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By Julie Kim, president, U.S. Business Unit, and Cheryl Schwartz, senior vice president, U.S. Rare Disease Business Unit at Takeda
Think back to the last time you had the flu. Most likely, you were diagnosed within a few hours. You took some medicine, got some rest and pretty soon, the flu was in the rearview mirror. Widening the lens to more complex diseases like cancer, time to diagnosis might be a few months depending on a number of factors including severity of symptoms, specialist referrals and testing.
But what if you had a disease that your doctor had not encountered before? What if you went to doctor after doctor, took tens or hundreds of tests only to have each turn up inconclusive? What if you spent hours scouring the internet, looking for any sign that someone else has experienced what you’re going through? And what if you received a diagnosis and treatment, but your symptoms didn’t resolve? You begin the cycle again.
This is what the often long and frustrating path to diagnosis looks like for the approximately 30 million people in the U.S. living with a rare disease, which according to the National Organization for Rare Disorders is categorized as any disease affecting fewer than 200,000 in the United States.
Often challenging to diagnose, it takes an average of six years from symptom onset to an accurate diagnosis. The reasons range from symptoms mimicking those of more common conditions to incomplete knowledge of rare diseases to lengthy delays to access specialists. Six years to diagnosis is a best-case-scenario — typically for those who are educated, economically advantaged, with regular access to health care and more often than not, white.
But what happens when you begin to factor in the social determinants of health that compound a person’s ability to achieve equitable access to any kind of health care in the U.S., let alone for a disease that affects only a few thousand people? Think of it like walking through a maze with no map.
Imagine a person from a disadvantaged or underserved community entering the maze at symptom onset to seek medical help but…
They live in a health care desert or rural area where the closest clinic or hospital is far from their home and transportation is a challenge.
It’s an obstacle.
Once an appointment can be scheduled, gender or racial biases may contribute to their symptoms being trivialized or dismissed.
This cycle could repeat multiple times over months or years as symptoms persist or progress.
If they do happen to get a referral to a specialist, they may not be able to take the necessary time off for appointments, tests or treatments. Some could lose their job, which could mean loss of vital health insurance.
All barriers to care.
And maybe because of language or cultural barriers, or a lack of health literacy, they do not feel comfortable asking questions of their doctor or offering information that might help inform a diagnosis or treatment plan.
Every patient’s journey is different, but the sad truth is that these—and many other—health inequities can delay an accurate diagnosis for years and prevent many individuals from having a fair and just opportunity to be as healthy as possible. Without addressing social determinants of health, disadvantaged and underserved communities will continue to suffer, and it could be years before they make it out of the maze, if ever.
That’s why Takeda remains committed to the rare disease community. We are focused on reducing the burdens that people living with a rare disease can face on the path to diagnosis, including ways to help those who already face serious health inequities.
We are proud of our many efforts to make tangible changes to access, education and science.
We partnered with Children’s National Hospital to create the Rare Disease Clinical Activity Protocol (Rare-CAP) program, a first-of-its kind system to develop, disseminate and curate clinical trial protocols to help standardize processes around diagnosis and care. The platform hopes to break down barriers for researchers, clinicians and patients by providing uniform standards of diagnosis and care, where there are currently none.
We are also focused on addressing some of the access challenges in the system while advancing value-based care in the U.S. We have initiated partnerships and value-based agreements, over 12 to date, including some that have an emphasis on the total cost of care or shared risk beyond drug costs. Two of them are focused on rare disease—hemophilia and hereditary angioedema (HAE). We are using data and insights generated from these agreements to improve treatment access and affordability for patients.
Access is only part of the equation if a patient—or their loved ones—struggles to understand a diagnosis, ask questions or explain it to others. That is why Takeda has undertaken a number of initiatives to create resources that meet patients where they are.
We created storybooks that can help explain a child’s MPS II (or Hunter syndrome) to a sibling and non-clinical explainers for schools to better understand the disease and the needs of that child.
Recognizing that significant portions of the population do not speak English as a first language, our bilingual community educators bridge an important gap. They connect with patients in the language they are most comfortable. For example, we engaged patients and their families exclusively in Spanish at Familia de Sangre, the largest Spanish language meeting in the bleeding disorders community.
Finally, and most important to the rare community, innovative science and research must continue to look deeper into the impact that these rare and ultra-rare diseases have on patients. At Takeda, nearly 50% of our pipeline has Orphan Drug Designation, which means that if approved, they could help target some of the rarest diseases affecting patients today.
Takeda is also actively contributing to the larger industry goal of greater equity in clinical trial recruitment and has created a roadmap to focus and inform our efforts in clinical trial recruitment by partnering with clinical study sites to address operational barriers, verify whether goals reflect real-world data and enhance the diversity of the investigative site staff.
We are proud of our efforts to positively impact the path to diagnosis for the rare community and more broadly, we are equally committed to addressing health inequities that we believe could have an impact on all patients.
We have a long journey ahead to realize our vision of health equity in the rare disease community and beyond, but it’s a worthwhile journey and it’s one we are deeply committed to in order to achieve our mission of better health and a brighter future for all.
This content was written by the advertiser and edited by Studio/B to uphold The Boston Globe’s content standards. The news and editorial departments of The Boston Globe had no role in its writing, production, or display.
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