Rare Disease Resources Every Pharma and Biotech Company Should Know
The realities of life with a rare disease can be startling. A typical journey for a patient living with rare disease begins with an average of six years seeking an official diagnosis. For some patients, there are available treatment options; for many others, there are not. Even with approved, available treatments, many patients face incredible financial challenges that inhibit access to therapy.
The Rare Disease landscape is only growing. There are now more than 6,000 identified rare diseases, with over 400 million people affected worldwide.
Creating access to medicines for people who have rare or life-altering diseases is why many of us chose to work in the pharmaceutical business. Whether focusing on distribution, commercialization, patient services operations, non-profit advocacy, or even as a health care provider, we all must keep the patient’s needs front and center in our decision-making.
That sounds easy but can be very challenging in practice.
We often advise our clients that there are many varied ways to better understand and engage with rare disease patients. One of the simplest is to take advantage of the educational and informational opportunities made available by the following outstanding resources.
Global Genes - connects, empowers, and inspires the rare disease community through education, advocacy, and legislative advocacy.
RareX - a global data-sharing platform that enables rare patient communities to more easily gather, structure, and securely share critical data
EveryLife Foundation– empowers the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.
NORD – a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services
The Disorder Channel – a compilation of rare disease films offered free on Roku and Amazon Fire TV. It’s virtually impossible to watch these stories and walk away without a heart full of empathy and compassion for all a rare disease patient or caregiver struggles with – and overcomes - during the journey.
On Rare Disease Day – and every day – our hope is that our industry continues our collective pursuit of improving the lives of patients living with rare diseases using these resources and other methods. Contact us today if we can assist your team in better understanding and ensuring access and support for rare disease patients.
Archbow Consulting helps pharmaceutical and biotech companies in the USA and Europe design, build, and optimize product distribution and patient access strategies. Archbow was founded by industry veterans to meet a need in the marketplace for consulting options that offer diverse real-world experience, are able to leverage deep connections across the industry, and can also provide actionable strategic guidance. We invite you to learn more about our team, services, and clients’ success, and connect with us via email, LinkedIn, Twitter or subscribing to this blog which you can do below.