What is health policy?

The term “healthcare policy” refers to the decisions, goals, and actions that determine how care is administered and accessed.  For example, policies can include legal and safety regulations by state or protocols regarding how care and medicine are delivered to patients at a specific hospital. “Healthcare policy” also refers to the policies set on a national level, in terms of access to healthcare, coverage, and cost. (St. Augustine Univ. of Health Sciences)

What does advocacy look like?

Personal –  Empowering individuals to advocate for themselves and their loved ones within their healthcare, personal, and community needs. 

Local – This includes advocating within your community for changes to make the local meet the needs of the KIF1A community. Community advocacy is a strategic approach to influencing outcomes and driving change on behalf of your community. It involves representing your community’s rights and needs to the local government. Local laws only apply to the narrow community that the governing body has authority over. 

State – This involves representing your community’s rights and needs to the state government officials, including regulatory and elected officials (state houses & Governors’). State advocacy can be beneficial over local advocacy as this ensures widespread access throughout the entirety of the state rather than smaller communities. State authority is broader than local activity, but will not apply to all individuals. Any state legislation or regulatory action only applies to individuals living in the State and individuals with state-regulated insurance. State regulated insurance includes ACA marketplace plans, state Medicaid programs, and fully-insured health plans. 

Federal – This involves representing your community’s rights and needs to the Federal Government including Members of the House of Representatives and Senate. This may also include engaging federal agencies such as the Department of Health and Human Services (HHS), or the Centers for Medicaid and Medicare Services (CMS). Federal legislative or regulatory change has the broadest impact. Federal laws and regulations apply to all persons, unless the legislation explicitly exempts certain types of individuals. Medicare and self-funded employer sponsored health insurance can only be regulated by the federal government. Approximately 50% of the US population receives their insurance from their employer.

Policy and Advocacy Survey

Given health policy and advocacy is a new area of work for KIF1A.org, we surveyed community members to identify key issue areas for our community. Below are some of the key findings from the 2023 Health Policy Community survey:

Type of insurance	Access barriers	What issues would you like to see KIF1A engaged in	Community would like more information on:
60% of respondents have both private and public insurance  21% of respondents have private insurance only 18% of respondents have public insurance only	78% of respondents have been impacted by prior authorization  36% of respondents have been impacted by step-therapy  Other access barriers experienced by community: Costs of procedures; Cost and coverage of out-of-state care; Cost and coverage of medical equipment; Prior authorization for EEGs Getting on medicaid waiver programs	90% of respondents interested in expanding access to clinical trials  60% of respondents interested in expanding access to diagnostic testing  54% of respondents interested in addressing current barriers to KIF1A symptom treatments	85% – Drug development process  81% – Biomarker testing  47% – Family Medical Leave Act (FMLA)  38% – American with Disabilities Act (ADA) 28% – Copay accumulators  23% – How to apply for state medicaid 19% – Step therapy  14% – How to appeal health insurance

Based on the above findings for 2024, and organizational capacity for engagement, the Advocacy Committee has prioritized working on one health policy issue related reducing barriers in access to current treatments. For 2024, engagement the advocacy committee began engaging on prior authorization reform. In 2025, advocacy will expand to one health policy issue relating to expanding access to clinical trials and treatment and one issue relating to access to current treatments and care.

What is prior authorization?

What is prior authorization: Prior authorization policies require a health care provider or an insurance plan enrollee to obtain approval from the insurer or its pharmacy benefit manager before the plan will cover the cost of a health care product or service. This practice can delay access to treatments and can be applied in a manner that is inconsistent with medical standards of care and best practices. 

What does prior authorization reform look like: Prior authorization reform is most often legislation that requires health plans to respond to prior authorization requests within a 24 or 72 hour time period. Prior authorization reform may also look like exempting certain health care providers from prior authorization policies if they already receive approval for certain treatments or services between 80%-100% of the time – this is known as “Gold Card” status. 

2025 Engagement

• On the federal level we will continue to support: The Safe Step Act, The MINI Act, Accelerating Kids’ Access to Care Act, ORPHAN Cures Act, and The National Plan for Epilepsies.
• At the state level, we will continue to support: prior authorization reform and efforts to protect Medicaid.
• Other emerging issues that are important to our families including but not limited to: investment in rare disease drugs and paid family caregiver programs.

2024 Activities

State Engagement: 

• 34 States have passed prior authorization reform (map)
• KIF1A.org submitted the following letters of support during the 2024 legislative sessions:
  • Vermont 

Federal Engagement:

• Safe Step Act – Step therapy requires you to try and fail on alternative treatments before your health plan will cover the originally prescribed medication. This can most commonly happen with medications for prescription drugs used for managing epilepsy, but can also occur with services or other coverage requests. State laws have been passed that provide exceptions for when an individual can avoid a step therapy policy and get immediate access to their prescribed medication (see if your state has passed a law here). However, these state laws only apply to health insurance plans that are purchased off the exchange, and some Medicaid plans. Any health plans provided through an employer can typically only be regulated by federal laws. Therefore, state-based protections around step therapy do not apply to employer-sponsored health plans. The Safe-Step Act would require that employer-sponsored health plans ensure there are exceptions to step therapy. Read the bill here.
  
• Rare Pediatric Priority Review – The Rare Pediatric Priority Review program provides pharmaceutical manufacturers that develop a drug or biological product for a rare pediatric disease with a priority review voucher (PRV). The PRV allows manufacturers to receive an expedited review for a different product. This voucher can also be sold. This program is intended to incentivize drug manufacturers to develop treatments for rare disorders. This program was not renewed in the 2024 end-of-year package. Therefore, it will be a priority for many groups in 2025 to ensure Congress continues to support rare disease research and development.
  
• National Plan for the Epilepsies – A national plan on epilepsy is passed by Congress and signed by the President to provide a framework for a national plan to address epilepsy. Epilepsy does not currently have a national plan. A national plan could include priorities such as (1) reducing epilepsy-related mortality from all causes; (2) improve the detection, prevention, diagnosis, and treatment of epilepsy and co-existing conditions; (3) decrease socioeconomic, racial, geographic, and other disparities in case; (4) increase the breadth and depth of epilepsy research; (5) expand national surveillance to capture the magnitude and diversity of epilepsy; and (6) enhance public awareness and engagement. Use this tool to see if your Representatives and Senators are on the Epilepsy Caucuses.
  
• MINI Act: The Inflation Reduction Act allows for prescription drugs to be eligible for negotiation with Medicare after a 7 year exclusivity period for small molecule drugs, and 11 year exclusivity period for biologics. Genetically targeted therapies, like ASOs, are not biologics or small molecule drugs, therefore, it is unclear when these drugs could be subject to negotiation. Therefore, the MINI Act would clarify that ASOs and genetically targeted therapies would not be subject to negotiation until after an 11 year exclusivity period, similar to biologics. This clarification is important to ensure manufacturers continue to invest in research and development of genetically targeted therapies like ASOs. 
  
• Home and Community Based Services Act (HCBSA) and Home and Community Based Services Relief Act (HCBSRA): Home and community-based services support disabled people and older adults living independently in their own homes by providing support in day-to-day activities such as getting dressed, preparing meals, assisting with medications, job support and using transportation. The HCBSA and HCBSRA would require Medicaid programs to cover these services and provide quality wages for these jobs, support funded respite care and caregiving services, reduce or eliminate caregiving waitlists, and support state Medicaid programs investment in caregiving support programs to ensure individuals living in institutions can be return to their communities.
  

Coalitions We Love!

Advocacy and policy change is a lot of work, which is why we work with several organizations that support our efforts and priorities. Here are some coalitions we love:

Care Can’t Wait – Care Can’t Wait is a coalition of organizations, stakeholders and advocates committed to building a comprehensive, 21st century care infrastructure — that means robust investments to expand access to childcare, paid family and medical leave (PFML), and home- and community-based services (HCBS), and ensure good jobs for the care workforce. 

Little Lobbyists! – This Little Lobbyists is a family-led organization that advocates for the needs of families and children with complex medical conditions. Little Lobbyists supports expanding the right to healthcare through Medicaid expansion programs, long-term services and support at home and with community-based services, access to affordable prescription drugs, and expanded access to mental and behavioral health services. This organization also supports the right to education for children with complex medical needs without restraints, seclusion, and other limitations in school settings. Little Lobbyists also supports expanded services to ensure children and people with disabilities can live and thrive in their communities. 

EveryLife Foundation – The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.

National Organizations for Rare Disorders — NORD was first national nonprofit to represent all individuals and families affected by rare disease. Today we’re the only organization working at the intersection of care, research, policy and community for all rare diseases.

Aimed Alliance – Aimed Alliance is a non-profit health policy organization that seeks to protect and enhance the rights of healthcare consumers and providers. Aimed Alliance provides resources that help educate consumers on how to challenge health insurance denials and better understand state and federal laws that are applicable to health plans. (P.S. KIF1A family member Ashira Vantrees serves as Lead Counsel at the organization – feel free to reach out if you need help challenging a health insurance denial avantrees@aimedalliance.org).

Global Genes – Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.

FREQUENTLY ASKED QUESTIONS

I want to write to my state or federal representative about an issue. How do I start?

Great!! State and federal legislatures can’t fix the problems our families face unless they know about them!

If you live in a state that has an active bill to reform prior authorization in your state or on another issue, we encourage you to email your locally elected official and request them to support the legislation. 

Your letter should (1) explain who you are and why you are writing to your official (tell them you live in their district/region/city — this is important); (2) explain why the issue is important to you; and (3) explain how passing this legislation will help you, your family, and families like yours. 

Prior Authorization Example: 

Dear [Add Name of Elected Official]: 

My name is [add name], and I am writing to you regarding XXX bill number. This bill would reduce prior authorization burdens for families in our state. Prior authorization policies require a health care provider or an insurance plan enrollee to obtain approval from the insurer or its pharmacy benefit manager before the plan will cover the cost of a health care product or service. This practice can delay access to life-saving treatments and can be applied in a manner that is inconsistent with medical standards of care. Personally, my family has experienced multiple challenges with prior authorization. For instance, [explain a time when you have been impacted by prior authorization]. 

This bill will [explain what bill would do to address the problem you experienced]. Therefore, I urge you to support and swiftly pass this legislation. 

Thank you, 

[Signed]

Does KIF1A.org advocate for issues globally?

Although KIF1A is a global community, current engagement is limited to the United States. The Advocacy Committee is still developing opportunities for global engagement, and aims to have opportunities for global engagement in the future. If you are interested in helping develop global advocacy opportunities please contact us at impact@kif1a.org.

Principles of Advocating on Behalf of the KIF1A Community 

KIF1A advocacy is on behalf of current and future individuals and families with KIF1A. KIF1A.org advocacy supports health care providers and researchers that are supporting the development of treatments and standards of care for individuals with KIF1A.  KIF1A policy activities include:

• Supporting and advocating for the development of KIF1A genetic therapies and treatments to help manage KIF1A symptoms; 
• Supporting and advocating for initiatives that improve access to care and quality of life for individuals and families living with KIF1A;
• Empowering individuals, caregivers and families with KAND participation in legislative, regulatory, and other advocacy initiatives; and
• Empowering individuals to develop creative solutions to current accessibility barriers while also recognizing that full participation in society should not require individuals to be a strong, vocal, and creative advocates to receive the necessary services they need to live with KIF1A or care for an individual with KIF1A.

What does a “win” in advocacy look like?

Advocacy wins can be big and small! Often legislators decide how they will vote on a particular issue based on how much feedback they receive from their communities about the particular problem. In some cases one or two letters urging an elected official to support an issue may be what makes them decide to vote in favor of something, rather than opposing it. 

Therefore, sending a letter regarding an issue, having a meeting with your elected official, or helping educate them on an access problem are all small but important wins! It can take years for certain legislation to be passed, therefore, continual conversations with elected officials helps ensure they understand the impact of the issue on their community, and can empower them to become an advocate for reform within their elected state houses. 

State officials passing legislation can also signal to federal legislators that their community cares about a certain issue. This can help support federal officials passing national laws that replicate state-based protections. 

Why do we need to engage in state advocacy and federal advocacy? 

State laws only apply to state based insurance companies such as individual and small group plans, and Medicaid. State law only applies to employer-sponsored health insurance that is privately insured. 

State law does not apply to employer-sponsored health plans that are self-insured. These plans can only be regulated by federal law. Sixty five percent of employer sponsored health plans are self-insured, therefore, federal advocacy is needed to ensure all individuals with ensure are entitled to certain protections. 

Lastly, rare disease development is primarily regulated by the FDA which can only have new mandates from the federal congress.

Where we’ve been & where we are going:

• 2023 RARE Advocacy Summit – 9/2024
• 2024 Biotechnology Innovation Organization International Convention – 6/2024
• 2024 Harrisburg State Advocacy Day – 9/2024
• HealthyWomen’s Policy Event: “Capitol Conversations” – 4/2025
• “All Hands Meeting” for NINDS at NIH – 4/2025
• Faster Cures’ Vital Voices Workshop: Opportunities for Patient Organizations to Engage with CMS – 5/2025
• Biotechnology Innovation Organization International Convention – 6/2025