines, the authors explain.
But reality seems completely different. For example, an assessment of cancer drugs approved by the EMA between 2009 and 2013 shows that most of them were approved without evidence of clinically meaningful benefits for patient outcomes (survival and quality of life), and several years later little has changed.
Moreover, post-marketing research often does not happen and in the world regulators do little to sanction non-compliant companies, they write.
Evidence also suggests that many of the investigational drugs add benefit only to subgroups of patients. "For the general population of patients, the current outcome of drug development can lead to even less progress than our estimates suggest," the authors said.
Clinicians and patients deserve unbiased and complete information on what to expect from a particular treatment, including information on the benefits of alternative treatments or without treatment, the authors write. But given the current gaps in information, this is not possible.
"As a result, patients' ability to make informed treatment decisions in line with their preferences can be compromised, and any healthcare system that he hopes to call" patient center "does not meet his ethical obligations.
They believe that regulators need to become much less tolerant of shortened drug development programs and instead require solid evidence from longer-lasting and sufficiently large randomized controlled trials to demonstrate efficacy and safety that can be parallel used to collect health technology assessment data.
Information gaps may be further closed by a mandatory requirement to conduct active controlled studies, they add. Although cost recovery and pricing decisions "should avoid stimulating marginal results for patients or results based on very uncertain evidence but rather rewarding the achievement of relevant results."
In the longer term, health policy makers need to take a more active approach, they write. "Instead of waiting for the pharmaceutical companies to decide what to develop, they could define the needs of the health system and implement measures to ensure the development of the necessary treatments."
They conclude: "Combined action at EU and national level is needed to define public health objectives and review the legal and regulatory framework, including the introduction of new drug development models, in order to achieve these goals, and to focus on what should be the top priority in healthcare: the needs of patients. "
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