Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.
The difficulty of balancing paying for innovation with budgets has plagued Europe for years, and the emergence of cell and gene therapies has only exacerbated that conundrum.
Regenerative medicine could be a game-changing approach to meet the needs of insulin-dependent patients. Although the field is still in its infancy, the initial clinical data looks promising. This article takes a closer look at a few innovative companies that are racing to bring the first regenerative medicine for diabetes to the market.
Over the past 20 years, hope has grown around the use of stem cells to enhance regeneration following a heart attack, wherein stem cells would facilitate tissue repair and help restore heart muscle function. However, the legitimacy of cardiac stem cell research is now being called into question.
While recent scientific breakthroughs for regenerative cell therapies are bringing great hope to patients and providers in desperate need of treatments and cures, how do you determine payment for something of enormously high value that has literally never existed before?
Progress in development of gene and cell therapies around the world has potential to transform standards of care for a range of diseases and address significant areas of unmet need in healthcare over the coming years. In the U.S. alone, almost 20 gene and cell therapy products have been approved thus far,1 with many other development programs reaching later clinical stages. The technology platforms of many of these drugs also offer the potential for curative efficacy and expansion for use in multiple indications.
The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.
A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations
According to a review of recent research studies that measured U.S. public opinion related to gene medicine, the public is not yet aware of gene medicine, is unfamiliar with the terms being used to describe the topic, and, when faced with multiple therapeutic options, is worried about making informed decisions.
Current reimbursement models in general do not accommodate many of the unique factors that are common among gene and cell therapies.
