Over the past few months, several articles have been released about new drug prices and their direct impact on inflating pharmacy costs. Today, we would like to offer more information on gene and cell therapies. While many of these products are very expensive and typically cost more than other specialty drugs, they are used to treat cancer (lymphoma and multiple myeloma) and other rare to ultra-rare conditions/diseases. For a reference point, a rare disease would be defined as impacting about 200,000 people in the US; an ultra-rare disease is defined as affecting about 50,000 people.

These drugs receive expedited review through an accelerated FDA approval process due to the nature of the diseases they treat and the lack of available therapies in the market.

Some examples of ultra-rare gene and cell therapies include:

  • Luxturna costs $850,000 per treatment and is used for Leber congenital amaurosis (1/50,000,000 births in the US)1 2
  • Rethymic costs around $2,700,000 per implant and is used for congenital athymia (~17-24 new cases each year in the US)3 4
  • Zolgensma costs $2,100,000 for a 1-time injection and is used to treat kids under 2 with spinal muscular atrophy (1 out of 6,000 to 10,000 births in the US)5 6

Understanding that treatment for these conditions is crucial, fast-tracking the FDA approval process can present its own challenges as the number of patients that receive treatment is very small due to the rare nature of the diseases themselves. The end result is limited clinical data, presenting insurers with a difficult choice of whether the therapies should be covered.

Although the overall pharmacy drug trend isn’t as high as some experts forecast, more and more gene therapies are in the pipeline and could bring significant change to the market. By 2025, 37 additional cell and gene therapies are projected to be approved. These new products will likely lead to an increase in trend.

Have additional questions about the impact gene and cell therapies may have in the marketplace? Contact PillarRx today!

    1. Padhy SK, Takkar B, Narayanan R, Venkatesh P, Jalali S. Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to Date. Appl Clin Genet. 2020 Nov 25;13:179-208. doi: 10.2147/TACG.S230720. PMID: 33268999; PMCID: PMC7701157
    2. https://medlineplus.gov/genetics/condition/leber-congenital-amaurosis/#:~:text=Frequency&text=Leber%20congenital%20amaurosis%20occurs%20in,causes%20of%20blindness%20in%20children
    3. https://www.benecard.com/fda-approves-novel-treatment-for-rare-immune-disorder-in-children/
    4. https://scidcompass.org/scid-compass/news/join-idf-congenital-athymia-101  
    5. https://www.statnews.com/2019/05/31/spinal-muscular-atrophy-zolgensma-price-critics/ 
    6. D’Amico, A., Mercuri, E., Tiziano, F.D. et al. Spinal muscular atrophy. Orphanet J Rare Dis 6, 71 (2011). https://doi.org/10.1186/1750-1172-6-71