Here's how you can advocate for patients with rare diseases effectively.

One thing I have found very useful while advocating for cancer patients is Empathy, this is the best way to help them and encourage them for betterment. I have learnt that not always medicine cures the patient Sometimes it's just your love and care which they long for , an ear to listen and a heart to understand their pain.

This topic is near & dear to my heart as my daughter was born w/ a rare congenital heart defect & had her first open heart surgery just after birth. It’s important for industry professionals to have a deep understanding of the challenges patients face. Collaborate by working closely w/ patient advocacy groups to gain insight. Get active! Join patient-focused drug development initiatives. A lot of regulatory agencies require companies to engage w/ PTs in the drug development process. Advocate for improved access & support initiatives to reduce barriers to coverage. Create awareness campaigns, speak at conferences,share patient stories. Lastly, engage w/ policymakers at the local, state & national levels to help shape policies.

Educating yourself as a medical profession and an advocate only makes your own journey stronger. It is refreshing when a medical profession has heard about a rare diseases and even more so when they understand how it impacts treatment. If you don't know, be willing to learn.

This is incredibly important. Our knowledge base is forever growing and developing. It's impossible for a Healthcare professional to know and understand everything. That's why it's so important to advocate, especially for those that can not advocate for themselves.

Very true, advancement in knowledge is the key to success in any task. Not all things can be known by a single person thereby sharing information is a very important prospect of medical sciences this is why we need documentation . Documentation reaches places where humans can't reach and guide them for the best available options.

If you happen to work with the elderly, don't just do this yourself, encourage them to do this as well. Often, residents or patients feel as though they no longer have autonomy or purpose. Those are the very people that the politicians need to be hearing from!

In the short term, policy impact priorities focus on immediate health needs like accelerating drug development, promoting product approvals, or reducing out-of-pocket expenses. Long-term goals include building infrastructure to support rare disease patients. Advocates should choose negotiation methods that align with local culture and context, ensuring practicality and acceptance among stakeholders. What works in one country might not be feasible in another. Uniting stakeholders from diverse backgrounds—patient groups, clinical specialists, professional organisations, and health policy researchers—is crucial. This coalition can transform the advocacy agenda to reflect broader concerns.

Nurses have a critical role in increasing awareness about rare diseases and also there are some useful ways like Host fundraising events, Organize educational events, Set a world record, Be consistent in education, Design and distribute swag, Boost social media awareness, Produce content others find valuable. Make informational pamphlets to raise awareness of rare diseases.