White Papers

Gene therapy is becoming a promising, novel and innovate approach in treating diverse inherited diseases. Ophthalmology is a specialty in which many advances in this area have been achieved. By using different genetic engineering techniques, vision is preserved in those patients who, otherwise, were confined to lose their eyesight in the past. Thus, this breakthrough treatment will bring a new hope in an area where no treatment was available.

Atopic dermatitis is a chronic, pruritic, inflammatory skin disease that occurs most frequently in children, but also a great number of adults. Clinical features of atopic dermatitis include skin dryness, erythema, oozing and crusting, and lichenification. While many patients with  mild or moderate disease can be managed with topical and traditional treatments, there are unmet needs for chronic and severe cases. New; and developing therapies such as small molecule inhibitors and biologic hold promise for real advances in management of this complex disease.

Epidermolysis bullosa is a heterogeneous group of rare disorders characterized by mechanical fragility of epithelial tissues. Researchers around the globe are investigating new therapies to improve the quality of life of patients with little or even non-available therapeutic options so far, particularly for the more severe variants. New horizons seems could be opened in the coming years in one of the ”Worst Disease You’ve Never Heard Of ”according to DEBRA (Dystrophic Epidermolysis Bullosa Research Association of America).

Real-world evidence (RWE) derives from data collected outside of highly-controlled randomized clinical trials (patient reported outcomes, registries, databases, surveys, etc). RWE is rapidly gaining importance in healthcare decision making, as reflected in the various guidances that have already been issued or are planned by the EMA and FDA. In this review, we introduce the concept of RWE, describe the main RWE study designs, comment on the most relevant criteria that have to be taken into account, and include the TFS-RWE algorithm to find the most suitable design for you. Finally, we provide an overview of the European Union and United States regulations on RWE.

Immuno-oncology (I-0) has emerged as a promising area of research in oncology. The diverse investigational products that are currently under development in both the United States and the European Union target a plethora of biological mechanisms and pose unique challenges for clinical researchers. Innovative trial designs that take into account ethical considerations, adequate data collection and measurements, and the use of biomarkers as predictive or prognostic markers have helped researchers in designing and executing clinical trials that can provide valuable effiacy information. Furthermore, comprehensive planning and risk management in these high risk trials, which can have enhanced and unexpected toxicities, are imperative for the trial’s success and an adequate safety data collection. In this white paper we present in a clear and concise manner study design and execution strategies as well as data collection points and measurements necessary to undergo a successful I-O clinical trial.