Value & Cost

Understanding the “value” of sickle cell treatments has become a priority across the sickle cell disease (SCD) community. Value assessments, or health economic assessments, can advise whether a new treatment should be used, and if so, which patients are most likely to benefit from it. 

Recommendations stemming from value assessments are used to inform the decisions of clinicians, patients, and payers and can impact the coverage and access for new treatments. At Sick Cells, we focus on improving value assessments for sickle cell disease through:

  • A transparent and collaborative approach
  • representation of patient and caregiver perspectives
  • high-quality databases that adequately account for the diversity of SCD patients
  • methods that support equity

Sick Cells works with patients, researchers, health economists, payers, and providers to find the right approach to measuring cost and value for SCD.

2023 ICER Review

ICER is currently examining the comparative clinical effectiveness and value of two gene therapies for sickle cell disease (SCD):

ICER’s review process for these emerging SCD therapies will last 8 months. More information on ICER’s timeline and materials can be found here. For additional resources:

  • Review the ICER Draft Evidence Report here, published on 4/12/2023
  • Public Comments to ICER were submitted on 5/9/2023. See the two letters below.
  • Sign up for the ICER Public Meeting on 7/27/2023 here.

Sick Cells will be working to ensure the community’s attitudes toward emerging gene therapies are represented in the ICER review. The 2023 ICER toolkit helps provide information to advocates on the ICER process and how to bring community voices and opinions to ICER.

2023 ICER toolkit

(click the image to view the toolkit)


To stay engaged with Sick Cells’ work during the 2023 ICER Review, email us at


Public Comments to ICER

Two public comment letters for the Draft Evidence Report were submitted to ICER on 5/9/2023. Click the images below to view the letters.

This letter outlines Sick Cells recommendations on the 2023 Draft Evidence Report for the Treatment of Sickle Cell Disease

This Community Sign-on letter includes recommendations to include the community perspectives in the revised 2023 Evidence Report for the Treatment of Sickle Cell Disease

One public comment letter for the Final Evidence Report was submitted to ICER on August 10, 2023. Click the image below to view the letter.

This letter outlines Sick Cells comments on the 2023 ICER Final Evidence Report for the Treatment of Sickle Cell Disease.

Learn more about Value Assessments in our FAQ section and see our previous work on the 2019 ICER review below.


  • 01. What is Value in Healthcare?

    The Food and Drug Administration (FDA) evaluates new drugs before they can be sold. They must ensure that:

    • The treatment works correctly
    • Health benefits outweigh risks
    • The treatments are safe and effective

    The process normally takes up to 2.5 years, however, there are several approaches making drugs available as rapidly as possible for serious conditions with unmet medical needs, like sickle cell disease.

    After FDA approves a new treatment,
    several other important decisions are being made:

    Drug Manufacturers:

    Make decisions regarding the price of their drug and how it will be distributed.

    Insurance companies and Pharmacy Benefit Managers:

    Make coverage decisions and determine the amount they will pay


    Make decisions on which of these treatments and other healthcare services are best for them.


    Make decision about what treatment is best for individual patients

    One question that everyone needs answered it: What is the VALUE of this new treatment? What is value in healthcare?

    • Value is defined as outcomes achieved per dollar spent.
    • Value should always be defined around the customer.
    • Value in health care remains largely unmeasured and misunderstood.
  • 02. How is Value Determined?

    Organizations, such as the Institute for Clinical and Economic Review (ICER), develop value assessment frameworks and produce reports on the value of treatments.

    Value assessments involve undertaking an assessment of comparative effectiveness and cost-effectiveness.

    • Cost effectiveness is a health economics analysis that compares the relative costs and outcomes (effects) of treatments. Some insurance companies use ICER’s assessments when determining if they will cover new medicines.

    This may impact access and affordability of these much-needed new medicines in the future.

  • .04 What do we mean by Cost of SCD?

    When we talk about the “Cost of Sickle Cell,” we include direct medical costs, indirect and non-medical costs that result from caring and treating sickle cell disease.

    • Direct medical costs include cost of medical appointments and Rx medicine. These costs are typically paid by private and public insurance programs, and patients are responsible for co-pays.
    • Indirect costs include other incurred losses such as lost wages, lost productivity, and costs resulting from transportation, parking, or childcare during medical visits.
    • Out-of-pocket costs are the direct payments you pay that are not reimbursed from insurance or other third-party source. These include medical supplies, pain management or mental health services, and vitamins or nutritional supplements. 

    Have you heard the term “economic burden of disease” before? Generally, “economic burden of disease” means the TOTAL cost of providing care for a disease, and it is usually measured on three levels: (1) the cost to the individual, (2) the cost to the family, (3) and the cost for public or private systems (i.e. hospitals, clinics, insurance, etc.).

    Some people consider direct costs (medical bills) to be the only measurement of cost, but at Sick Cells, we also think non-medical and out-of-pocket costs need to be included to understand the impact of SCD.

2019 ICER Review

In August 2019, ICER announced its plans to assess the comparative clinical effectiveness and value of new treatments for sickle cell disease. Their review concluded in March 2020. At Sick Cells, we were focused on ensuring the voice of patients and advocates were included in the value discussion. Below you can read about our advocacy efforts during the review.

Stage 1
The Scoping Document
Stage 2
Patient-Generated Health Data
Stage 3
The Public Comment Period
Stage 4
The Final Report

The Scoping Document

During the “Scoping Phase” of the review, Sick Cells submitted public comments from 92 sickle cell patients and community stakeholders. We also submitted the following documents:

Patient-Generated Health Data

Sick Cells developed and fielded a patient and caregiver survey during the ICER SCD review. The “My Life with Sickle Cell Disease” survey helped elevate the patient and caregiver voice through patient-generated data. ICER’s model inputs for the societal effects of SCD were informed by this survey, which provided data about:

  • impact of SCD on patients’ ability to work and attend school
  • productivity loss from SCD
  • out-of-pocket costs of caring for SCD
  • caregiver burden

We received a  total of 547 survey responses, of which 454 were used in the analysis:

  • 289 (64%) were patients living with SCD 
  • 165 (26%) were caregivers of patients living with SCD
For more information on limitations of the a Draft Evidence Report, tune into the Value our Health webinar.

 The Public Comment Period

The draft evidence report was open for public comment for four weeks, during which the community rallied around this cause and delivered impactful materials. Sick Cells submitted public comments from 24 sickle cell patients and community stakeholders. We also submitted the following documents:

The Final Report

The Evidence Report published on March 12, 2020 is the final report for sickle cell disease. Due to the coronavirus outbreak, the public meeting was canceled. 

In June 2020, ICER’s shared their statement on Racism in America.  Read Sick Cells’ response letter to ICER below.

Even after this ICER review has ended, our organization remains committed to fighting for fair access and coverage to SCD treatments.  Stay update to date with these efforts through our Ambassador Program.

The Importance of Sharing Your Story

The Institute for Clinical and Economic Review (ICER) released a new report regarding the value and clinical effectiveness of 3 sickle cell treatments -Adakveo, Oxbryta and Endari. The report could affect patient’s access to these drugs, which is why we called on warriors and caregivers to share their stories and express the need for these treatments.

ICER’s report was currently missing key perspectives around fatigue, managing pain at home, caregiver stress and anxiety, and much more. By sharing their stories, individuals helped ensure the report accurately reflected the voice of the sickle cell disease community.

The SCD community submitted comments and shared their story. Watch Derek’s and Pam’s stories about the cost of treatment and what gives them hope.


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