The approval by the European Medicines Agency (EMA) and the Italian Agency (Aifa) of the update of the technical data sheet “allows de facto that agalsidase alfa, indicated as enzyme replacement therapy for patients with Fabry disease, can be self-administered by part of a trained patient or caregiver” at home, says Barbara Capaccetti, Medical & Regulatory Director Takeda Italy. “An advantage for the patient in terms of quality of life, but also for the caregiver – he underlines – A saving of resources in terms of time to access the facilities and receive the administration, but also for the patient who may feel a little less sick”.
This is “an important update that is based on the tolerability and manageability profile of aglasidase alfa and an additional way of accessing treatment in addition to the already existing ones, i.e. hospital or assisted home care”, adds Capaccetti, recalling that “the decision of switching to self-administration must be evaluated by the attending physician after training of the patient or in any case of whoever assists him “. Another aspect that should not be underestimated is that “this update also allows for an advantage in terms of a lower risk of exposure to infections – highlights the medical director of Takeda – We have learned from the pandemic how much ensuring that you have home therapy can reduce the risk of a contagion and we know how important it is for these patients to ensure continuity of therapy”.
As evidence of the biopharmaceutical company’s innovation and attention to developing therapies “that can make a difference for patients”, argues Capaccetti, there is a figure: “A third of our pipeline has received the status of breakthrough designation”. Moreover, “the commitment in R&D translates into” a commitment of “more than 5 billion dollars a year with an increase in investments, in this last year, of around 10%”. Looking to the future, “the goal for the next 3-5 years is to invest 70% of the portfolio in rare diseases – he continues – We know how much rare diseases, especially lysosomal storage diseases, if not adequately diagnosed and treated, can evolve rapidly to serious and disabling conditions, up to the death of the patient”.
The company’s commitment is on several fronts. “First of all – lists Capaccetti – to increase awareness with training programs for health personnel to have a correct diagnosis in a short time: the diagnostic delay ranges from 4 to 7 years” and is “due to a series of factors: the lack of information, the number and complexity of rare diseases do not help.Takeda also supports screening programs through rapid and simple diagnostic tests that allow patient management in a timely manner”, alongside “patient support programs, offered through qualified partners, who help ensure continuity of care and therapy”.
“There are no diseases, but sick people – reflects Takeda’s medical director – That’s right. Through support programs, we can provide a service by trying to respond to the specific needs of the patient: from psychological support programs, rather than physiotherapy or training, up to home therapy”.
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