• Oncology already sits as the top-selling therapeutic area on the orphan drug market, but a new report predicts it to account for even more — half, in fact — of sales in the coming years.
• Global sales of orphan drugs totaled $125 billion in 2017 and will grow to $262 billion by 2024, according to market intelligence firm EvaluatePharma. Cancer treatments accounted for 46% of orphan drug sales last year, but will rise to 50% by the end of the period as products like Revlimid, Darzalex and Imbruvica continue to deliver impressive returns.
• EvaluatePharma anticipates Celgene will be number one in terms of orphan drug sales in 2024, driven by the performance of Revlimid and Pomalyst, both of which carry orphan drug designations for their approvals in multiple myeloma. Johnson & Johnson, Novartis, Roche and Merck & Co. would round out the top five by sales.

Life-changing, pricey and sometimes controversial, orphan drugs have been making larger and larger waves over the past few decades. Since 1983, the Food and Drug Administration has approved more than 600 orphan drug indications, with last year setting a record high of at least 50.

The exact definition of an orphan disease varies by regulatory agency. The Food and Drug Administration classifies it as a disease that affects fewer than 200,000 people in the U.S. In Japan, which has a much smaller population, it’s fewer than 50,000.

Oncology has proven particularly compelling. EvaluatePharma predicts that, among the top 10 drugmakers in the space at 2024, 60% of their orphan drug sales will come from oncology therapies and only one company won’t have any sales from oncology orphan drug indications.

There are many reasons behind the uptick. Regulators want patients to have access to more treatments for rare diseases; tax and research incentives have made it more financially feasible to answer that call; and products such as Celgene’s Revlimid (lenalidomide) and Alexion Pharmaceuticals’ Soliris (eculizumab) have shown other companies just how lucrative the market can be.

Yet headwinds are blowing stronger as of late. Lawmakers are expressing concerns about the steep price tags set for many orphan drugs. Critics are also slamming manufacturers for gaming the incentives system to keep profits high at the expense of patients’ out-of-pocket costs.

“The fact that this year’s report shows that seven of the top 10 companies by orphan sales are still big pharma groups, will not help the ongoing debate as to whether these well-funded groups should be benefiting from the regulatory and commercial advantages associated with orphan drugs,” EvaluatePharma wrote in its report.

Payers are providing another source of pushback. The U.K.’s National Institute for Health and Care Excellence, which determines cost-effectiveness of drugs, recently decided not to recommend use of BioMarin Pharmaceutical’s Brineura (cerliponase alfa). A treatment for Batten disease, Brineura costs $27,000 per biweekly infusion, resulting in an annual price tag of $702,000.

Drugmakers are also looking at one-time gene therapies to treat rare diseases. But if Spark Therapeutics’ eye disease drug Luxturna (voretigene neparvovec) is any indication, the current pricing paradigm used between manufacturers, insurers and pharmacy benefit managers will surely need amending.

Even so, Evaluate predicts orphan drug sales will continue to rise. The firm pegged a compound annual growth rate of 11.3% — roughly double the rate for non-orphan drugs — between 2018 and 2024. That would have orphan drugs comprising nearly 22% of global prescription drug sales by that latter year.

In 2017, mean orphan drug cost to patients in the U.S. was around $150,000. Alexion’s Soliris fetched the greatest revenue per patient at $501,000, followed by Revlimid at $184,000 and J&J and AbbVie’s Imbruvica (ibrutinib) at $127,000.

“In general, as the number of patients treated increased, the revenue per patient decreased,” the report found. “The strength of this correlation increased further for orphan drugs that treated fewer than 10,000 patients, confirming that there is the potential for big gains in those companies willing to invest in ultra-rare diseases.”