Patient Advocacy Strategies

09/30/2025

“No child should die or suffer disabilities if a simple bloodspot can prevent it.” - Dr. Guthrie

If it weren't for Dr. Guthrie's persistence in developing a way to test babies for PKU in order to treat them as early as possible, we would not have as it is today.

We are grateful for all of the stakeholders who make up the Newborn Screening system - scientists, clinicians, public health programs, geneticists, specialists, and drug developers.

And we are extra grateful for all the family advocates who work toward the change needed to save lives.

09/29/2025

No matter how the diagnosis occurs, the child will still have the disease, and the family will adjust to the new reality. The difference is that a diagnosis through Newborn Screening gives options that don’t always exist after a diagnostic odyssey. Newborn Screening brings diagnosis with hope. - Lesa Brackbill, Rare Mom and Advocate

As Awareness Month continues, our Director of Advocacy highlights the power of newborn screening for . It provides families with early diagnosis, hope, and a range of options.

This is a great example of how industry and advocacy can partner to make a real impact. PAS is here to help with your NBS strategy.

09/25/2025

"There are so many problems to solve in rare disease, but it all starts with listening to the patients; letting them know their story, their pain, their loss matters.”

PAS’s Director of Advocacy, Lesa Brackbill, understands the pain and perspective of the rare disease community, having lost her daughter, Tori, to Krabbe disease.

In the ten years since, Lesa has become a fierce advocate for newborn screening and speaking on behalf of rare disease families.

In her role at PAS, Lesa helps rare patients tell their stories so those within the rare, medical, and life sciences communities are aware of and can take action to fight back against rare illnesses.

09/22/2025

When it comes to rare, childhood conditions, early diagnosis is critical. At Patient Advocacy Strategies, we champion policies that make it happen, like . Our advocacy team, led by a rare mom and newborn screening expert, is dedicated to ensuring patients have a voice and access to the treatments they need.

Learn how we can help your company or advocacy group - link in bio.

09/19/2025

September is Newborn Screening Awareness Month!

Newborn screenings, which include blood spot tests, heart screenings, and hearing screenings, are crucial for a baby's health. The goal of these tests is to identify serious but treatable health conditions early, often before any symptoms appear.

Early detection allows for immediate intervention, which can prevent death, disability, and long-term health problems.

Each state’s Newborn Screening system operates differently and screens for different numbers of conditions.

Our team at Patient Advocacy Strategies can provide expertise to guide you through the complexity of the system and help ensure that every baby is screened for as many conditions as possible.

09/17/2025

"The work we do creates hope for patients, and that hope is paramount to the success of biotech companies."

Mike Walsh, PAS founder and CEO, shares how giving patients a voice creates hope and can be a catalyst for change.

Contact us: https://patientadvocacystrategies.com/contact/?utm_source=linkedin&utm_medium=link&utm_id=storytelling

09/17/2025

Gene therapy is saving lives but most of the patients cannot afford the price of $2 to $3 million dollars. These gene therapies shouldn’t just be for the rich or a luxury. It is time for insurance company models to change so everyone can get these lifesaving gene therapies if they require them.



Read on here

It can cure deadly diseases, save long-term healthcare costs and transform lives. But the U.S. insurance system still isn’t ready to pay for it.

09/15/2025

Diversity is critical in cancer clinical trials, however reaching diverse populations for these trials can be a challenge.

New technologies such as digital monitoring, telehealth, and home-based testing are making trials more accessible.

Representation is the first step towards equitable cancer care.

Read on here

Kimberly (Cary) Demirhan discusses the need for more diversity and inclusivity in clinical trials for patients with cancer.

09/09/2025

Significant changes in medical education: According to a recent Do No Harm report, three major healthcare accreditors still enforce DEI regulations, while seven have pulled down or ended them. Is healthcare training returning to merit-based standards?

Learn more here:

Today, Do No Harm published a new report titled “Are Medical And Healthcare Education Accreditors Abandoning DEI?” The report, a follow-up to Do No Harm’s previous report exposing accreditors for injecting identity politics into medical and healthcare education, found that the accreditation la...

07/30/2025

Patient-centricity is the way of the future for clinical trials, not just a trendy term. This Technology Networks piece analyzes how digital biomarkers are gathered via smartphones, wearables, and remote monitoring devices which are helping more individualized, easily accessible, and effective clinical research.

Learn more on how technology is speeding up therapeutic advancements and changing patient participation here.

Explore how digital biomarkers are transforming clinical trials with real-time data, continuous monitoring and AI-driven patient insights.

07/22/2025

Trials are becoming more inclusive, successful, and easier by incorporating wearable technology and remote participation:

Wearables provide continuous health monitoring, ending the need for frequent clinic visits. They also:

-Improve trial access for participants who live in remote areas or have limited mobility.

- Offer real time data collecting, which increases patient safety and accuracy

-Help researchers recruit, keep, and use richer datasets more quickly.

Oncology and other therapeutic fields are witnessing a transition toward genuinely patient-focused designs as AI supports these technologies. It's a significant step toward clinical research that is more effective, accessible, and equitable.

Read more here

Experts discuss the use of artificial intelligence (AI) and patient-centric strategies in oncology clinical trials.

07/17/2025

We’re thrilled to shine a spotlight on Ardelyx’s bold initiative to put patient voices first. Notable advancements have already been made since Dr. Laura Williams became Chief Patient Officer:

Ardelyx went above and beyond the usual trial recruitment process by engaging with both urban and rural sites, partnering with community organizations, and focusing on the people most affected by these conditions.

In their dialysis trials, 45% of patients were Black and 28% were Hispanic, indicating a level of inclusion in clinical research that is rarely observed.

Their patient-centered approach goes beyond hiring. Patients also have input on research design, objectives, dosage, pill size, and even regulatory submissions. Their narratives are incorporated into the statistics, not only numerical values.

This method guarantees that the medications created accurately represent and benefit the varied lifestyles of those who require them the most, in addition to making trials better.

Ardelyx’s newly minted chief patient officer is infusing patient experiences into every element of the business.