Access & Coverage Advocacy
With the first two FDA approvals of Duchenne therapies in the U.S., the Duchenne community has entered in a new environment for access and reimbursement. PPMD has been leading the effort to educate and guide the community through these unchartered waters.
ICD Code Refinement
On October 1, 2018 – the start of FY 2019 in the federal government – a distinct ICD-10 code for both Duchenne/Becker and FSHD was added to the amended CMS ICD-10 codes.
What does this mean?
Previously, Duchenne muscular dystrophy was classified among a broad category of diagnoses in the standard International Classification of Diseases (ICD). The International Classification of Diseases (ICD) is the foundation for the identification of health trends and statistics, and the international standard for reporting diseases and health conditions. The lack of an ICD code specific to Duchenne had proven a barrier to diagnosis, care, surveillance, research, and access.
For nearly two years, PPMD led an effort to refine the ICD code for Duchenne/Becker that included key federal agency and clinical partners, and was supported by the Foundation to Eradicate Duchenne (FED). In the late stages of the effort, PPMD recognized that the same evidence our team had established to refine the ICD code for Duchenne/Becker also supported the refinement of the ICD code for Facioscapulohumeral muscular dystrophy (FSHD) and we reached out to our partners at the FSH Society to work together to bring the nomination for the refined FSHD ICD code forward simultaneously.
The refinement of the ICD 10 code for Duchenne will:
- Ultimately facilitate better surveillance of these diseases
- Allow more accurate estimates of incidence, prevalence, survivorship, mortality and its causes, injuries, symptoms, and health visits of these diseases
- Will help to identify factors that influence health status
- Will assist in the recognition and management of secondary conditions, impacting both mortality and morbidity
On a larger scale, ICD codes can be used to compare health information and outcomes across hospitals, regions, clinical settings, countries, and even across time in a given location; and they serve to keep track of reimbursements and to facilitate the evaluation of guidelines (implementation, adherence, and impact).
At a time when therapeutic treatments for the specific forms of muscular dystrophy are being approved for the first time in history – the new ICD codes will enable us to identify patients eligible for these therapies, longitudinally evaluate the impact of those therapies, and ultimately improve quality and quantity of life for those living with Duchenne and Becker.
Access and Coverage Resources
PPMD began assessing the landscape and working to develop resources that would help ensure as favorable an access environment as possible, well before our first approved therapies. Our access efforts have included:
- Data gathering through The Duchenne Registry, PPMD’s national patient registry, and in partnership with the CDC surveillance program, MD STARnet
- Leading health economic studies
- Establishing a distinct ICD-10 code for Duchenne and Becker
- Engaging with public and private payers
- Leading national newborn screening efforts in Duchenne
- Building resources for the patient and clinical community to support navigation of access processes
- Working with the Institute for Clinical and Economic Review (or ICER, a research organization that objectively evaluates the clinical and economic value of prescription drugs), valuators and broad-stakeholders in the access community towards access that reflects the community’s need
Following the approval of EXONDYS 51, PPMD launched an Access & Coverage Resources in order to inform the community about the process of accessing approved therapies for Duchenne. Upon the commercial launch of Emflaza by PTC, the site was expanded to include information about access to Emflaza. In addition, we have recorded webinars about navigating the access ecosystem and the many stakeholders involved in this new access and coverage landscape. These webinars feature speakers with decades of experience in payer, pharmacy, access, and reimbursement issues, and provide community members with a deep understanding of access considerations, an overview of current policy issues with potential impact coverage, and tools to empower personal access.View Access & Coverage Resources
PPMD is committed to working towards community access to all approved products and will continue to work with all relevant stakeholders to ensure a favorable access environment for our community.
Unapproved Investigational Therapies
Before a drug is even approved by the FDA, there are mechanisms for patients to access unapproved therapies. Terms like “compassionate use” and “expanded access” are often used interchangeably and can be confusing when trying to understand the rules, benefits, and risks, associated with these programs.
PPMD has created a guide to all the programs associated with accessing unapproved drugs and how they may differ from each other.View Overview on Access to Unapproved Investigational Therapies