Science has developed very rapidly, and for a large number of diseases, there are already therapeutic alternatives that allow recovery, improve the quality of life or increase life expectancy.
We live in unprecedented times when it comes to technological innovation in modern medicine, with the scientific community increasingly committed to developing more effective and safer treatments. We seek to find solutions to prevent the development of diseases or mitigate their severity.
A good example is the development of cryopreservation services for mesenchymal stem cells from umbilical cord tissue. Stem cells, also called mother cells, have the ability to produce specialized cells that make up various tissues and organs in the body.
Its unique properties allow the replacement of cells that die throughout life as well as the repair of damaged tissues, which can contribute to the treatment of various diseases.
However, the unmet medical needs in the field of genetic diseases are undeniable. Gene therapy is a new therapeutic paradigm that goes beyond traditional disease management and aims to complement the genetic deficiency that is its origin, with the aim of halting or slowing its progression.
Currently, there are 4 to 6 approved treatments (between Europe and the USA). This approach has transformative potential, changing the course of the disease and reducing the burden and long-term costs associated with it. For patients and their families, the potential benefit of gene therapy goes beyond clinical effect, including reduced medical costs, improved quality of life, reduced burden on caregivers, reduced associated suffering and potential reintegration into work and society.
Families face significant financial, logistical, and personal burdens related to ongoing patient care, including medical devices and equipment, medications, tests, health care appointments, and hospital stays. This burden is exacerbated by necessary expenses at home, to improve accessibility, and for transportation in adapted vehicles for people with limited mobility.
The wide range of gene therapies in the pipeline could pose an important financial and sustainability challenge for the near future of healthcare systems, considering their price of entry. In addition, at an early stage of bringing a treatment to market, there is uncertainty associated with health outcomes and how long the effects of these treatments will last throughout the patient’s life.
For the health system, these are new treatments that require specialization of technical and professional resources, as well as adequate coordination between institutions providing health care. There are technical requirements and a level of preparation for healthcare professionals that should be expected.
It is also necessary to develop a digital infrastructure for patient monitoring and data management. Political authorities must anticipate these challenges so that Portuguese patients can access gene therapy in a timely manner.
In fact, there are many challenges in this area, and there is still much that can be done. Since these challenges go beyond the remit of individual water service and network entities, the formulation of an action plan for gene therapies is best articulated at the central government level.
It is desirable to create an organizational and institutional context conducive to change in the therapeutic paradigm already underway, a change that must be anticipated and prepared for, but cannot be stopped.
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