The American College of Medical Genetics and Genomics Have Addressed High Drug Costs for Patients with Rare Diseases

Recent medical developments have led to new drugs designed to treat rare genetic disorders. However, the high cost of many of these drugs limits the number of patients who have access to them. The American College of Medical Genetics and Genomics (ACMG) has released a statement that addresses this issue and suggests potential policies that could be used to ensure their affordability in the US. You can find the source statement that this article is based on by clicking here.

Healthcare Developments and Costs

In their statement, the ACMG talk about how new medical developments over the past decade have led to new drugs being created for genetic disorders. However, they highlight the high costs of ‘speciality’ drugs for rare diseases, using the example of the projected costs for nusinersen (SpiranzaTM) of $750,000 for the first year, and $350,000 for each year after that. In the statement, the ACGM say that high prescription drug costs are “a national concern.” They say that this, coupled with ‘cost sharing’ schemes, in which patients pay some of the costs of treatment, can lead to patients finding it very difficult or impossible to afford appropriate care.

Orphan Drugs

The Orphan Drug Act of 1983 encourages companies to develop drugs for rare conditions. However, according to the AGCM, the high costs of research, development, and clinical trials for these, combined with the comparatively limited number of people who would need the drug, can mean that drug makers need to set high prices in order to recover the costs of developing the drugs.

Potential Solutions

The AGCM outline seven potential policy steps that they believe could be used to help people afford and access medical treatments for rare disorders in the US. These are summarised below.

  1. Make the drug pricing process transparent and publicly available, so that companies are less likely to increase prices based on what they believe most people would pay.
  2. Carefully evaluate which drugs should benefit from the Orphan Drug Act, to prevent abuses.
  3. Make prior authorisation policies for drugs clear, timely, evidence-based, and publicly available to prevent unnecessary delays in starting treatments.
  4. Cap the amount that patients need to pay out-of-pocket for speciality drugs.
  5. Match dosing recommendations and the quantities included in the drug packaging, so that patients do not need to buy more than is necessary.
  6. Allow the federal government to negotiate drug prices.
  7. Stop producers from using financial incentives to block or delaying generic drug equivalents.

This list is a brief overview of the AGCM’s recommendations. For more detailed information, you can read their full original two-page statement by clicking here, or view it on Eureka Alert here.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

Follow us