Did you know that approximately 300 million people worldwide are living with one of the 6,000+ rare diseases, most of which have no or limited approved treatment options?
On this Rare Disease Day, February 28, Janssen stands in solidarity with the patients, families, and caregivers whose lives are turned upside down emotionally, physically and financially by rare diseases.
As Chief Global Value and Access Officer at Janssen, it is my personal mission to facilitate access to life-changing, innovative treatments for unmet medical needs. Because rare diseases are usually chronic and progressive and because science is advancing rapidly, we are racing the clock to secure medicine access for those living with such medical issues.
Speed demands local collaboration. I believe delivering transformational medical innovation for individuals living with rare diseases requires the different local health system players to work even more closely together to create the types of remarkable outcomes patients expect and deserve.
I believe delivering transformational medical innovation for individuals living with rare diseases requires the different local health system players to work even more closely together to create the types of remarkable outcomes patients expect and deserve.”
In local collaboration for patient benefit, I like to refer to various “E’s” to consider in achieving rare disease patient access:
Evidence (Endpoints and Extrapolation)
As we know there is no ideal measure of value across all diseases or geographies. Emerging new therapeutics in rare diseases often present unique challenges to agreeing on evidence of treatment success.
Ultimately, gaining the perspective of individuals living with a rare disease at the beginning, middle and end of development is critical to defining rare disease patient-relevant endpoints.
It is necessary to select endpoints that are explicit enough to facilitate decision making. Extrapolation of results to predict the real value of a medicine to patients, caregivers, and society is necessary and needs to be an accepted approach. The uncertainties inherent in extrapolations and debates about these cannot lead to delays in patient access.
Economic Consequences
Without innovative treatments and medicines many rare disease patients require frequent hospitalizations, costly medical and lifestyle interventions, and ongoing care. Treatments and medicines therefore benefit those living with the condition, as well as families, friends, and caregivers -- eventually employers, societies and economies.
Education and Early Engagement
When government agencies and payers around the world assess the value of a rare disease treatment, “One-size Fits All” measures fail to recognize unique patient circumstances. Education for clinicians, regulators, value assessors and payers from the perspective of people living with rare disease is paramount.
When government agencies and payers around the world assess the value of a rare disease treatment, One-size fits all measures fail to recognize unique patient circumstances.”
Janssen sets out for early engagement with all these stakeholders. This means sharing information with patients, clinicians, policymakers, and payers -- as early as local law allows -- to define the many E’s of medicine access for those living with rare disease.
