Our Caregiver Resource page has information and resources to help and support caregivers in the SCD community. Check it out here:

Caregiver Resources

Value Assessment


  • 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. Who is ICER?
    • ICER (the Institute for Clinical and Economic Review) is a non-profit research organization.
    • ICER uses systematic literature reviews and health economics to evaluate the comparative clinical and cost effectiveness of new drugs
    • ICER’s assessments may be used by manufacturers to select a fair price for the drug based on the clinical benefit it provides to patients
    • ICER’s assessments may be used by payers to make decisions regarding fair coverage of the new drug
    • More information about ICER can be found on their website.

    Webinar overview: Do you have questions about value assessments and drug pricing? Sick Cells developed a webinar series to discuss value assessments and drug pricing.
    Learn more here.

    Please note: Contributing information or advice
    to ICER is in no way an endorsement of their work.

    Need a crash course on ICER and value assessments? Catch up on the Sick Cells’ webinar series here.

ICER’s Sickle Cell Disease 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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