White Paper: Exploring the role of medical writing in raising awareness and advocacy for rare diseases
Table of contents
Overview
Key findings
Summary
Abstract
This white paper delves into the crucial role that medical writing plays in raising awareness and advocacy for rare diseases. Rare diseases pose unique challenges due to limited understanding, diagnosis, and treatment options. Effective communication is pivotal to foster understanding, advance research, and advocate for policy changes. By elucidating the various facets of medical writing, this paper highlights its impact in driving awareness campaigns, patient education, research dissemination, and policy advocacy within the realm of rare diseases.
Key messages
- Medical writers create patient-friendly materials that empower individuals and families affected by rare diseases to make informed decisions about their healthcare
- They play a vital role in summarising and sharing fragmented rare disease research, making it accessible to professionals and the general public
- Medical writers influence healthcare policies by highlighting the impact of rare diseases and proposing solutions, nurturing positive changes in the field
Introduction
There is a secret realm of diseases that only a small portion of the population experiences in the extensive healthcare ecosystem, where the focus frequently shifts towards popular diseases and conditions. These are rare diseases, a broad category of disorders that affect millions worldwide, many of whom encounter challenging obstacles on their journey to diagnosis, treatment, and support. According to current reports, there may be well over 10,000 rare diseases, and an estimated 80% have a genetic basis.
There is no current global standard definition for rare diseases; the United States (US) considers a disease rare if it affects fewer than 200,000 individuals, while the European Union (EU) defines rare as affecting fewer than 1 in 2000 persons. Collectively, rare diseases are not uncommon as they affect over 300 million persons globally. Although the phenotype and physiology of each rare disease are distinct, patients and families across disease groups frequently report sharing common traits associated to the low prevalence of their disease. These may include but are not limited to, feelings of isolation and being overwhelmed with complex information regarding the disease. Due to the intricacy of the material and the fact that it is always changing, publicly available genetic information frequently requires improved readability.
There is no current global standard definition for rare diseases; the United States (US) considers a disease rare if it affects fewer than 200,000 individuals, while the European Union (EU) defines rare as affecting fewer than 1 in 2000 persons. Collectively, rare diseases are not uncommon as they affect over 300 million persons globally. Although the phenotype and physiology of each rare disease are distinct, patients and families across disease groups frequently report sharing common traits associated to the low prevalence of their disease. These may include but are not limited to, feelings of isolation and being overwhelmed with complex information regarding the disease. Due to the intricacy of the material and the fact that it is always changing, publicly available genetic information frequently requires improved readability.
As a result, families who have recently received a diagnosis are often referred to rare disease patient organisations (RDPO), also known as patient advocacy groups (PAGs), for comprehensive, up-to-date information. Typically, these organisations act as comprehensive support networks for both patients and their families.
The expression "alone we are rare, together we are strong" rings true in the community of people with rare diseases and patient organisations. It can be challenging for PAGs for rare diseases to raise awareness, create a supportive community, conduct research, create registries, and advocate drug development and therapy access.
In this context, medical writers serve as invaluable allies, wielding the power of words to educate, inspire, and bring together diverse stakeholders. They enhance the exchange of knowledge and understanding among patients, carers, healthcare professionals, researchers, policymakers, and the public by producing patient-friendly materials, medical reports, policy briefs, and scientific papers. Using the art and science of medical writing, these specialists enable the rare disease community to raise awareness, advocate for change, and assemble support.
In this white paper, we offer an in-depth exploration of the frequently overlooked status but key role of medical writing for raising awareness and advocating for rare diseases.
What are the benefits of patient-centric medical writing?
Patient-centric writing is a fundamental aspect of healthcare, especially in the context of rare diseases. It revolves around putting the patient at the centre and takes into account their needs, preferences, and values. This approach considers various factors such as health literacy, cultural background, and personal values.
For instance, when developing informed consent forms, patient centricity plays a crucial role. It directly impacts how well a patient can comprehend the content and subsequently make informed decisions based on that information. Including relevant and meaningful information tailored to the reader is another facet of patient-centric medical writing.
- 43% of UK working adults cannot understand health information
- This increases to 61% when numbers are involved
- Patients can only recall 49% of information shared by their doctor during appointments
Medical writers have the capacity to tailor content for specific conditions, disease stages, and treatment plans. This tailoring is based on the patient population and feedback, particularly when creating patient education materials. By advancing health literacy through patient-centric writing, we have the potential to enhance patient outcomes.
In today's digital age, patients are increasingly turning to the internet for information about medical conditions. Many disease societies create and distribute informative booklets, both in-depth and easily understandable, aimed at assisting patients and caregivers in effectively communicating their treatment and support needs to healthcare providers.
For example, having patient education leaflets or relevant online content can enable healthcare professionals to connect with a broader audience, emphasising the importance of their services in the healthcare industry.
“Getting the patient perspective is critical, including what’s important to them and what makes sense to them.”
The predicted course of a rare disease must be clearly understood by patients and their families. In this context, medical writers are essential in disseminating knowledge about potential outcomes, which includes topics like disease progression, prognosis, and quality of life complications. This information must be conveyed with compassion and empathy, taking into mind the emotional impact that a diagnosis of a rare disease can have on individuals and their families.
Furthermore, it is noteworthy that public involvement has proven to have an impact on several research cycle phases, including project design, recruitment, data collecting, analysis, writing, and dissemination. However, participation from users or the general public throughout the writing process of research publications is relatively uncommon. This may help to partially explain the limited reports on the impact of public involvement on the development of research publications.
Nonetheless, there have been instances where researchers have significantly increased their influence by including users' perspectives and thoughts in their results. Researchers have emphasised the benefits of obtaining public feedback while creating written materials, especially those working in the field of disability research. Patient involvement significantly improves the usefulness of the data for the intended audience.
Why do patients value good-quality information?
When an individual is diagnosed with a rare condition, they often find themselves in an unfamiliar environment, similar to being in a foreign country with no map or guidebook. They must make important health decisions without access sufficient information tailored for their specific condition. However, most current medical information is designed to “talk down to patients” rather than assisting them.
Traditional medical care focuses mainly on treatment options, ignoring the broader range of options that newly diagnosed patients must consider. Patients struggle to understand how each treatment option affects various aspects of their lives, frequently lacking the necessary context. If the pharmaceutical industry can help healthcare providers accurately show how a treatment option fits into the bigger picture, patients can then make informed decisions that are consistent with their lifestyles, personal needs, and values.
It is important to effectively communicate this to patients, but this is often not included in medical training or is only considered within the field of advertising in life sciences. Moreover, lack of time has been the primary obstacle to patient-centred communication for making informed decisions. Practitioners may feel they don't have enough time to talk with patients to listen, explain, and negotiate. Sometimes patients are interrupted by the provider before they can finish expressing their initial worries. Patients were interrupted after an average of 23.1 seconds in a trial that involved doctors and agenda-setting with patients. However, studies demonstrate that when given open-ended questions and given uninterrupted time, patients rarely spend longer than 2-3 minutes to tell their entire narrative.
While shared decision-making does need time, it often only adds 10% to the whole time, or 2 minutes for a 20-minute session. While time constraints may make patient-centred communication challenging, medical writers are key in providing patients with the information they need to make informed decisions. By removing these obstacles, collaborative and patient-centred care can be fostered, which will eventually improve treatment adherence and patient outcomes.
Moreover, medical writers should understand how to create reliable, objective information for patients without being promotional (intentionally or unintentionally) to find receptive audiences among patients, supporters, and health authorities.
The challenges in writing for patients
Effective patient information
- Information should be clear and relatable, not just scientifically precise. It should explain the medical details and translate them into what they mean for the patient
- Plain language is more than just simplifying complex terms, it involves providing context and explanations to ensure patients grasp the significance of the information
Understanding patient needs
- Effective writing begins with understanding what patients or the general public want to know, what they need to know, and what they might already know
- When it comes to medicines, patients prioritise four key areas: understanding the medicine's purpose, dos and don'ts during use, potential side effects, and how it impacts daily life
Supportive communication
- Rare diseases often involve a convoluted healthcare system with multiple specialists, treatments, and support services
- Patient communication materials should empower patients to navigate this journey effectively, offering advice on seeking second opinions, understanding insurance and finances, and accessing support networks and advocacy groups
The medical writer's role is to present this information in the most understandable style acceptable for the patient. This is sometimes far from simple, especially when the reader's first language is not English, they have cognitive or visual disability, or they are unable to read at all (requiring the careful use of visuals). It takes expertise and skill to recognise possible roadblocks to understanding, let alone overcome them.
Various types of context (scientific, medical, and societal) may be required to fully understand the messages that are being provided. In essence, medical writing is an invaluable tool for raising awareness and advocacy for rare diseases. It ensures that information is not only accessible but also meaningful, which eventually contributes to improved healthcare outcomes and a more informed patient community.
Creating easy-to-read materials for patients
Effective communication begins with a thorough understanding of the rare disease at hand. To truly understand the nature of the disease, including what causes it, its symptoms, and how it progresses, medical writers need to dive into scientific literature, consult experts, and conduct comprehensive research.
Rare diseases often involve complex medical jargon and scientific concepts that can be difficult for patients and their families to comprehend. Medical writers play a crucial role in simplifying this information, translating it into layman’s terms, and presenting it in a clear and easily digestible format. For information to be effective, it should use short words and short sentences in an active voice. Only essential information should be included.
Long or unfamiliar words can be difficult to understand and slow down reading. For easy comprehension, medical terminology can be improved by incorporating glossary lists, and key medical terms can be either bolded or hyperlinked to their definitions. The content should focus on one or two key objectives and be suitable for the age and culture of the intended audience. If medical terminologies are necessary, they should be clearly defined so patients can understand their meaning and context.
Humans tend to prefer information that includes pictures. In one study, patients remembered 85% of information with pictographs compared to only 14% without. Additionally, studying how rare disease patients and caregivers search for health information is crucial due to the limited availability of information. A survey of 136 RASopathies caregivers revealed a preference for digital and visual health information, especially regarding specialised medical practitioners and self-care with parents being more inclined to share trustworthy health articles.
Using visual aids can result in more efficient communication with patients, which can lead to increased recruitment and retention rates in clinical trials as well as improved medication use. To personalise the document to its intended audience, creating effective patient materials requires additional knowledge, skills, and expertise in health literacy. Medical writers who adhere to best practises frequently assess their work for "readability." While automated readability scores are available, they have limitations in that they only examine word and sentence length without taking into account content or vocabulary.
As a result, it is beneficial to go the extra mile and have patient materials reviewed by individuals as close to the target audience as possible to ensure that the materials are clearly understood and comprehended.
Pharmaceutical companies should consult with patient advocates to ensure their medical knowledge assists individuals with rare diseases. Medical writers can use language and terms that patients have heard from advocacy groups or other sources to create information that patients can easily understand. This is an important step in ensuring patients receive the necessary information to make informed decisions about their health.
To take it a step further, research has indicated that incorporating patient advocacy groups or target audience members in the review process can ensure that the materials are easy to comprehend and contain all the key information. Individual interviews can be particularly suitable when materials focus on sensitive health topics or involve patients who may face challenges participating in group activities due to their health condition, location, or other personal factors. Developing a plan and process for audience testing comes with evident ethical, logistical, and budget considerations that need to be taken into account.
Guidelines for easy-to-read patient education material
Think like a patient
- Understand the content’s relevance to the target audience
Write in plain language
- Use plain language, e.g., “It could make you sick” instead of “It cause adverse health effects”
- Explain correlations and causation in simple terms
Avoid assumptions
- Answer common patient questions
- Encourage questions to physicians
Inform readers
- Clearly state actions you want readers to take
- Use concrete nouns and an active voice
- Consider using narratives, such as stories, testimonials, or educational entertainment
Use familiar analogies
- Explain using relatable comparisons, e.g., “Feel for lumps the size of a pea”
Balance text and visuals
- Combine text and visuals, e.g., video animation, infographics
Patient advocacy
Medical writers play an essential role in shaping healthcare policies and funding initiatives through their contributions to policy briefs, position statements, and advocacy materials. They assist in elucidating the socioeconomic burden of rare diseases and propose policy solutions that drive positive change. Rare disease patients' proactive involvement has led to the developing of treatments that might not have existed otherwise. Patient networks and advocacy groups significantly influence rare disease research, clinical trials, regulations, and marketing efforts. These groups are particularly vocal, passionate, and involved, making their impact profound.
Advocacy groups have driven international cooperation among regulatory bodies, investigators, researchers, and patients. Their influence extends to social media, where they can augment or challenge the efforts of pharmaceutical companies' clinical and sales teams. Collaboration between pharmaceutical companies and advocacy groups can be highly productive based on open, honest dialogue, mutual respect, and shared goals. While advocacy groups do not market specific products, they can work with marketing groups to provide patient access to commercial organisations through sponsorship of educational meetings, community events, or fundraising activities.
Figure 1. Current challenges facing rare disease PAGs.
Participants can select challenges relevant to their organisation, such as those that necessitated advocacy for change or hindered strategy goals. Funding and awareness remain key concerns for most organisations.
What is the future?
Writing materials in plain language is now seen as a crucial way to give rare disease patients better treatment options. But figuring out how to create these materials so they genuinely help patients, rather than just meeting regulatory requirements, is a big task for the entire pharmaceutical industry.
Efforts to discuss and set standards for the content of these materials will definitely improve the quality of the information. However, because clinical research in rare diseases is so diverse, this is a complex challenge that we need to tackle head-on.
Once we've created these materials, we also need to make sure they're easy for patients to find and use. Historically, the pharmaceutical industry hasn't done this very well, but that's changing.
Working together with rare disease patients and patient advocacy groups can be a game-changer. They can help the industry understand the current issues and prepare for future challenges. And let's not forget the importance of giving healthcare providers the right tools and guidance to communicate effectively with patients and their caregivers. This way, patients get clear information they can use to improve their health.
Improved quality of information for individuals with rare diseases and the general population is becoming increasingly important. The good news is that regulatory bodies are taking notice and taking action. The standards governing clinical study outcomes are more tightly enforced in the EU and North America.
This could become a global trend, increasing patient engagement and trust in clinical research. Sure, it will be difficult, but it will be a positive change for everyone concerned. The pharmaceutical sector can now speak directly to the general public, which was previously prohibited. Patients and the general public can access fair, credible, evidence-based, and easy-to-understand information if done correctly.
To make this happen, we must actively listen to rare disease patients. We can accomplish this by testing information or speaking with patients or patient advocacy groups. Medical writers can assist by explaining medical terms and ensuring that material is presented in the best possible way.Conclusion
Medical writing occupies a pivotal role in raising awareness and advocating for rare diseases. By facilitating effective communication, translating complex information, and influencing policy changes, medical writers contribute significantly to improving the lives of those affected by rare diseases. As the landscape of healthcare evolves, the impact of medical writing in rare disease advocacy remains steadfast.
Conflicts of interest
The author declares no conflict of interest.
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Author
Aarti Chauhan
Medical Writer II & Social Media Lead