Frequently asked questions on gene therapy
While gene therapy has long been a topic of discussion within the hemophilia community, the idea of gene therapy as a treatment option is becoming a reality. There are many questions that remain: what are the eligibility criteria to qualify for this therapy, will it be affordable and accessible to patients, what is the long-term durability and efficacy and is it safe?
In addition to gene therapy, we also have other non-factor therapy options on the horizon, with one such therapy being released in 2018. We are entering an era of innovation and abundance in treatment options for those affected by bleeding disorders. At NHF we believe it is our responsibility to serve as a trusted resource of accurate, non-biased educational content that explains the science, the promise, and the potential cautions surrounding these new technologies. Our goal is to provide the bleeding disorders community with information and resources that will enable patients to have access to the data that will assist them in determining, alongside their provider, which option is right for them. NHF’s educational initiative offers a multi-pronged approach to ensure accurate information, tools and resources are available to consumers, payers, policymakers, and providers.
There is an abundance of information on gene therapy and the Rare Disease community is drawing closer together. The partnerships, collaboration and support of one another around patient education is unprecedented. There is great celebration around the innovation that holds the promise of better treatments for some and first treatments for many. Collectively we are becoming an even more powerful voice in the advocacy and policy arena.
The necessity of the work being done to educate payers on the value of innovation to our community is of utmost importance. These therapies may provide improvements in the quality of life for patients and their caregivers. We must do the foundational work necessary to successfully advocate for access to all FDA approved therapies.
Finally, we must join together and be diligent in our efforts to ensure the ongoing safety of products developed to treat bleeding disorders. We remain mission focused in our dedication to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research.