Gathering Meaningful Data

Hemophilia and Bleeding Disorders Toolkit

Building the Business Case: Gathering Meaningful Data


Employers who have experience with hemophilia in their covered populations 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.

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.

Figuring out how to quantify current total cost of care and determining cost saving strategies start with drilling down and looking at how care is being 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 make sure they are collecting actual script data and patient bleed logs.

Review the steps on this page and/or download the Employer Checklist to learn what the prevalence of hemophilia is in your population. Evaluate your current strategy related to hemophilia and know what steps can and should be taken with vendors to improve overall quality of care and reduce related costs. Even if all the steps cannot be completed, there is value in completing as many as possible.

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.

Employers should work with their vendor partners and/or HTC to capture as much of this data 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.

Hemophilia Treatment Centers

Hemophilia Treatment Centers (HTCs) have a wide range of data reporting capabilities which can be requested by employers. Data points can include:

  • Patient classification by diagnosis
  • Total cost of clotting factor or substitution 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.)

Steps to take with Vendor Partners

Download Checklist


Use these steps to evaluate your current benefits 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.

Step 1: Evaluate your current benefit plan design to:

  • Determine which specialty pharmacy network strategy is in place (exclusive, open). 
  • Ensure contracted in-network specialty pharmacies adhere to MASAC #188 guidelines.
  • Verify at least one Hemophilia Treatment Center and one specialty pharmacy are in-network.
  • Confirm members have access to more than one type of health plan (e.g. PPO, EPO, HDHP, etc.); educate them around the different levels of financial responsibility for each.
  • 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 – one bad bleed can lead to permanent joint damage or even death).
  • Determine if 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 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.
  • Develop prospective data sharing requirements such as this sample data collection template which includes:
    • •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:
    • •Are required to follow the MASAC 188 guidelines.
    • •Do not auto ship medications.
    • •Require monthly communications 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: Check stop-loss policy (if utilized) and claims to confirm:

  • Whether medical and/or prescription drug claims are covered in combination or as separate stop-loss thresholds.
  • Amount of coverage and qualifying thresholds for stop-loss coverage to begin.
  • Types of claims covered and timeframe for coverage.
  • Number of treatment episodes reaching payment thresholds over three individual, but consecutive, plan years.
  • Total amount of coverage provided for each.