Investigator initiated trials in Portuguese rheumatology.

a Investigação Clínica) and the experimental drug (INFARMED) should be required for the trials including IIS. Clinical trials testing medical devices follow the same rules as medications, with some particularities depending upon the type of trial and device’s specificities. To conduct IIS, cooperation among several stakeholders is necessary, which represents an organizational effort for clinical investigators, hospitals, academia, research sponsors, patients, physicians, and regulator. Each stakeholder offers a different set of tools to support the essential components of a clinical trial. In fact, Clinical Investigation Centres based at Hospitals, can be of help to the investigators to develop and implement clinical trials. Furthermore, academic institutions either Universities or research Institutes have facilities such as biostatistics, legal support and communication offices. Pharmaceutical industry is a fundamental partner for the success of an IIS, through the promotion of research grants and a know-how for conducting RCTs. Together, these stakeholders create the infrastructure that supports clinical research. The Portuguese Rheumatology Society (SPR) has recently supported 3 IIS in distinct areas: the GO-DACT, the biomarkers in AS and ViscoOA. GO-DACT is a nationwide multicentre, interventional, double-blinded, placebo-controlled, parallel design trial of golimumab in combination with MTX versus MTX monotherapy, in MTX-naïve psoriatic arthritis patients with active dactylitis. This trail aims to demonstrate differences of efficacy of golimumab in combination with MTX in comparison with MTX monotherapy, in improving dactylitis at 24 weeks versus baseline, in MTX-naïve PsA patients. GO-DACT Includes patients older than 18 years, with the diagnosis of psoriatic arthritis according to the CASPAR criteria, and ≥1 tender dactylitis, refractory to at least two systemic NSAIDs, at optimal dosage, for 3 months. Each subject will participate in the trial for a maximum of appro ximately 32 weeks from the time the subject signs the Informed Consent Form (ICF) through the final contact. After a screening phase of approximately 28 Randomized controlled clinical trials (RCTs) are considered the highest level of evidence in human experimental medicine. Usually designed to assess the efficacy and safety of new therapies or treatment strategies, that ultimate led to its approval and application in cli nical practice; they are, in the majority of cases, funded and conducted by a pharmaceutical company. Investigator initiated trials (IIS) are often a unique way to ans wer particularly relevant questions to clinical practice. They complement available safety and efficacy information and sometimes the mechanism of action, of a specific therapy. While searching at clinicaltrials.gov, 835 IIS can be identified from a total of more than 200,000 registered studies, indicating that ISS are still a small fraction of the running clinical studies. IIS needs to be encouraged and supported in order to improve evidence-based decision-making and consequently bring maximum public health benefits. The development of an IIS starts from a clinical research question. Indeed, physicians while providing medical care for patients often are confronted with re levant clinical questions: which treatment is the best option for this disease or symptom? Is there any mar ker of treatment response? Which symptom is predictor of a poor outcome? In order to test the clinical hypothesis, the adequate design (blinded/open, controlled/non controlled), treatment-arms, length of thera py, permitted concomitant therapy have to be defined at an early phase of protocol development. The outcome measures for accessing the primary (and secon dary) endpoint have to be carefully chosen to ensure validity of results. Finally, regulatory aspects must be taken into account, and approvals of the National regulatory authorities that guarantee the protection and confidentiality of patients data (Comissão Nacional de Protecção de Dados), ethic issues (Comissão Ética para