Gathering Meaningful Data

Hemophilia Toolkit

Building the Business Case: Gathering Meaningful Data

Employers who have experience with hemophilia recognize the high costs of treatment. However, they may not recognize treatment involves a class of therapeutic medications that require proactive management. Approximately 90% of the total cost of hemophilia is related to specialty drug spend, however adequate data and information on these claimants are not routinely provided by vendor partners. For employers to effectively manage their hemophilia spend, pertinent and timely data is needed.

Quantifying total cost of care and determining cost saving strategies start with examining how care is delivered.

  • Is care provided by a PCP, general oncologist/hematologist or a hematologist within a Hemophilia Treatment Center?
  • Is a review of the specialty pharmacy benefit design in place to determine if there are opportunities for more effective management?

Since hemophilia medication is often self-infused or self-injected at home, this information is not always captured using traditional sources such as medical or pharmacy claims data. Gathering this information often requires working with vendor partners to ensure they are collecting actual script data and patient bleed logs.

The impact of hemophilia on your company’s spend and/or hospital and ER use can be verified with ICD-9 or ICD-10 codes, hemophilia drug J-codes and/or NDC numbers. Work with your vendor partners to collect the information below.

“We don’t get data on the medical side or receive detailed reports from our PBM for hemophilia therapies, so we were not aware how much we were overpaying for inappropriate prescribing. The toolkit helped us figure this out and fix it.”

- MBGH Employer Member


Employer Action Steps

Download these steps here.

Work with your vendor partner, benefit consultant, and/or HTC to proactively evaluate your current benefit strategies, track prevalence, and identify and reduce wasteful spending. Even if all the steps cannot be completed, there is value in doing as many as possible. Gathering this information will help employers evaluate the effectiveness of an existing program, which could lead to plan design changes

Experts from the National Hemophilia Foundation are available to assist employers in evaluating their data at no cost. A consultant/broker may also be able to conduct a similar retrospective review of this data. Be sure to check with them regarding fees that may be charged before starting.

✅ Step 1: Evaluate your current benefit plan design to:

  • Understand your pharmacy network strategy.
    • Verify at least one Hemophilia Treatment Center pharmacy is in-network.
    • Confirm with your benefits consultant and/or vendor partner that your specialty pharmacy adheres to MASAC #188 guidelines.
  • Educate employees on different health plans about how they help them manage and treat their disease. Consider putting into place a decision support tool to help members choose the best health plan for themselves and their dependents 
  • Ensure appropriate case management and care coordination is evidence-based using an integrated multi-disciplinary team approach (see Hemophilia Treatment Centers).
  • Identify what utilization management strategies are in place (e.g. prior authorization, step therapy, preferred drug list, etc.); make certain criteria is not overly restrictive and patients have timely access to care without unintended or negative consequences (e.g. severe patient needs to demonstrate a certain number of bleeds in order to get access to particular therapies or is limited to one type of prophylaxis).
  • Determine if a co-pay accumulator adjustment program is being used; they are not recommended for use with high-cost, high-value drugs that have no generic equivalent.

✅ Step 2: Determine the number of individuals with hemophilia and the current cost to your plan.

  • The following links will allow you to cross-reference both your pharmacy and medical claims data to identify claimants and analyze opportunities for savings.
  • Ask your carrier/claims administrator to pull ER and hospital claims data with hemophilia as the primary diagnosis code.
  • If hemophilia drugs are linked to a carve-out approach, a larger component of the drug cost will show up in prescription drug benefit claims.

✅ Step 3: Hold your vendor partners accountable.

  • Work with a neutral third-party to conduct a retrospective claims audit that can be used as the baseline for driving prospective strategies and to ensure your vendors are doing what is required through quarterly reporting.
  • Share this sample data collection template with your health plan and/or PBM to help you assess clinical and financial performance, including:
    • Assay management performance reviews:
      • Identify target dose as written compared to actual dispensed dose.
      • Identify current contracted allowable +/- over target.
    • Dose management performance reviews:
      • Ordered versus shipped – oversight/reporting to confirm dispensations match shipments.
      • Patient bleed logs (collected and reviewed) to determine medication adherence and inventory on hand at patient’s home.
  • Ensure your in-network specialty pharmacy providers:
    • Follow the MASAC 188 guidelines.
    • Do not auto ship medications.
    • Require monthly communication with the patient.
    • Identify current inventory on hand before sending additional doses.
  • Consider integrating performance guarantees into your PBM contract – See Employer Example

✅ Step 4: Gain a better understanding of financial exposure and impact of individuals seeking care.

  • If you have a stop-loss policy, does it provide coverage with condition specific exclusions?
  • What exclusions exist? Are they applied differently to existing employees, new hires, or newly added dependents?
  • Are claims subject to reimbursement limits? How are claims managed if costs span across multiple years?
  • What kind of gene coverage policies does your plan include (if any)?

For more information on financial exposure, click here.


A laser in a stop loss policy is a provision that assigns a higher individual deductible to a specific member who is expected to have unusually high medical claims in the upcoming plan year. This shifts more financial risk back to the employer for that individual before stop loss coverage begins to reimburse.

“Employers don’t know what questions to ask their vendors, and it can get awkward to challenge them. Getting access to the toolkit’s step-by-step guidance in simple language is incredibly helpful.”

- MBGH Employer Member

Hemophilia Treatment Centers (HTCs) have a wide range of data reporting capabilities which can be requested by employers, including:

  • Patient classification by diagnosis
  • Total cost of factor replacement therapy
  • Quarterly assay management reporting that includes target prescribed dose versus dispensed dose deviation
  • Dose management reports that include patient bleed logs, doses on hand and extra doses shipped (including the reason for extra doses)
  • Whether a patient had any ER visits or hospitalizations related to hemophilia
  • ER avoidance report
  • Adherence monitoring
  • Total cost per patient
  • Number of patient contacts (clinic visits, follow up, telemedicine, email, etc.)

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