Tips for Success

• Keep presentations balanced. Although you want your disease community to participate in clinical trials, you also want each person or family to make the right choice for their situation. Balanced information is key to retaining your patient community’s trust.
• Set realistic expectations for any clinical trial. It is important for your disease community to understand that even interventions that make it to Phase 3 may not gain FDA approval. 
• Consider an opt-out for emails and social media about new clinical trials for those that do not want to participate. 
• Explore the websites of other groups to see how they provide general information about clinical trials as well as information about specific trials. 
• Reach out to the study coordinator and/or principal investigator if a trial pops up for your disease community that you were not expecting.

Overview

If clinical care guidelines have been previously developed and published for your disease, you will want to ensure an updated version is published when a therapy receives U.S. Food and Drug Administration (FDA) marketing approval. You can work with your medical advisors, specialists involved in the clinical trials, and the specialists involved in the previous version of the guidelines to make sure the updates are clear and continue to provide a treatment decision framework for patients and their doctors. Addition of a new therapy will require the guidelines go through an approval process similar to the original development. 

If clinical care guidelines have not previously been developed for your disease, the approval of a new therapy can be a great time for your patient group to promote and encourage the development of clinical guidelines. Patients and their caregivers can provide input about symptoms, side effects of different treatments, and the importance of therapies.

Benefits of Guidelines

Clinical care guidelines serve as a framework for clinical decisions and support best practices. Guidelines are developed through a critical review of the medical literature, outcomes research, and clinical practice standards of care for diagnosing and treating the symptoms of disease. Benefits include:

• Provides evidence-based clinical guidance.
  • The level of evidence for each recommendation is included.
• Assesses the likely benefits and harms of a particular treatment
  • Strength of a specific recommendation is also rated and includes overall assessment of the treatment and impact of treatment.
• Enables clinicians to select the best care for a unique patient based on the patient’s preferences, the medical evidence, and the available options. In other words, the goal is patient-centered treatment. 
• Integrates newly approved medical interventions so that patients and their doctors can make informed decisions.
• Ensures current standard of care is provided to patients with rare diseases or diseases that are challenging to treat especially when access to specialists familiar with the disease is limited.

Disseminating the Guidelines

After publication of new or updated clinical care guidelines, your group can play a role in making sure clinicians treating your disease are aware of and have access to the guidelines. Your group can:

• Post the guidelines on your website.
• Use strategies listed in NCATS Toolkit module Develop Programs to Speed Diagnosis: Raising Awareness.
• Encourage patients who are not being seen by specialists familiar with your disease to share the guidelines with their doctors. 
  • Primary care doctors who may be coordinating a patient’s care may also benefit from the guidelines.
• Develop a Users Guide to provide additional information about how the guidelines were developed and how they can be used.
  • Friedrich’s Ataxia Research Alliance (FARA) provides information about their involvement in the development of clinical management guidelines for Friedrich’s ataxia (FA) and User Guide in: Consensus Clinical Management Guidelines For Friedreich’s Ataxia (FA) .
• Consider offering alternative ways for patients to keep a copy of the guidelines with them at all times. For example:
  • FARA, in collaboration with a major pediatric hospital, offers patients with FA a USB bracelet to store the FA clinical care guidelines as well as a patient’s personal medical records. The bracelet provides a physician easy access to all of the patient’s medical information. 
    • Information about the bracelet is towards the bottom of their Consensus Clinical Management Guidelines For Friedreich’s Ataxia (FA)  webpage.
  • Some patient groups are also developing applications for smartphones to provide similar information. 

Guideline Development Process

The development of clinical care guidelines has been established to ensure the guidelines present recommendations based on current evidence and are feasible, measurable, and achievable. In many cases, guideline development involves a collaborative effort.

• Collaborators on clinical guidelines may include: 
  • Patient groups and associated medical advisors.
  • Specialists for a specific disease.
  • Medical professional societies. Societies most often provide a website with information on the guidelines development process. A few examples include: 
    • American Academy of Pediatrics (AAP) Clinical Practice Guideline Manual
    • American Academy of Family Physicians (AAFP) Clinical Practice Guideline Manual
    • Endocrine Society Practice Guidelines Methodology
    • American Academy of Neurology (AAN) Systematic Review and Guideline Development Process
• General process: Clinical Practice Guidelines We Can Trust: Standards for Developing Trustworthy Clinical Practice Guidelines (CPGs) (2011) is a compilation of standards that can be used to determine how to successfully create clinical practice guidelines. 
  • The 8 standards are:
    • Establishing transparency.
    • Managing conflict of interest.
    • Guideline development group composition.
    • Clinical practice guideline/systematic review intersection.
    • Establishing evidence foundations for and rating strength of recommendations. 
    • Articulation of recommendations.
    • External review.
    • Updating.
  • The document provides recommendations for composing a group to create the guidelines and how to review and update the guidelines.
  • Medical professional societies base their own process on the standards set forth in this document.
  • These guidelines were developed by the Institute of Medicine (IOM), a division of the National Academies of Sciences, Engineering, and Medicine (the National Academies)
    • The document is only 2 pages in length. 
    • The IOM was renamed the Health and Medicine Division (HMD) in 2016.
    • The National Academies are a series of private, non-profit institutions that have been in operation since 1863, when President Lincoln signed a congressional charter to the National Academy of Science. The National Academies conduct independent, objective activities to solve complex problems and inform public policy decisions related to science, technology, and medicine. 

Supporting Guideline Development

Depending on who is leading the development of the guidelines, your involvement may differ. However the voice of the disease communities is increasingly essential to the development of clinical care guidelines, especially for rare or complex, challenging-to-treat diseases. Your group can:

• Organize an executive committee to oversee the development of clinical guidelines.
• Collaborate with a medical professional society or a group of specialists already in the process of developing guidelines to ensure your disease community’s concerns, preferences, and unmet medical needs are considered. You can provide data from:
  • Patient registry
  • Natural history study
  • Patient preference studies
  • Patient benefit risk studies

Overview

Following marketing approval, your group may continue to play a role in ensuring the new therapy is accessible and available to your disease community. Enabling broad access to a newly approved therapy often requires working with public and private insurance providers about these new changes in patient care to inform appropriate decisions on coverage. 

Currently there is a lot of debate concerning the cost of accessing cutting edge therapies, but no clear solutions. Thus, there are limited resources providing tips or strategies available on this topic. As patient groups and other key stakeholders work together to find ways to ensure accessibility, more information and relevant resources will be added to this section. 

You are encouraged to share any helpful resources, tips, or strategies that your group used to successfully address access barriers to a new therapy in your disease community for possible addition to NCATS Toolkit through the Contact Page . You may also wish to share current challenges and even strategies that have not been successful.

Tips for Success

• Talk to other patient groups at conferences and other events to share strategies that have been successful and those that have not been. 
  • National Organization for Rare Disorders (NORD) and Global Genes provide multiple opportunities each year to meet other groups. 
  • Rare Disease Day events may also provide opportunities.
• Watch for conferences focused on achieving reimbursement through public and private insurance providers for newly marketed therapies.
• If your newly marketed therapy uses a cutting edge technology, such as gene transfer, consider collaborating with other patient groups who also have a newly marketed therapy using the same technology. 
• Request members of your disease community share with you their own successes with specific insurance providers. 
  • You may be able to leverage one person’s success into a more global acceptance by the specific insurance provider. 
  • You can share the strategy used to gain approval with other members of your community who have the same medical insurance provider.
  • Determine whether there are any common strategies that might be applicable to other insurance providers.
  • Develop a toolkit of possible strategies that individual patients/families may use to gain reimbursement approval.
• Involve members of your Scientific/Medical Advisory Board as well as the specialists who treat your disease communities to advocate for insurance provider reimbursement.
  • Many professional societies have advocacy committees. You, members of your Scientific/Medical Advisory Board, or specialists in your clinical care network may consider reaching out to these committees to determine if a collaboration can be formed.

Working with Insurance Providers

Public and private insurance providers make decisions on the value of a newly marketed therapy and determine how the new therapy will be covered. The following resources provide insight into the complexities of insurance provider reimbursement and help you navigate ways to identify the barriers and promote coverage.

• FasterCures Value and Coverage Program is working to create a shared strategy to ensure that newly marketed therapies are not only developed but accessible to patients. 
  • At the core is bringing together the key stakeholders:
    • Patient and patient communities
    • Medical professionals working with the patients
    • Public and private medical insurance providers
    • Pharmaceutical manufacturers
    • Investors.
  • Partnering with Patients on Value, Coverage, and Reimbursement (2015) is a brief, but powerful written summary of a FasterCures workshop that included patients, insurance providers, and industry. The workshop sought to allow for a better understanding of challenges to insurance provider coverage and reimbursement. 
    • Key takeaway points include:
      • Early engagement with insurance providers can improve alignment between the evidence needed for the Food and Drug Administration (FDA) marketing review process and postmarket insurance provider reimbursement.
      • Both patient groups and insurance providers need to broaden their understanding of each other’s role.
      • Insurance providers need to build systematic processes for soliciting and using input from patient groups and communities.
      • Patient groups can provide data on unmet medical needs, burden of disease, patient and caregiver experience, and the value of therapies to support insurance provider reimbursement.
      • The disease community should maintain as united a front as possible, and collaborate when possible with other groups who are also working towards getting a newly marketed therapy approved for reimbursement.
    • Other helpful insights include the importance of:
      • Being aware that patient registries, along with clinical trials, can provide data supporting insurance provider reimbursement approval. 
      • Understanding what motivates different types of insurance providers to pay out claims to tailor messaging more effectively. For example:
        • Self-insured companies may strongly consider reimbursement of a new therapy if a part of the cost will be offset by the employee missing fewer days of work. This consideration usually applies to time taken off to care for a partner or child, if the employee is not the person who will receive the therapy. 
        • Managed care and pharmacy organizations might be more motivated by factors that will give them a competitive edge in the market, such as customer satisfaction.
      • Knowing the interactions and key players within a primary provider that may factor into the approval of a newly marketed therapy. For example: 
        • Pharmacy benefit managers may be focused on decreasing the cost of pharmaceuticals.
        • Managed care organizations may be focused on decreasing the cost of overall care.
        • Risk-bearing provider entities may also be involved in the decision making process. 
      • Conveying an understanding of trade-offs, both about the benefits and risks of therapy and the costs and outcomes of access, in order for requests for insurance provider reimbursements to be credible from the viewpoint of the insurance provider.
  • Links to more resources are available on FasterCures Value & Coverage Reports and Briefs webpage.
  • Consider contacting FasterCures to learn about this Program’s latest efforts: fastercures@milkeninstitute.org.
• Working with Regulators: A Focus on CMS (2014) is an online, text-based toolkit that provides information for patient groups about the FDA marketing review process and the changing patient engagement landscape. The toolkit was developed by the Cancer Policy Institute of the Cancer Support Network .
  • The focus of the toolkit is on the Center for Medicare and Medicaid Services (CMS) from the perspective of cancer patient groups but the messages apply to patient groups in general.
  • The toolkit provides:
    • Information to help you develop a working knowledge of the mechanics of the regulatory process and the role that CMS plays.
    • Tips to help you:
      • Monitor CMS activity to be aware of proposed new rules.
      • Learn about the engagement opportunities available to patient advocates in the national coverage process.
      • Identify the types of evidence and data that influence the decision-making process.
      • Understand the benefits, and potential risks of proactively requesting a coverage decision.
      • Develop strategies to optimize engagement with CMS and its key staff.
• The Patient Voice in Value: The National Health Council Patient-Centered Value Model Rubric (2016) can be used to evaluate whether existing value models are patient-centered and to guide patient engagement in the development of future value models.
  • Value models are used to help assess the value of new therapies.
  • The rubric was developed by the National Health Council . 

Developing Reduced Cost Programs

Depending on how your disease is diagnosed, you may be able to work to offer free or reduced diagnostic testing either through your clinical network if one is established or with the help of a larger umbrella group that includes your disease. 

• Work with your Scientific/Medical Advisory Board and clinical network to brainstorm possible ways to speed diagnosis.
• Collaborate with the Sponsor and/or manufacturer of the newly approved therapy to develop diagnostic programs.
• Consider factors that may affect your ability to develop a program. For example:
  • It may be easiest to develop a program for diseases diagnosed or confirmed by specific tests, such as:
    • Genetic testing
    • Bloodwork
    • Biopsies
  • It may be more difficult for diseases that are still diagnosed clinically, through exclusion of other possible causes, or require multiple tests.