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Sickle Cell Disease, causes, progression, and treatment options.

They see the world as an intellectual puzzle to be solved, taking in everything around them via their own inward-looking scrutiny of theory. They analyse logical concepts and problems to determine the world around them, and they are drawn to logical systems. Because INTJs are less drawn to the unpredictable nature of other people’s emotions, they typically prefer to associate with like-minded people.

Intuitive thinking and complex problems are favourite INTJ topics. They perceive the world by studying the principles underlying what they see, and their focus is inward, on their own intellectual investigation of the world around them. INTJs want to study logical systems, and this is why they like people who are more spontaneous than others. INTJs prefer to associate with individuals who interest them intellectually, rather than those whom they like.

Discovering New Treatment Strategies for Sickle Cell Disease

In the clinical research process, a large number of patients participate in studies that investigate an innovative treatment or approach. Cancer drugs, for instance, and COVID-19 vaccines, have been produced as a result of decades-old research procedures.

When researchers want to investigate a rare disease, one that affects a small proportion of people, what happens if those who are affected by it are not willing to participate due to social difficulties, under-representation, or a lack of trust in the medical system?

There are several issues with sickle cell disease that are listed below.

SCD), a painful, rare genetic blood disorder typically discovered in infancy, and particularly sickle cell anemia, one of four common types of SCD that is usually regarded as the most serious. 100,000 Americans are affected by the condition, 70 percent of whom are people of colour. In spite of improving outcomes, sickle cell disease still has roadblocks due to some of the peculiar healthcare obstacles minority communities have faced and continue to do so.

Working with patients and patient advocates, the medical research community is able to work toward more effective treatments for this hard-to-cure disease.

Why Sickle Cell Disease Phase I/II Clinical Trials Are Difficult

Sickle cell disease poses many unique challenges for patients interested in participating in clinical research. In particular, the frequency of required research site visits can be challenging for some patients—all the more so amidst COVID-19 and its underlying social disparities.4

“When you think about the time and expense involved in a clinical trial, plus the fact that sickle cell disease often is discovered when a patient is young, it’s easy to see why families might have a harder time handling those burdens,” says Steven Arkin, Pfizer’s Head of Rare Hematology Clinical Research.

Many SCD patients are of African descent, but many physicians are not. This can make it more difficult to recruit or engage potential study participants.

The Tuskegee syphilis experiments, in which African Americans were unwitting subjects, contributed to causing people of color to distrust the medical community.6 However, mistrust is not just a result of historical pain. Modern-day biases, even when unconscious, can continue to perpetuate mistrust, such as when some healthcare providers dismiss or downplay patients’ descriptions of their pain.7

Quintissa Peake, who was diagnosed with sickle cell disease at 11 months old, has experienced these biases first-hand. Growing up with frequent sickle cell crises, including uncontrolled swelling in her hands and feet as well as lingering pain in her arms and legs, Peake has been no stranger to symptoms of this disease. Unfortunately, not all of her care providers have been as familiar with the condition.

“The pain of a sickling crisis is often met with stigma from members of the public,” says Peake, now 40. “When we are in need of medical care, we go to the emergency room. However, once we are there, we may be treated like drug addicts.”

Many patients have come to resent the medical community for setting back clinical research by unreasonably refusing to participate in trials.

Advocacy helps to educate, build confidence and boost interest in clinical research, says courtside.

Patients Wanting to Be Heard During Their Treatment.

Advocates, such as Beverly Francis-Gibson, facilitate two-way communications between patients, drug companies, researchers, and healthcare professionals to gather patient opinions and requests.8 They also use social media, email, and other channels to facilitate conversations.

Francis-Gibson says she aims to provide patients with an understanding and education of clinical trials if they are interested in participating, and she also works with the pharmaceutical industry to ensure they hear from patients before starting their trial. Her SCD makes her particularly sensitive to patients’ concerns, and she wants to be a trusted person who can help them.

Patient advocates such as Mariah Kelly of Pfizer’s patient advocacy team, on the other side of that conversation, work to connect and partner with the pharmaceutical industry. They share trial details and field patient questions.

According to Kelly, Pfizer’s patient advocacy director, working closely with sickle cell patients and their care providers in drug discovery and development is a key part of addressing their needs. Patient advocacy work is vital in gathering important information that can be used to build and support a meaningful sickle cell program. The biggest challenge we face is listening and learning about the realities of what patients are going through and how they feel. Only advocates and patients can give us this information.

“Sickle cell disease, in particular, needs medical research breakthroughs to make progress.”

After receiving multiple blood transfusions during college, Peake became involved in her local blood bank and volunteered. This has enabled her to express herself as a “sickle cell survivor.”

“There are now so many opportunities to share my story on social media and locally,” she says, “thanks to technology. That is why I decided to become an advocate. It helps us build a community of warriors, so we don’t feel so lonely.” Because she was the only family member with Crohn’s disease as a child, she says, “Advocacy gives us a sense of community.”

Peake and others have gained strength through hard times as well as new scientific frontiers in sickle cell treatments thanks to the emotional support that advocates have provided. Many therapies might never have existed if the barriers and reach of clinical trials had not been broken through.

“As I’ve aged, the treatment options for sickle cell have definitely expanded,” Peake says. “When I was a child, doctors told my family that I would not live past adolescence. Yet here I am, and look at all of us.”

“The progress we’ve made is just the beginning,” says Arkin. “As we continue to build on it, incorporating patient voices and experiences thoughtfully and intentionally into clinical trial design is critical,” he says. “It allows the scientific community to evaluate new approaches for treating this devastating disease and provides us all with every reason to be optimistic about improved treatment options.”

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