What’s the outlook for the Orphan drug market in 2022?

In Clinical Development, Featured on App, Orphan Drug Congress, Reports, Uncategorized, Whitepapers by Karina Kusova

By Lisa Urquhart, EP Vantage Editor and Report Contributor

Annual “EvaluatePharma® Orphan Drug 2017 Report” sees continued growth for orphan drugs, approximately double overall prescription market growth

The steady and inexorable growth of the orphan drug market remains one of the prominent themes in the fourth edition of EvaluatePharma Orphan Drug Report 2017 from life science commercial intelligence firm, Evaluate. What has changed in the last 12 months is the increased scrutiny of the price of these lifesaving products.

There is little doubt that insurers will continue to cover orphan drugs, due to the fact they are frequently the only option for patients and, for now, remain a relatively small part of overall budgets. But according to the findings in this year’s report the market will carry on expanding rapidly, with sales growth forecast at 11% per year, more than twice the rate predicted for conventional drugs.

In fact, sales of orphan drug are expected to almost double between 2016 and 2022, to hit $209bn. This rapid growth and current willingness of payers to stump up for the huge price tags are two of the main reasons why the sector has become more and more attractive to some of the industry’s biggest players.

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The image of the plucky small biotech striving to develop treatments for the rare diseases largely ignored by big pharma is long gone. Instead, this year we again find big pharma dominating the sector. Seven of the top 10 companies by orphan drug sales are global industry players, who have won approval for their biggest products in various niche indications.

However, some see big pharma’s interest in this space as a potential problem, claiming that these groups are taking unfair advantage of the lower R&D cost, speedier approval times, favourable patent life and pricing incentives for developing orphan products.

And the pricing incentives are substantial. According to this year’s report, of the top 100 drugs in the US the average cost per patient per year for an orphan drug was $140,443 in 2016, compared with $27,756 for a non-orphan­­.

In what might spell trouble for the industry, payers and politicians are taking note.

Exondys 51 and Spinraza – both treat rare and fatal childhood illnesses – might have won broad approvals from the US regulator, but some payers are insisting on much narrower interpretations of their clinical effectiveness.

In February, Republican senator Chuck Grassley announced that he would be starting an investigation into potential abuses of the Orphan Drug Act. President Donald Trump has also expressed his concern over drug pricing; whether this will spill over into orphan drug pricing is unknown but not impossible.

As such, if the orphan drug industry is to continue to thrive it must continue to generate innovations that justify the huge cost of these life transforming treatments.

EvaluatePharma Orphan Drug Report 2017 Highlights:

  • Worldwide orphan drug sales forecast to total $209bn (CAGR 2017 to 2022:+11.1%); approximately double overall prescription market growth
  • Orphan drugs set to be 21.4% of worldwide prescription sales by 2022 (excluding generics)
  •  Median cost per patient 5.5 times higher for orphan drugs compared to non-orphan
  •  Celgene set to climb to number one position in orphan drug sales to 2022
  •  Shire to be largest company by sales in the orphan non-oncology space in 2022
  •  AstraZeneca, Abbvie and Johnson & Johnson set to march up the orphan drug sales ranking table
  •  Revlimid (lenalidomide) No.1 orphan drug in 2022
  •  Opdivo (nivolumab) No.1 orphan drug in Europe in 2022
  •  Orphan drugs forecast to account for 55% of the cumulative value of the European pipeline to 2022
  •  Axicabtagene Ciloleucel (Kite Pharma)is most valuable R&D orphan drug
  • First decline since 2012 for FDA orphan designations in 2016 with 333; Record number of applications made to the FDA for orphan designation in 2016.

The, “EvaluatePharma Orphan Drug Report 2017”, based on commercial intelligence and consensus forecasts from the EvaluatePharma service can be downloaded at www.evaluategroup.com/orphandrug2017

Visit Evaluate at Booth 313 at the Terrapin World Orphan Drug Congress USA 2017, April 21-22 in Washington, DC. Register now to attend >>

Visit Evaluate on Twitter @evaluatepharma.