Sun. Dec 15th, 2019

new standards for new times

In 2007 the European Union regulated the calls advanced therapies from maximum guarantees of safety, quality and efficiency thanks to a centralized authorization system by the EMA (European Medicines Agency). Spain did it some time later, but our regulations have been outdated due to the speed of the progress in research and development; a scientific advance that is allowing spectacular results in fields as sensitive as Hematology and Oncology.

Access to these therapies, which are riding among the medicines and sophisticated health technologies, have become a legitimate aspiration of patients and because of strong controversy regarding its costs. Hence, advanced treatments constitute a major challenge for public health systems. And that advanced therapies are in their initial stage.

After the first steps of cell therapies, today the gene therapies, of the CAR-Ts (Chimeric Antigen Receptor-Cells T). Thanks to them, hematological cancers have potential healing and in the near future they will also defeat solid cancers.

The European Medicines Agency has already granted its authorization to two of them. In the trial phase there is a considerable number. As we said, the price of authorized CAR-Ts is high, which is because its research and manufacturing completely individualized They are very expensive. But their optimal results (saved lives) are there and it would not be honest to relativize them with arguments focused only on the pharmacoeconomics.

It would not be ethical for those who run public health systems to choose to raise bureaucratic barriers to hinder or delay patient access. Some of these barriers may already be emerging in the form of successive commissions, chained with the objective of minimize spending. Commissions in which, by the way, the presence of representatives of the target patients of these therapies.

It would also be to reject that, for economic reasons, the patient is not given access to a CAR-T authorized by the EMA and is only offered to participate in a clinical trial, which will probably be cheaper, but which would lack the advantages of a CAR-T available and authorized by EMA.

Try to relativize the differences among the CAR-Ts, authorized by the EMA, calling the CAR-Ts trials "academic", rather than a clinical trial, it doesn't seem reasonable, because this semantic creativity could contribute to the disorientation of patients. Nor does it help citizens' information to compare, as some do, the price of a CAR-T authorized by EMA with the cost of an essay (the so-called "academic" CAR-T).

The accounts will not be correct, if it is hidden that in trial costs factors such as facilities, equipment or personnel expenses have not been included; costs that, curiously, are "transferred" to the whole of the health system spending.

But that said, no one should argue that public systems they should try to get the best prices and a advantageous financing. However, this should be achieved with new models and new standards, which provide full transparency and total legal certainty.

It is well known that the lack of approval of the Royal Decree on Prices and Financing hinders these objectives. This new regulation is important and urgent task, because there are serious patients who cannot wait indefinitely. Moreover, if we take into account that the current generation of CAR-Ts for hematological cancers will be followed (predictably sooner rather than later) similar therapies for solid tumors.

The Plan for the approach of the advanced therapies, of November of the last year, supposed to recognize the health relevance and economic of advanced therapies, including CAR-Ts. This, in itself, is a positive fact, but the examination of the content of the aforementioned plan reveals insufficiencies and inconcretions, which have already indicated regional authorities and experts.

It seems that they will be corrected and it sure will be, but we would not be on the right track if they are not regulated as soon as possible financing model and guarantees of access to patients For this, public officials should demonstrate sincerity, transparency and fairness. Only then would we get the economic sustainability of advanced therapies and the best health results walk hand in hand.

. (tagsToTranslate) Health

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