Afleveringen
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From a career devoted to finding better ways to connect patient volunteers to clinical trials, Lisa LaLuna knows why the public needed an online search tool that is easier to use than the traditional methods of access.
âMembers of the public often want to find a trial to access the latest medicine, or help a family member, or just to participate as a healthy volunteer to further medical research. Pharmaceutical companies that run clinical trials of course want to enroll patients so their research can be successful. In spite of this mutual desire to work together, patients and trial organizers often cannot find each other,â Lisa explains.
Some trials are stopped because they cannot enroll enough people and others are delayed because enrollment takes too long. In this unfortunate scenario â one that happens too often â this slows down the development of treatments and cures that patients depend on.
Lisa explains the importance of this search tool in providing a simple, fast way to discover trials specific to a disease, a location, and the personal characteristics of volunteers being sought.
âYou can even opt in to be notified when more trials are posted, and send a message to the local trial site to request contact to learn more and possibly be screened for qualification purposes. â Lisa explains.
This free website can be found by anyone at centerwatch.com/clinical-trials.
Visitors to the site will find basic information about what a clinical trial is and why someone would want to participate.
Patient Advocacy groups are providing this website to their members to educate them about the value of clinical trials and where to find them, in order to support the search for cures. Patient groups like the Michael J. Fox Foundation for Parkinsonâs Research, the Alzheimerâs Association, and the rare disease group The Rett Syndrome Foundation are enthusiastic about how their use of this tool is helping them help their communities of patients and their families.
âClinical trial search tools have been too complicated in the past and we sought to create a tool that is simple and fast so anyone can use it,â Lisa explains. âSeeing the adoption by Patient Advocacy Groups is most satisfying.â
Lisa LaLuna of WCG Patient Advocacy has thirty years working in clinical trials and is a passionate advocate for easing the search for trials for those wishing to volunteer. -
Bernadette Siddiqi, associate director of research partnerships at The Michael J. Fox Foundation for Parkinsonâs Research talks about how they give members of the public access to the Fox Trial Finder. This clinical trial search tool that allows anyone to search for Parkinsonâs disease clinical trials. More than 300 are listed on the site.
Itâs a bottleneck in clinical research. Patients are eager for a cure, but it can be difficult for them to connect to trials and for researchers to connect to volunteers.
Bernadette speaks of why it is critical to the foundationâs mission to provide an easy way to make this connection. Just as important, she says, is educating people about research. Fox Trial Finder provides both. Roughly 700 people visit the site each month.
The Fox Trial Finder is powered by CenterWatch iConnect, and The Michael J. Fox Foundation for Parkinsonâs Research is part of growing list of patient advocacy groups that deploy this search tool to inform and empower their patient community.
The Michael J. Fox Foundation for Parkinsonâs Research is dedicated to finding a cure for Parkinsonâs disease through an aggressively funded research agenda. -
Zijn er afleveringen die ontbreken?
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After more than 25 years working to plan and conduct clinical trials at pharmaceutical companies, Ros Cheetham decided to put herself into the patient role: She volunteered to participate in a COVID vaccine clinical trial.
She discusses why she decided to participate in the trial and shares her experiences. So far, itâs all been positive: She speaks of modern methods, safety and the well-organized approach and thoughtful communications used by the clinical research site.
She also talks about the barriers, such as distance, that prevent some people from participating in clinical trials, and calls on the industry to help potential participants to overcome those barriers. Studies must be as inclusive as possible so clinical research can benefit a more representative cross-section of our society.
She also exhorts individuals to, like her, become clinical trial volunteers. Trials need volunteers, and everyone should have the experience of being in a clinical trial.
Cheetham is a pharmaceutical development professional providing expertise in global drug development, global clinical operations solutions and partnership and alliance strategy. Her broad industry experience encompasses several vice president positions. She has led product development teams through successful filings and commercialization of new drugs as well as having implemented innovative global operational solutions that provided cost savings with timely execution and high quality. -
Movement disorder trials have always been demanding. Now, sponsors face an additional challenge: How do you ensure data integrity in the era of remote clinical trials? In this podcast, Bob Dagher, MD, WCG MedAvante-ProPhaseâs Chief Medical Officer, addresses this in conversation with Steve Smith, WCG President of Patient Advocacy. This is the fourth episode in our series, âTransforming CNS Trials During COVID-19âand Beyond.â
Sponsors planning to restart movement disorder programs need to be clear-eyed and realistic, Dagher counsels. âWhen it comes to remote assessments, we have to be vigilant and aware of what we can do and also about what we really cannot do.â Some assessments simply cannot be performed remotely. This means sponsors will need to figure out how to account for variability and missing data in their statistical plan before locking the database.
FDA guidance on the conduct of trials during COVID-19 helps provide a roadmap. For example, in the case of anticipated missing data the guidance clearly states to capture specific information in the case report form that explains the relationship to COVID-19.
But it also requires in-depth scientific and clinically-informed knowledge so that the quality of the data captured isnât sacrificed as direct result. Accordingly, WCG has been working with clients to tailor remote assessments for sponsorsâ trials, yielding assessments that are as close as possible to the in-person versions (minus those particular items that cannot be done remotely).
Dagherâs message? âEnsuring data integrity at every step of the process should be the primary guide for any decision-making process when deciding to restart your study.â
Prior to joining WCG MA-PP, Dr. Dagher, was the Chief Medical Officer of Cadent Therapeutics in Cambridge, Mass. Before that, he served in many leadership roles at small and large biopharmaceutical and clinical research organizations, including GlaxoSmithKline, Genzyme, Sanofi and Covance, among others. Dr. Dagher has fostered the development of multiple products in different indications and development platforms and advanced several small molecules and biologics across all stages of clinical development. His background includes therapeutic experience in psychiatry, neurology and rare diseases. -
As a global strategist for Intelâs Health and Life Sciences Group, Bryce Olson had an up close view of innovation happening in the labâespecially in terms of genomic sequencing and gene therapy. As a man diagnosed with aggressive stage 4 prostate cancer at 44, he had as well an up close view of how that innovation was not being translated into the clinic.
He was started on a one-size-fits-all standard of care. It didnât work, and chemo made him ill. In this podcast episode, he talks about how he demandedâand receivedâDNA sequencing. That data led him to find the right trial and the right medicine for his type of prostate cancer.
Heâs now a very vocal advocate of sequencing. Among other endeavors, he founded Sequence Me (www.sequenceme.org), a patient-driven movement to accelerate access and adoption of new genomic sequencing innovations for cancer. -
In major depressive disorder, researchers have used remote assessments for decades. As a result, many of these studies have had an easier time adapting to the new clinical trial landscape. But that doesnât mean they are pandemic-proof.
So what are the critical success factors for clinical depression programsâespecially now?
In his conversation with WCG President of Patient Advocacy Steve Smith, Mark Opler, PhD, MPH, Chief Research Officer at WCG MedAvante-ProPhase offers his insights on the future of clinical depression trials. This is the third episode in our podcast series, âTransforming CNS Trials During COVID-19âand Beyond.â
Weâre headed in the right direction by enabling more patient-friendly evaluation, but â especially in light of the pandemic â we must continue to refine these systems and make sure that we have the input of patients, caregivers, and of course, investigators.
Among his recommendations: Ensure clinicians have the right depression-assessment tools and that they are using them correctly.
Itâs also critical, he says, to have a solid remote assessment methodology in place. This means not only incorporating the right tools, but also establishing a way of continuously protecting data quality, such as WCG's Study Insight Analytics Platform.
Mark Opler, PhD, MPH, is Chief Research Officer at WCG MedAvante-ProPhase. Dr. Opler was the founder of ProPhase and served as its CEO and Chief Scientific Officer, among other roles. He serves as adjunct assistant professor of psychiatry at New York University and assistant professor of clinical neuroscience at Columbia Universityâs College of Physicians and Surgeons. He is also leading the development of the forthcoming edition of the PANSS Manual. -
LaTasha Lee, PhD, MPHâs path to becoming a sickle cell disease (SCD) researcher and policy advocate began as a child. Because the SCD trait ran in her family, she became a patient advocate while still small. Today, she is Vice President, Social and Clinical Research & Development, at the National Minority Quality Forum. In this wide-ranging interview, she touches on an array of issues affecting the SCD community. Among them: adult patients who end up in the emergency room, the increased number of available therapeutics and the robust pipeline of SCD therapies.
Of particular concern is the challenge of accessing pain medication â both in the ER and in primary care practices. âIndividuals with this disease often arenât believed. They are considered drug seekers when they arenât.â
She also makes a passionate call for increased minority participation in healthcare and in research to enhance equity and improve outcomes.
Dr. Lee is responsible for the oversight and implementation of research projects and programs focused on reducing patient risk and identifying optimal care to reduce health disparities and bring about health equity at the National Minority Quality Forum. Before that, she served as Senior Manager of Partnership Engagement of the Sickle Cell Disease Clinical Trials Network (SCD CTN) at the American Society of Hematology (ASH) Research Collaborative. She was responsible for establishing, implementing and maintaining effective partnerships with stakeholders including patients, academic medical centers, other clinical research sites, industry, and federal agencies regarding their interests in SCD CTN. As an experienced research manager at ASH she was tasked with the development of a patient engagement strategy and clinical trials network for SCD. While at ASH, Dr. Lee was also responsible for the day-to-day management and implementation of projects, programs and activities related to ASHâs Call to Action on Sickle Cell Disease. -
At 51, Jeffrey Borghoff was diagnosed with Alzheimerâs disease. What would devastate many people turned Borghoff into an advocate. The former software architect and developer is now a national spokesperson for the Alzheimer's Association and board member of the Greater New Jersey chapter.
In this podcast, he talks about how the diagnosis forced him to reprioritize his life. âI've been pretty successful at that. I've been leading a very purposeful, engaging, joyful life.â With his care partnerâhis wife, Kimberlyâhe travels around the country making the case for research fundingâand for clinical trial participation.
Thatâs critical because important Alzheimerâs trials focus on younger-onset AD patients. Unfortunately, many people who are beginning to show signs at a very young age want to deny they have the disease. They wonât go into trials or even get a diagnosis. His messages to them: This disease does not define who you are, and research is important. -
Remote assessments have become a necessity across CNS trials, but some areas are more complicated than others. Schizophrenia provides one such example; the risk of variability is high, and sponsors are concerned. In this podcast, Mark Opler, PhD, MPH, Chief Research Officer at WCG MedAvante-ProPhase, sits down with WCG President of Patient Advocacy Steve Smith to discuss the topic. This is the second episode in our podcast series, âTransforming CNS Trials During COVID-19âand Beyond.â
PANSSâthe gold standard rating scale for schizophrenia primary outcomesâis based largely on face-to-face interviews; it wasnât developed for remote assessments. Is it still usable? Yes, says Dr. Opler. âIâm pleased to report that the anecdotal experience, as well as available data, suggests assessments conducted with patients with schizophrenia via video can be done reliably and can be done in a way that produces valid data.â
It can be doneâthe tools exist. But itâs the way sponsors and CROs approach remote assessments that can make or break data integrityâand he delves into that topic in this podcast. In particular, he shares how sponsors and CROs can leverage remote assessments in their next clinical trial.
Mark Opler, PhD, MPH, is Chief Research Officer at WCG MedAvante-ProPhase. Dr. Opler was the founder of ProPhase and served as its CEO and Chief Scientific Officer, among other roles. He serves as adjunct assistant professor of psychiatry at New York University and assistant professor of clinical neuroscience at Columbia Universityâs College of Physicians and Surgeons. He is also leading the development of the forthcoming edition of the PANSS Manual. -
Lisa Deck was a healthy 21-year old a week away from college graduation when her first stroke put her in the hospital. She barely made it to her graduation ceremony. She suffered two more strokes before she turned 25, and after that, a fourth stroke.
After serious misdiagnosis and incorrect treatments, she was finally diagnosed with Moyamoya disease, a rare condition without a cure, but it does have a surgical treatment.
In this podcast, she shares her diagnostic odyssey, her commitment to advocacy and the value of peer and family support. She also talks about the importance of staying positive and why patients must advocate for themselves. -
How can CROs and sponsors detect potential problems and issues in clinical trial conduct and data quality? Thatâs one of the critical issues discussed in Steve Youngâs interview with Linda Sullivan, MBA, Executive Director of WCGâs Metric Champion Consortium (MCC). Young, Chief Scientific Officer at CluePoint, discusses how risk-based quality management (RBQM) methodology, data analytics, and data surveillance are being utilized today to go beyond just ferreting out fraud. Now, Young says, clinical trial stakeholders are deploying advanced statistical engines to detect quality and data breaches in investigational studies. According to Young, while there are still instances, to varying degrees, of major misconduct in clinical trials, CROs and sponsors are increasingly turning to online solutions to risk planning. Itâs important, he adds, that when risks are discovered, the solutions are integrated into the workflow of clinical trials. In fact, Young asserts, one of the biggest challenges facing the industry today is to embrace change and accept the use of centralized monitoring data to aid in the discovery and resolution of risk.
Interested in attending the MCC Clinical Trial Risk and Performance Management vSummit Sept. 8-10? Visit https://www.centerwatch.com/mcc-summit-2020.
If you subscribe to this podcast, you can use the code POD15 to receive 15% off your registration fee. -
For many of the same reasons underrepresented populations lack access to appropriate healthcare services, they are also left out of clinical trials. The problem is widely known, but solutions have been hard to come by. In this episode, Michael Poku, MD, MBA, biopharma medical director at Signify Health, explains why this is the case and then shares some approaches he and his colleagues have successfully used to remove the barriers to clinical trial participation.
An important aspect is understanding the larger pictureâthat inequality is due to in large measure to longstanding and systemic issues. He discusses how unmet social needs rarely occur in isolation. Addressing those needs together can lead to better representation in clinical trials.
âSponsors are incredibly excited about opportunities to make their trials more representative and to have underrepresented minorities participate,â he says.
In addition to his work at Signify Health, Dr. Poku is on clinical faculty at Methodist Dallas Medical Center and the University of Texas Southwestern Medical Center, where he cares for patients as an internist and teaches trainees and medical students. Prior to his role at Signify Health, Dr Poku was a consultant at McKinsey & Company. -
Dana Deighton, an esophageal cancer survivor, shares her diagnostic odyssey of false starts and life-threatening detours.
Deighton had always been healthyâshe didnât smoke, didnât drink and exercised every day. Then came a joint pain that wouldnât go away, visits to numerous doctors, a series of misdiagnoses, invasive tests, and growing symptoms. The correct diagnosis of stage 4 esophageal cancer âwas not the diagnosis anyone wants.â But she refused to accept physiciansâ recommendations for palliative care as a solution.
She describes her persistent advocacy to convince doctors to consider her as an individual different from expectations and generalizations. She asked the hard questions and had the hard discussions, resulting in an outcome better than expected.
She recommends physicians and patients do the homework, energetically collaborate and consider the individual patient in the quest for a solution. âDonât be a passive patient.â
Dana Deighton works for the online patient community provider Inspire.com that helps connect patients with others experiencing the same disease, for a wide variety of diseases and over 4 million members. She also serves as an Executive Board Member of the Esophageal Cancer Action Network (ECAN), is a patient representative on the Locally Advanced Esophageal Cancer Guideline Panel for the American Society of Clinical Oncology (ASCO), a member of NCI Patient Advocate Steering Committee and NCI Esophago-Gastric Task Force and serves on the Esophageal and Stomach Cancer Project Patient Advisory Committee, a project led by the Broad Institute of MIT and Harvard. She lives outside of Alexandria with her husband and three teenagers. -
Can patients be trained to report their symptoms more accurately? Thatâs one of the key topics discussed in Nat Katzâs and Art Moralesâ interview with Linda Sullivan, MBA, Executive Director of WCGâs Metric Champion Consortium (MCC). Katz, the Chief Science Officer of WCG-Analgesic Solutions and Morales, Vice President, Technology Solutions at WCG, assert that, with proper design and implementation, you can structure clinical trials, such as pain management studies, so that patients learn how to better report their symptoms. With proper training, patients learn to accurately report their symptoms, resulting in more reliable data, according to Katz and Morales. During COVID-19, they add, sponsors have had the opportunity to examine ways to introduce new technologies, such as telehealth, to clinical trial operations and to use more science-based tools for evaluating the performance of clinical trials. COVID-19 also has accelerated the scientific evaluation of clinical sites by optimizing the analysis of patient data. The result: Clearer and more reliable data collected during the conduct of study â without unblinding the data.
Interested in attending the MCC Clinical Trial Risk and Performance Management vSummit Sept. 8-10? Visit https://www.centerwatch.com/mcc-summit-2020. If you subscribe to this podcast, you can use the code POD15 to receive 15% off your registration fee.
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How are clinical trial companies pivoting to implement Risk-Based Quality Management programs during the pandemic? Thatâs one of the deep dives explored in Duncan Hallâs and Rachel Oakleyâs interview with Linda Sullivan, MBA, Executive Director of WCGâs Metric Champion Consortium (MCC). Hall, founder and CEO of Triumph Research Intelligence (TRI) and Oakley, Vice President of RBQM Services at TRI, note that, when the COVID-19 pandemic first hit, CROs and sites performed risk assessments on their existing studies. Then those organizations took advantage of innovative technology that had a key tool for optimizing clinical trials â centralized monitoring. According to Hall and Oakley, that tool enables critical oversight and monitoring of investigational sites, even when in-person visits are not feasible. They point out that regulatory agencies are issuing new guidance during the pandemic, and that sponsors, CROs, and sites need to figure out how to comply with both those new mandates as well as existing rules. According to Hall and Oakley, there has been a renewed sense of community in the industry in dealing with the pandemic as stakeholders try to stay current through a variety of trusted sources, including MCCâs weekly COVID calls. Finally, they note, one component for optimizing clinical trials is taking center stage â training, especially in the area of RBQM.
Interested in attending the MCC Clinical Trial Risk and Performance Management vSummit Sept. 8-10? Visit https://www.centerwatch.com/mcc-summit-2020.
If you subscribe to this podcast, you can use the code POD15 to receive 15% off your registration fee.
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Whatâs the secret to success for implementing a world-class holistic approach to a Risk-Based Quality Management (RBQM) system? Thatâs one of the key topics from Artem Andrianovâs and Johanna Proeveâs interview with Executive Director of WCGâs Metric Champion Consortium (MCC), Linda Sullivan, MBA. Andrianov, CEO, and Proeve, Chief Science Officer at Cyntegrity emphasize the importance of developing a clinical trial platform that enables your organization to manage the entire quality management cycle rather than continuing with traditional silo-based approaches. This comprehensive approach, they explain, enables pharmaceutical companies to become more efficient in reducing risk, which is especially important during the current pandemic. In addition, they note, in the COVID-19 era, more organizations have had to turn to holistic risk-based quality management systems because, for example, Excel doesnât always work for managing RBQM, especially in larger clinical trials. Today, both CROs and sponsors are implementing innovative RBQM technology and asking for additional training to deal with data quality deterioration and other issues brought about by the onslaught of the pandemic, according to Andrianov and Proeve. Listen to this episode to find out how COVID-19 has magnified festering clinical trial shortcomings and why itâs essential that pharmaceutical company leaders become âdata scientists.â
Interested in attending the MCC Clinical Trial Risk and Performance Management vSummit Sept. 8-10? Visit https://www.centerwatch.com/mcc-summit-2020.
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Ever since elementary school, Gregory L. Hall, MD, knew he would be a doctor. In this interview, he shares that journey and how he came to write the bookâliterallyâon health disparities for African Americans.
His focus on disparities began more than 30 years ago with his appointment to the Ohio Commission on Minority Healthâthe first such commission in the country. Eventually, when he became the chairman, he had to give quarterly reports. The research he conducted on disparities for those reports opened his eyes, he says.
âI naively believed that providers and physicians didnât contribute to health disparities. I thought it was just poverty and a lack of insurance. I didnât know that, actually, providers contribute to it, health systems contribute to it.â He was shocked, and what he learned led to him write his book, Patient-Centered Clinical Care for African Americans: A Concise, Evidence-Based Guide to Important Differences and Better Outcomes. It is for physicians and clinicians and is a guide outlining specific differences in communication, clinical therapies, medications, protocols and other critical approaches to the care of African Americans.
The book discusses a wide range of disorders that affect African Americans It includes clinical pearls, but it also provides the background clinicians need to understand just what drives disparities.
Providers donât want to exacerbate disparities, but they are often unaware. If you donât know what youâre doing wrong, you canât fix it, he says. He is helping with both.
Even when providers do a better job addressing the specific needs of African-American patients, hurdles remain, he says. For example, lack of trust remains a huge issue among African-American patients; the shadow of historic discrimination in medical settings looms large. Almost half of African Americans report low trust of healthcare providersâand whatâs interesting, he notes, is that this distrust extends to providers of all races. As a result, African Americans often donât seek out the care they need, and they are wary of clinical trials.
In addition to being a practicing primary care physician in Cleveland, Dr. Hall has a dual appointment on the teaching faculty at Northeast Ohio Medical University College of Medicine as associate professor of both internal medicine and integrative medical sciences. He also has an assistant clinical professor appointment at the Case Western Reserve University School of Medicine. -
"What impact does a remote clinical team operating model have on clinical trial performance?" Thatâs one of the hot topics from Ken Getzâs interview with Executive Director of WCGâs Metric Champion Consortium (MCC), Linda Sullivan, MBA. He describes several projects that the Tufts Center for the Study of Drug Development (Tufts CSDD) is currently working on; ones that will provide robust, data-driven analysis and strategic insights that help drug development professionals improve pharmaceutical R&D. Getz is the Deputy Director of Tufts CSDD and Professor at the Tufts University School of Medicine.
At any given time, Getz states that Tufts is conducting between 12 and 15 grant-funded projects. The ideas for their studies are often generated from conversations in working groups, like MCCâs member networks. They also arise from thought-provoking conversations at industry conferences. But no matter where the studies come from, the studies always provide a âthink tank for that very high-level, macro-level view of whatâs happening in drug development,â Getz explains.
Listen to this podcast to learn how Tufts â and the data it publishes â offers insights for organizations to transform clinical trials. âEveryone I speak with tells me theyâve tried to measure and benchmark. There are lots of internal forces that have tried to tackle benchmarking, so weâre excited to have an evidence-based approach that provides an opportunity to help organizations shape their practices moving forward.â
Interested in attending the MCC Clinical Trial Risk and Performance Management vSummit September 8-10? Visit https://www.centerwatch.com/mcc-summit-2020.
If you subscribe to this podcast, you can use the code POD15 to receive 15% off your registration fee.
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Pharmaceutical executive Peter A. DiBiaso, MHA, a triathlete who has successfully completed the grueling Ironman, learned at age 49 he had early-onset Parkinsonâs disease. He shares his experiencesâand triumphsâin this episode.
Staying fit and continuing to be an athlete. He is still running marathons and recently scaled Mount Kilimanjaro, the highest mountain in Africa.Engaging with patient advocacy groupsâin particular, the Michael J. Fox Foundation. (In fact, his Kilimanjaro trek was part of a Team Fox effort to raise money for the foundation.)His ability to navigate the system.
What started as a small tremor in his right hand and a stiffness in his right ankle ended up changing his life.
It was a blow, but he knew what to do: The diagnosis brought his personal and professional life full circle. Five years later, he reports minimal disease progression. He is doing well and remains incredibly active. Other than well-managed medication, to what does he attribute his success so far?He has also been involved in six clinical trialsâboth interventional and observational. He knew the value as an industry insider. Now, he appreciates the value from the patient perspectiveâand he better understands the frustrations patients face. It too often falls on the patients to initiate discussions about clinical trials. Thatâs fine for him, he says, but what about those who donât know trials are available?
His professional life has focused on patient recruitment issues, so he brings a 360-degree perspective. Sponsors, he says, need to focus on more than the academic and clinical elements; they must address real-world issues and trial participantsâ challengesâeven those that seem minor, like parking.
It comes down to patient-centricity, he explainsâputting patients at the center of the clinical research enterprise. It is becoming more common, but thereâs still a long way to go.
Peter DiBiaso, MHA, is a consultant to drug development companies and has worked at Pfizer, Shire, Vertex, and IQVIA. He is married and has two children, and he and his family split their time between Paris and Boston. -
Lisa Carlton, PhD, understands rare childhood diseases both professionally and personally. She was already a scientist specializing in rare diseases when her daughter was diagnosed with one: tuberous sclerosis. As VP of Global Regulatory Affairs for a biotech drug developer she is involved in development of treatments for other rare diseases too. This makes her an ideal person to co-chair an industry-patient-advocacy group called the Working Group on Regulatory Science, a part of the EveryLife Foundation for Rare Disorders that seeks to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.
Tuberous sclerosis, a rare genetic disorder, affects one in every 6,000 newborns in the United States. It is a disorder that causes benign growth in almost any tissue; itâs of particular concern when it occurs in the brain, lungs or kidneys. Some people who have tuberous sclerosis may have learning problems or difficult-to-control seizures. Her daughterâone of a pair of twinsâwas diagnosed in utero. Sheâs doing well, although the speech and language disorders associated with the condition have made it hard to access the curriculum in school.
Dr. Carlton talks about the challenges of developing new treatments for rare diseases and the approaches taken to try to overcome them. She also talks about her daughterâs disease, and how families can bring their best to their journey as they adapt to support their child. Based on her experience as a parent and advocateâas well as her experience in the industryâshe has this advice for families: âTake a breath.â Drug development is slow, especially in rare diseases. âYou are in for a marathon of supporting your child.â
When possible, connect with other parents and advocacy groups. Or build your own tribeâwork, friends, family, etc. âYou have to be healthy and rested enough to do this. You have to be at your best.â
Sheâs often asked how she manages to keep going. âI donât know how I wouldnât do it. Youâve been given this gift of a special child. Now itâs time to find your tribe and push ahead.â
Her advice for a biotech company considering launch of a clinical trial for a rare disease? Nobody knows more about a patientâs journey, the patientâs experience, than the patientâor a parent or caregiver. âJust listen to what patients are saying. You are going to learn a lot.â
Lisa Carlton, PhD, has previously worked in regulatory roles at the National Institutes of Health, Otsuka Pharmaceuticals and International Partnership for Microbicides. She is currently serving as Vice President of Global Regulatory Affairs at REGENXBIO, a gene therapy-focused biotech company with headquarters in Rockville, MD. Lisa received her PhD in Pharmaceutics and MS in Medicinal Chemistry from the University of North Carolina in Chapel Hill and a BS in Biochemistry from North Carolina State University. - Laat meer zien
