Partnering in medical education: rare disease organizations bring experts and a patient voice to the conversation

Abstract

As new data from clinical trials and research studies are published about rare diseases, clinicians are challenged to stay abreast of the evidence, analyze it for clinical practice implications and then apply it to their medical practice. The Aplastic Anemia & MDS International Foundation (AA&MDSIF) realizes the important role our key medical advisors from the research and clinical community, as well as our patients – play in educating health-care professionals who may only occasionally see a patient with a rare bone marrow failure disease such as myelodysplastic syndromes (MDS). Patient-focused organizations can and should support medical education programs. AA&MDSIF sponsors live and web-based continuing medical education programs, provides tools to aid in diagnosis and classification of MDS, and provides free patient education resources to enable physicians, nurses, medical social workers and others to educate patients about their disease. Patient organizations have a responsibility to facilitate the delivery of rare disease-specific medical education to health-care professionals and an equal obligation to provide plain language information about our rare diseases for distribution among patients to help those patients better understand their diseases and treatment options.

Keywords::

• medical education
• myelodysplastic syndromes
• patient education
• patient organization
• rare disease

1. Why does a patient-focused non-profit organization need to support medical education?

For > 30 years, the Aplastic Anemia & MDS International Foundation (AA&MDSIF) has provided answers, support and hope to patients with aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH), 3 of the > 7000 rare diseases affecting over 30 million Americans.

MDS, characterized by hematopoiesis disorders, has an overall incidence of only 3 – 4 cases per 100,000 population per year and is considered a rare disease [1,2]. As a patient-focused, patient-centered organization, AA&MDSIF is a valuable resource for the estimated 12,000 – 15,000 patients newly diagnosed with MDS in the United States each year [3].

In a recent survey, our patients told us that their top source of information about MDS is their doctor (80%) [4]. Our goal is to ensure that when patients visit a hematology/oncology provider, they receive the best information possible from their physicians and nurses.

We recognize that many community-based hematologist/oncologist physicians, nurses and allied health-care professionals may have limited experience diagnosing and treating patients with a rare bone marrow failure disease like MDS. This creates a knowledge gap among practitioners regarding the best use of current therapies, particularly those practitioners who may only have an occasional patient presenting with a rare disease like MDS. There is an ongoing need for medical education presented by experts who compile and critically appraise new evidence and then interpret its application to clinical practice.

2. AA&MDSIF – offering answers, support and hope through live and enduring medical education helps MDS patients get the best care possible

Using evidence-based medicine to support clinical decision-making is the accepted approach for medical practitioners. However, as new data from clinical trials and research studies are published about rare diseases, health-care practitioners are challenged to stay abreast of the evidence, analyze it for clinical practice implications and then apply it to their medical practice. This lapse in the transfer of information from bench to bedside creates a knowledge gap among practitioners regarding the best use of current therapies.

Providing educational programs and products to health-care providers is one way we ultimately serve and benefit our patients. AA&MDSIF sponsors live and web-based continuing medical education programs, provides tools to aid in diagnosis and classification of MDS, and provides free patient education resources to enable physicians, nurses, medical social workers and others to educate patients about their disease.

Many rare disease patient organizations have key opinion leaders from the clinical or research communities advising their volunteers and staff. AA&MDSIF is guided by a 23-member Medical Advisory Board (MAB) representing leading researchers and clinicians in our three bone marrow failure diseases. Together with other nationally and internationally recognized experts, our MAB advises us on the best ways to fill the knowledge gaps for health-care providers who may see few patients with MDS in their practice. Patient-focused organizations can and should support medical education programs. The following highlights a few of the educational opportunities AA&MDSIF regularly sponsors and supports.

2.1 American Society of Hematology satellite symposium

For the past 7 years, AA&MDSIF has sponsored a satellite symposium in partnership with the Cleveland Clinic Taussig Cancer Institute prior to the annual meeting of the American Society of Hematology (ASH). The live program provides an in-depth and up-to-date review of research related to the biology, prognosis, natural history and therapeutic management of MDS and other bone marrow failure diseases. Recordings of symposium presentations are hosted online to allow additional health-care providers to learn at their convenience. Attendees of these satellite symposia repeatedly indicate a need for continued education on numerous topics of care for patients with bone marrow failure diseases, especially patient care strategies and new therapeutic approaches, practical and pharmacological aspects of therapies and care, patient selection, and diagnostic issues [5]. Outcome evaluation results show that these courses are successful in improving the clinical practice of participants.

2.2 International bone marrow failure disease-scientific symposium

Every 2 years, AA&MDSIF convenes physicians treating bone marrow failure diseases and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of controversy, share recent research results and propose specific recommendations for the highest priority directions for basic and clinical research needed to advance the field. The proceedings of this symposium are presented in several forms, including interviews with experts at the symposium to summarize the highlights for professionals and patients; an enduring program focusing on the most significant presentations; and a lay language Summary for Patients available online and in print booklet formats.

2.3 Bone marrow failure disease symposium: an update on the latest advances in diagnosis and treatment

Together with the University of Maryland in Baltimore, AA&MDSIF provided an update on the most recent scientific and clinical research related to bone marrow failure diseases. This included MDS, myelofibrosis, aplastic anemia and PNH. The presenters focused on the latest advances in the diagnosis, genetic characteristics, and pathology and treatment options for these diseases. Over 100 physicians, nurses, pathologists and researchers attended this jointly sponsored live meeting. The web-based recorded sessions provide enduring education opportunities. In addition, this format will be replicated in the 2014 Bone Marrow Failure Disease Symposia at Northwestern University in Chicago, IL, Penn State College of Medicine in Hershey, PA, and at Washington University in St. Louis, MO.

2.4 Rounds and other medical education

In conjunction with area medical/academic institutions, AA&MDS offers a continuing medical education series using case-based presentations with interactive discussion of clinical challenges in the treatment of myeloproliferative neoplasms and MDS. For the past 3 years, presenters in Chicago, IL, shared their best practices and participants learned the latest information on therapies and advances in the management of MDS and MPN.

Other AA&MDSIF efforts to provide disease-specific education to health-care professionals in recent years include:

• Discussion of the Management of Patients with MDS – In this recorded webcast, three physicians with extensive experience treating patients with MDS discuss the challenges in its diagnosis and management.

• Treatment Decision-Making through the Spectrum of MDS – A series of three web-based CME programs addressing communications strategies between health-care providers and MDS patients.

• Oncology Nursing Society Satellite Symposia – Recognizing that hematology/oncology nurses are in a critical position to monitor and impact the effect of MDS and the medical management of this rare disease treatment on patients’ quality of life [6], AA&MDSIF sponsored satellite symposia at the past two meetings annual of the Oncology Nursing Society. The goals of the most recent ONS Symposium activity were to update participants on the therapies available for MDS patients, to discuss the impact of comorbidities and psychosocial factors in disease management, and to improve the quality of care for this patient population. Understanding the education provided should be practical and easily applied by community-based practitioners [7], case studies were presented to illustrate the challenges of monitoring MDS and adapting therapy to improve outcome and quality of life for patients. The Oncology Nursing Society has awarded continuing education contact hours for the enduring recordings of the 2013 and 2014 symposia, which are available on the AA&MDSIF Web site.

3. Providing free apps and patient education tools helps health professionals educate patients

Upon hearing a diagnosis of MDS for the first time, many patients may not be able to comprehend what the disease is, their prognosis, or their treatment options. They likely have never heard of this rare disease prior to their diagnosis and do not know anyone who has had MDS. This can be an overwhelming and confusing time for patients and families. AA&MDSIF created a number of products to help health professionals teach their patients about their disease. It is often helpful to offer information in a variety of ways – one-on-one discussion, print materials, visuals, video presentations and recommendations for reliable Web sites. Clear presentation of information and active engagement of patients in the management of their disease can help clarify misperceptions and improve patient understanding of MDS. Our patient education tools for health-care providers include:

• The What is MDS? iPad app for clinicians and patients. Clinicians can use the video on the app as a resource when teaching patients about MDS.

• The Treating MDS Toolkit has been provided free of charge to over 800 hematology/oncology practices. AA&MDSIF surveys showed that patients with MDS have a limited understanding of their disease characteristics, prognosis and treatment goals. Sharing the expert reviewed patient education materials with health-care providers in this Toolkit provides them the opportunity to offer plain language, disease and treatment-specific information to inform and empower patients.

• MDS Risk Classification mobile app assists physicians with risk stratification and selection of treatment options for patients with MDS. It includes medical calculators for the major MDS disease classification systems.

4. Conclusion – recommendations

As the world’s leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, MDS and PNH, AA&MDSIF realizes the important role our experts – both key opinion leaders in medicine and our patients living with MDS – play in educating health-care professionals who may only occasionally see a patient with these rare bone marrow failure diseases. We feel it is our responsibility to facilitate the delivery of rare disease-specific medical education to clinicians, researchers, nurses and other health-care professionals. In order to reach health-care providers with both disease education and patient education resources, we recommend other rare disease organizations that consider:

• Offering live programs at venues where physicians, nurses and allied health professionals who treat our diseases are already gathering and record those when possible for enduring availability;

• Providing a variety of resources to meet the needs of community-based professionals with limited numbers of patients with a rare disease, who may not be up-to-date on the latest disease information and treatment options;

• Developing reliable, plain language patient education materials in various formats to facilitate communication between providers and patients.

For more information, contact us at [email protected] or visit us at www.AAMDS.org.

Declaration of interest

The authors have no relevant financial affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.