Pharma Manufacturers face high uncertainty with orphan drug development

Competition is increasing in several orphan indications. Unlike the past when most orphan indications had no comparator products available, several indications now have multiple products available in the market.


In addition to potential competition, manufacturers also face uncertainties during the development of an orphan drug which include the following:

Difficulties faced by payers

It’s not just manufacturers that face challenges with orphan drug development. Payers also face difficulties when assessing orphan drugs.

  • Cost containment

Healthcare systems are under rising pressure to control current and future costs. Payers are carefully examining drug prices as well as level of patient access to ensure the appropriate balance is achieved.

  • Uncertain Data

Uncertain clinical evidence is a problem that affects all payers. There are many orphan diseases with small sample sizes, so the lack of standard of care can make payers’ decisions even more challenging. The lack of long-term data in a larger population can make these cost considerations, beyond the few years of clinical data, even more of a challenge for the cost-conscious payer.

  • Encouraging Innovation

Payers are facing a tough balancing act. While they are looking to cut costs and demanding more supportive evidence, they are also mindful that it is necessary to encourage innovation of medicines for patients in need. They understand that innovation is important to ensure that patients who currently have limited treatment options have access to the treatments that they need. Some markets value innovation more than other others, with some markets even implementing specific innovative ratings as part of their HTA process.

Where is the greatest misalignment for Manufacturers and Payers?

We ran a poll in our recent Orphan Drugs webinar which asked participants where they felt the greatest misalignment stems from considering the challenges faced by manufacturers and payers.

The result was as follows:

  1. Clinical trial evidence (80%)
  2. Pricing (20%)

The results of this webinar poll were quite interesting. From our own GPI insight, it was identified that price is generally where the misalignment derives from when dealing with commercial viability. However, majority of the poll showed clinical trial evidence being where most misalignment stemmed from.

Kate Anstee, Associate Director, Consulting | GPI comments, “With reference to clinical trial evidence of orphan drugs in Europe, potential misalignment could be due to the numerous payer types in Europe and their requirements for clinical trials.

For example, some payers accept indirect treatment comparisons, whilst others do not. Not to mention deriving various analyses on the same clinical trial to meet these various payer’s evidence requirements.

Drug manufacturers are trying to develop clinical trials that meet regulatory requirements and demonstrate statistical significance at an early stage in their development process. Their priority and focus are ensuring they can demonstrate the efficacy, safety, and benefits of their product.

In trying find the appropriate endpoints and factor in market access this becomes quite complex since there are so many payer types resulting in a clinical trial that is a ‘one size fits all’ scenario.”

Are you having trouble keeping up with the Orphan Drug landscape?

The challenges are real! As a payer or manufacturer, it’s important to have access to key information in order to make informed strategic decisions, that are critical to your assets success. It’s just as important to be able to change your scenario planning throughout the development process as and when the landscape changes.

We can help you get ahead of these challenges with a first of its kind method to forecast value and price using our award-winning methodology, GPI horizon. We’re excited to show you how this approach can unlock your asset’s full commercial potential!

To learn more about what GPI horizon can do for your orphan asset, reach out to us below. You can also stay up-to-date with the latest GPI content by signing up to our newsletter.

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