Orphan Biosimilars: an Untapped Opportunity?

Nicola Davies, PhD, has a background in health psychology and over a decade of experience in medical writing. In addition to writing both US and EU regulatory columns, she has produced many large industry reports for the pharmaceutical industry. Topics she has covered range from orphan drugs to biosimilars, digital health, clinical trials, medical affairs, and payer perceptions, among many others. She also produces independent medical education for healthcare providers wishing to develop their knowledge of the pharmaceutical landscape.
November 08, 2019
Biosimilars have made headlines for their potential to offer a more affordable treatment option to patients in need of biologics. Today, with the increasing knowledge and identification of rare diseases, biosimilars have a renewed purpose in healthcare; they represent a significant opportunity to fulfill the unmet medical needs of rare disease patients at a much lower cost.

The problem of access to orphan drugs
The cost of orphan drugs is inversely related to the number of rare disease patients; therefore, healthcare payers are reassured that the percentage of orphan drug spending in the entire healthcare budget is smaller than it is for other drug classes. Despite this fact, payers cannot view the cost of orphan drugs as a minor consideration. As shown in a recent report by technology and clinical research company IQVIA, funded by the National Organization for Rare Disorders, the average cost of 10 orphan drugs used by the greatest number of patients is $9676 per year. This means that many patients have to share the cost of orphan drugs, making access to these products extremely difficult and often impossible for individuals with rare diseases–a population that has long had few or no treatment options.

Why biosimilar orphan drugs are rare
Despite the huge opportunity to develop biosimilars for rare diseases, very few are in development. One currently in the phase 3 recruitment stage is Amgen’s ABP 959, a biosimilar of the monoclonal antibody eculizumab (Soliris) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease characterized by blood clotting, destruction of red blood cells, and impaired bone marrow function. This biosimilar will be a welcome alternative to Soliris, which costs up to $500,000 per year per patient and can be a budgetary burden to payers.

Given the potential benefits of orphan biosimilars, why the lack of interest from manufacturers?

The challenge posed by regulatory guidelines is a significant barrier to orphan biosimilar development. While manufacturers are now incentivized to innovate with orphan-designated drugs, such special treatment is not available within the orphan biosimilars space. As Barbara Valenta-Singer, Chief Medical Officer of Fresenius-Kabi SwissBioSim, who leads a biosimilar development team, says, “The current biosimilar guidelines do not reflect orphan drugs.”

We can see this problem in Mexico’s guidelines for orphan biosimilars, where scientific evidence for originator orphan drugs does not need to be endorsed by a health authority expert committee in the same way as other drug classes do. Therefore, the reference drug’s scientific evidence cannot be considered when assessing orphan biosimilars, making it difficult to establish therapeutic equivalence, efficacy and safety of the biosimilar.1

“The biggest barrier for orphan biosimilars is the long clinical development pathway, which is not much different to that of the originator,” says Valenta-Singer. “This results in clinical trials that are even more challenging than those for the initial submission because it is almost impossible to find the required patient number within a reasonable time.” Some researchers argue that the most essential step in demonstrating biosimilarity is typically achieved during the preclinical stage. However, in reality, regulators still require the totality of evidence, including clinical trial data, to approve orphan biosimilars.2

While the regulatory approval pathways for biosimilars have become more clearly defined in major health markets, the same is not true for orphan biosimilars. It remains to be determined whether regulators are willing to relax the evidence requirements for orphan biosimilars or to grant incentives of the same extent they do with nonorphan biosimilars.3

A strategy for commercializing orphan biosimilars
A pressing question is: What can be done to increase the development of orphan biosimilars? According to Valenta-Singer, “This can be achieved if development pathways that have been adapted for orphan drugs could get endorsed by healthcare agencies [for use in the approval of orphan biosimilars]. With orphan biosimilars, there is a need for a stronger focus on the analytical similarity with originators and then to adapt the pathway based on whether there is a need for clinical data.”

Orphan biosimilars clearly have huge potential to generate savings for payers and improve patient access to essential treatments. The path-to-market must be made more favorable, however. In particular, payer support, incentivization, and a pricing strategy will be critical to secure orphan biosimilar success.3 These drugs will need to be offered with significant discounts compared to the reference products or to other expensive treatments if they are to penetrate the market.

To move the orphan biosimilar market forward:
  • Clinical development and returns on commercialization must be reviewed to promote innovation,
  • The guidelines for establishing biosimilarity must be developed in consideration of the cost to identify and recruit patients and conduct full clinical trials, and
  • Payer support and discount-heavy pricing strategies must be present to enable a less strained path-to-market
If these steps can be achieved, the development and commercialization of orphan biosimilars will generate significant savings for payers and enable pharmaceutical companies to sustainably address the unmet needs of rare disease patients. It will also clinically benefit those patients with coinsurance, high deductibles, or no insurance. Ultimately, advancing the orphan biosimilar space is a win for all.

1. Esquivel-Aguilar A, Hernández-Guadarrama CM, Terreros-Muñoz E, et al. Are there biosimilar orphan drugs for Gaucher disease? An overview in Mexico. GaBI Journal. 2019;8(2):71-5. doi: 10.5639/gabij.2019.0802.008.

2. Kos IA, Azevedo VF, Neto DE, Kowalski SC. The biosimilars journey: current status and ongoing challenges. Drugs Context. 2018;7:212543. doi: 10.7573/dic.212543.

3. Bolger T, Zhang P, Renjen V, Sattin B, Lin A, Venugopal A. The emergence of orphan biosimilars. J Bioanal Biomed. 2016; \8:3(Suppl). doi: 10.4172/1948-593X.C1.020.



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