New Industry Report Finds Most Orphan Drugs Aren't Successful

Many syringes pointed into one vial

Orphan drugs underline the critical issues of drug prices, rare diseases and unmet medical need. The Orphan Drug Act of 1983 was created to encourage drug companies to develop drugs for rare or ultra-rare diseases. The National Institutes of Health (NIH) defines rare diseases as afflicting fewer than 200,000 individuals. 

Generally speaking, the Orphan Drug Act has worked. More companies are willing to invest the millions of dollars needed to develop a drug that has a very small market. Payers are generally willing to pay the usually very high prices for the drugs because they’re the only treatment available, although there are increasing restrictions or qualifying conditions on their payment. They also often find that the high costs of the drugs are less expensive than paying for the long-term care, patient visits and hospitalizations associated with these diseases.

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Trinity Partners, based in Waltham, Mass., released an annual report today that ranked dozens of new drugs approved in 2015 by the U.S. Food and Drug Administration (FDA) based on therapeutic benefits, R&D costs, and commercial sales. And for the second year, Trinity found that orphan disease drugs generally haven’t done all that well at launch and often continued to stumble after three years of sales.

Trinity’s report indicated that the most successful drugs were for non-orphan diseases like cancer, HIV and psoriasis. 

There were 57 unique drug and biologic approvals in 2015, most of which received one or more expedited review designations. Oncology was the area with the greatest number of approvals—30 percent in 2015. The highest-performing drugs from the 2015 approvals were Ibrance (palbociclib) for breast cancer, Darzalex (daratumumab) for multiple myeloma, and Genvoya (elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide), for HIV.

The report offers some analysis on the success of oncology products, particularly in terms of developing cancer drugs for multiple indications or for multiple lines within an indication. These, the analysts found, tended to be more commercially durable, stating, “For oncology products, moving up in line of therapy leads to higher commercial success, as seen with Darzalex, which received a fourth-line indication at launch, but has since added four additional indications within multiple myeloma.”

The report does point out that ultra-orphan products like Kanuma for lysosomal acid lipase deficiency (LAL-D) and Unituxin for high-risk neuroblastoma, tended to have a lower commercial performance. Some did well commercially, such as Orkambi for cystic fibrosis and Strensiq for hypophosphatasia.

The report looked at 2016 as well, noting that approvals dropped that year. It wrote, “Innovation in oncology continued an upward trend, with several novel breakthrough therapies.”

However, it went on, “Nearly half of new drugs approved in 2016 were indicated for orphan diseases highlighting that investing in this space remains a strategic choice for many companies. Notable orphan drugs include the controversial Exondys 51 (Duchenne muscular dystrophy), Spinraza (spinal muscular atrophy), and Ocaliva (primary biliary cirrhosis). Analysis of these therapies will help to fine tune our assessment of the commercial potential of orphan drugs relative to their therapeutic value.”

Also, the report points out that the top 10 most expensive drugs approved from 2014 and 2016 were for orphan diseases. And convincing payers to cover the drugs isn’t always that simple. 

The report also points out that increased pricing pressures, biosimilar competition, and even overall greater competition is forcing drug companies to be even more strategic. That’s consistent with reports that big biopharma is actively killing off more drug programs to focus finite resources on the drugs with the best chances.

But it’s not really bleak. The report notes, “Although the industry faces immense challenges, substantial room for growth remains as demonstrated by this report; therapies that provide compelling clinical benefit relative to the standard of care can achieve commercial success even in crowded markets.”

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