In the leukodystrophy space, we are seeing an increased amount of collaboration in Newborn Screening advocacy and we will continue to do our part to encourage collaboration because we believe it is essential.
At The Global Leukodystrophy Initiative meeting in October 2022, Dr. Florian Eichler spoke to the advocacy groups about this very topic in regard to treatment development and access:
“With progress comes disparity and disconnect, and without building community, there is no therapy.”Dr. Florian Eichler
This statement applies to Newborn Screening, too. “Without building community, there is no Newborn Screening.”
It takes researchers, clinicians, advocates, and industry working together to make treatments (and access to them through Newborn Screening) a reality.
Advocates are a vital part of this and can:
- Assist in raising awareness
- Bring stakeholders together
- Enable early diagnosis through Newborn Screening and assist with rapid recruitment to clinical trials
- Manage expectations and sustain efforts
- Example: Clinicaltrials.gov doesn’t explain why a trial was paused, leaving room for assumptions and fear. Advocates can provide detail and reassure stakeholders/community, and by doing that they can sustain hope.
In addition, advocates are able to:
- Highlight unmet clinical needs – such as lack of treatment – better than anyone because desperation drives action.
- Speak to decision-makers plainly about the risk vs. benefit of treatment and what that means to them (for example, 20% risk of death in treatment, 100% chance without treatment). Who should be defining success but the parents with terminally ill children?
- Be free from restrictions that paid lobbyists and state employees face and can be bold, persistent, and powerful because of their stories and experiences.
- Inform scientists about the unmet needs of patients and/or what needs to be understood about the disease.
Why Collaboration is Essential:
“It’s hard to work together but it’s harder to have a child die from a progressive disease.”Terri Klein, The National MPS Society
There are more than 7,000 rare diseases that have been classified, and more than fifty types of leukodystrophy. Advocacy groups are often small and carry a large burden of work to be done. They assist in seeing treatments and Newborn Screening tests developed (and adopted by states). In addition, these groups also encourage their community and help to share the patient voices.
Why not learn from groups that have gone before you? Learn about their failures, successes, lessons learned. There are so many areas where we can all work together and it’s time to do so. Because of limited resources, let’s share the load!
Collaboration in Newborn Screening advocacy is essential and we want to help make it happen.
To learn more about how we can help you advocate, click here.
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