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It’s All About Impact: Why Orphan Drugs are a Big Deal for Patients and Pipelines

300 million people worldwide are living with a rare disease. The “orphan” drugs created to treat them have been a fast-growing part of the pharma market for years – and their use is often high profile.

Rare disease

Every now and then a relative niche of the pharma world hits the mainstream – orphan drugs. It happened again recently in the UK when the NHS enabled a toddler with MLD to receive Libmeldy, a £2.9m gene therapy treatment. The drug by Orchard Therapeutics became the most expensive treatment ever provided by the NHS. Naturally, it hit the headlines, once again raising the profile of orphan drugs, treatments developed to treat rare diseases (defined in the US as those affecting less than 200,000 people, or less than 5 in 10,000 in Europe).

The reason that these stories get picked up in the mainstream media is obvious – there’s a clear human interest element. At Evaluate, we talk about “pipeline to patient”, the journey of getting the right drugs over the various clinical, regulatory and commercial hurdles to ensure they actually make a difference to patients’ lives. In the case of orphan drugs, the market for each treatment may be small, but the impact to individuals who previously had few or no options is huge.

The orphan market has been one of the fastest growing areas of pharma for several years, with more FDA approvals for orphans than non-orphans in four of the past five years. And for the moment, that growth shows no sign of abating. Evaluate Pharma consensus forecasts show the orphan sector growing at just under 12% CAGR between 2022 and 2028 versus around 7% for the wider market.

There are several reasons for this growth. Firstly, there are regulatory and commercial incentives to developing drugs for rare diseases. In fact, this is something we’ll be discussing in our upcoming webinar. The US Inflation Reduction Act (IRA) has the potential to shake things up in this space, but for the moment, the commercial and regulatory landscape treats the development of these drugs pretty favourably.

Continued interest in the orphan space by companies large and small has certainly delivered on the ‘spirit’ of the US Orphan Drug Act. This can result in orphan drugs often operating as a sandbox for new modalities. Approaches that start off specifically for a rare disease can deliver significant leaps forward for more widespread indications with a much larger market.

The human interest, the significant sums of money involved and the changing regulatory environment make orphans a particularly interesting corner of the pharma market. Our upcoming webinar will touch on all these points. I’ll be talking though the data highlights of our new orphan drug report with its author, Melanie Senior. We’ll also be joined by Jeremy Levin, CEO of rare diseases specialists Ovid Therapeutics who will discuss some of the potential implications of the IRA along with his views on what orphan drugs legislation needs to look like in the future. It’ll be fascinating conversation so do join us. The session takes place on Thur 16th March at 3pm GMT/11am EST and you can register here. Hope to see you there!


Andreas Hadjivasiliou

Managing Analyst


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