Advances in cancer research have achieved in recent years the appearance of drugs capable of acting on the so-called therapeutic targets, which receive the action of the drugs thus avoiding impact on healthy cells. is the call personalized or precision medicine, that gets the use of most effective treatment based on the individual characteristics of each patient, while improving their safety profile.
One of the most significant advances in this field has been the treatments for agnostic tumors, those drugs developed for act on the basis of a molecular alteration regardless of whether the primary tumor has appeared in one organ or another of the body, so that in this case the tumor location loses prominence against the therapeutic target.
However, these new therapies are posing a great challenge in the evaluation process and financing by the Administration. This was stated by the director of the Department of Access Farmindustry, Isabella Pinerosduring the European day Agnostic Tumor Oncologyorganized in Madrid this Tuesday by the Foundation for Excellence and Quality in Oncology (ECO). “In fact, there are currently four treatments for agnostic tumors approved in Europe and presented for commercialization in Spain between 2019 and 2021 that have not yet managed to obtain financing in our country and be available to patients,” said Pineros.
The first challenge arises in the evaluation of the clinical trials themselves. “Usually they are drugs with difficulties in recruitment, because they have a small population of patients in which the inclusion criterion is the presence of the specific biomarker. In addition, they are usually patients who have already exhausted other therapeutic alternatives. All this makes it impossible to perform large scale randomized clinical trials, which makes it difficult to generate conventional evidence that regulators request,” he explained.
In the authorization stage, however -Pineros pointed out- the regulatory agency has understood that these are different drugs and that the evidence they provide is different. “For this reason, the European Medicines Agency (EMA) has granted them a conditional authorization, which meets the premises of safety, quality and efficacy and therefore concludes with a favorable benefit-risk balance.”
The difficulty appears again, he insisted, in the financing and price phase of these innovations. “Financing policies for these medicines vary across Europe.. Some countries place more emphasis on clinical outcomes and comparative clinical benefit assessment, while others, such as Spain, focus more on the cost-effectiveness”, and in the specific case of these drugs, a consideration is added about the high uncertainty of the efficacy results of clinical trials, he stated.
New financing models
To solve the access problem with this type of medicine, Pineros considered it necessary establish a specific financing model for these therapies. “They could be candidates to have conditional financing taking advantage of the current reform of the Law of Guarantees and Rational Use of Medicines,” he said. The Farmaindustria spokeswoman stressed that this new model should combine an evaluation methodology with clear criteria, with the management of therapeutic and economic uncertainty and the contribution to the sustainability of the health system with the different agreements between the Administration and the companies: “We have What to get give access to these new medicinesmonitoring sustainability, and harmonizing its financing with the incorporation of biomarkers in the portfolio of services and adequate coordination throughout the National Health System (SNS)”.
“We have to manage to give access to these new medicines, monitoring sustainability, and harmonizing their financing”
Isabella Pinerosdirector of the Farmaindustria Access Department
A situation similar to that of these treatments also occurs in our country with the evaluation of new therapeutic indications for drugs already financed. “It would also be necessary to establish an approval model for these new indications. Each new indication must go through the funding process again, including the completion of a new Therapeutic Positioning Report (IPT) for each of the indications, and there is no clear criteria for companies to have the indications included in the pharmaceutical service”, pointed out Pineros.
This is especially serious in oncology, where there are clear progress therapeutic and a new indication may involve addressing a type of cancer that have no other treatment alternative financed. “From Farmaindustria we are collaborating with the Administration to try to improve the procedure and put on the table proposals that allow reaching successful financing agreements for our SNS”, he assured.
Efficient incorporation of innovation
Precisely, the efficient incorporation of innovation in hospitals and flexible purchasing models was one of the topics addressed by the director of the Access Department in her speech at the V Meeting of Experts in Health Management and Health Economics, organized this week by the Almirall Chair of the UPF Barcelona School of Management. The Farmaindustria spokeswoman highlighted that for some years now the laboratories have accepted that, together with the price, additional agreements can be established to manage the economic and therapeutic uncertainty that is perceived by the Administration. “What is important at this time is to establish and share with the companies clear criteria for these cases. Progress must also be made in the collection of information: adequate digital platforms are necessary that incorporate the information in a rigorous manner and are usable not only for the Administration, but also for patients, healthcare providers, and industry, ”he indicated.