Compassionate Use Programme

The compassionate use program represents a crucial instrument in the health and biotechnology field, designed to provide access to innovative treatments or medicines that are not yet on the market, especially for people with serious or life-threatening diseases who lack therapeutic alternatives.

The legislation setting compassionate use in Spain is mainly set out in Royal Decree 1015/2009. This decree establishes the criteria and procedure for access to treatments not yet authorized, ensuring that this access is carried out in a monitored way and based on the available scientific evidence.

This program prioritizes the immediate response to patient needs, relying on research to transform medical advances into concrete solutions. In this way, accessible and effective alternatives are offered to patients in critical situations where an urgency is demonstrated.

Value for Research

For companies developing drugs or new therapies, this system offers significant value, as it allows them to collect valuable data on the efficacy and safety of their developments in genuine patients, while completing the regulatory process to access the market.

These practices at the hospital environment are the best example of how to bring research into the clinical setting in a regulated and fast-track manner. This opportunity anticipates the scientific validation of investigational treatments and broadens their impact on improving the quality of life of hundreds of patients.

Benefits for Patients

Compassionate use becomes a vital solution for patients facing unmet medical needs, allowing them to access medicines in advanced stages of development prior to formal approval.

The process requires an application by the treating doctor, who must justify the absence of suitable therapeutic alternatives and obtain the informed consent of the patient or their legal representatives. It must follow the protocol required by the AEMPS (Spanish Agency for Medicines and Health Products), which regulates this practice for all hospitals in the country. It should be noted that access to treatment is completely free of charge for the patient, as priority is given to access to therapeutic options that benefit their health.

Under this framework, Mikrobiomik, a Basque company born to research, develop and produce biological drugs based on gut microbiota, has demonstrated the potential of MBK-01 at the end of its phase III clinical trial. MBK-01 is its innovative gut microbiota-based drug, in oral capsule format, delivered in a single dose of four capsules. Since 2023, more than 60 patients in Spain with severe Clostridioides difficile infection have been treated with the drug in 19 hospitals across the country. Most of these cases corresponded to critically ill patients who did not respond to conventional antibiotic treatments, facing frequent recurrences and severe episodes of diarrhoea.

While the data obtained will be analyzed in a retrospective study and are still confidential, the fact that clinicians are relying on the treatment by administering MBK-01 indicates that its therapeutic profile is promising. This reflects how compassionate use not only benefits patients, but also drives clinical research and strengthens the biotech industry’s commitment to public health.

A Commitment to the Future

The compassionate use programme highlights the importance of collaboration between the healthcare sector and research. The results obtained represent a valuable opportunity to demonstrate the efficacy and safety of investigational medicines for patients with unmet medical needs. This evidence, although generated outside the traditional clinical trial setting, can provide clinically relevant data to support their therapeutic impact and justify regulatory approval. It would be important for regulatory agencies to consider these data to facilitate early access to innovative treatments for those without viable therapeutic alternatives. Incorporating this data into the regulatory process would be critical to accelerate the availability of new therapeutic alternatives.

This is a clear example of how innovation can be applied to solve critical needs and ensure that scientific advances benefit those who need them most.