RARE Daily

Follow the Money

March 20, 2019

In the film All the President’s Men, it was in a darkened garage from behind the glow of a cigarette that the Watergate tipster Deep Throat (Hal Holbrook) advised The Washington Post reporter Bob Woodward (Robert Redford) in hushed tones to “follow the money.”

It’s a phrase that’s lived on in newsrooms and political scandals ever since.

“Follow the money” is also good advice for people who want to know where the pharmaceutical industry is betting its future lies and what the hopes are for seeing needed treatments for rare diseases come to market.  

Drugs to Watch 2019, a new report from the life sciences industry data and analysis provider Cortellis, offers encouragement for the rare disease community. It forecasts that three of seven drugs that are expected to enter the market in 2019 and achieve blockbuster status by 2023 are for rare disease indications. A “blockbuster,” drug in industry lingo, is a drug that generates sales of more than $1 billion per year.

Also noteworthy is the fact that there’s not a single cancer therapy in the list. That’s not to suggest that the industry has lost interest in oncology, but it may be a sign that the competition among the new immunotherapies may have grown fierce, companies are focusing on expanding indications for existing therapies and targeting smaller, niche populations with new therapies.

“This year’s Drug to Watch report demonstrates the impact of regulators’ incentives, including accelerated reviews and tax breaks, on drug development that targets rare diseases,” said Mukhtar Ahmed, president of Life Sciences at Clarivate Analytics. “It’s extremely encouraging to see how these incentives, along with discovery breakthroughs and new R&D approaches, are positively impacting patients in under-served populations. What’s more, these novel treatments demonstrate the power of collaboration across the industry, which ultimately accelerates innovation.”

The Cortellis list includes Novartis’ Zolgensma, a gene therapy that corrects the underlying genetic defect in spinal muscular atrophy, a rare and deadly muscle-wasting disease; Bluebird Bio’s LentiGlobin, a gene therapy that corrects the genetic defect that causes beta thalassemia, a rare disorder that can cause life-threatening anemia; and Alexion’s Ultomiris, a treatment for paroxysmal nocturnal hemoglobinuria, a rare and potentially fatal blood disease.

There was a time when the pharmaceutical industry took little interest in developing drugs for rare diseases because companies were chasing the next blockbuster. This often led to new drugs for common diseases that were not life-threatening and already had a number of available therapies.

Though windfall profits for drug companies and high drug prices are not generally a cause for celebration in the rare disease community, what’s noteworthy about the drugs in these expected blockbusters emerging from the class of 2019 is that six of seven of these drug target diseases that are characterized by genetic disorder or autoimmunity. That suggests the potential commercial success that comes with targeting the underlying molecular mechanism of a disease.      

With the increasing focus from payers on the value of therapies, it is pushing drugmakers to focus on drugs that deliver a significant impact on patients and deliver more reliable benefits for patients. One of the drivers toward precision medicine has been the lack of efficacy many large market drugs have on substantial portions of the patients who use them—anywhere from 38 percent to 75 percent of drugs in a class are on average ineffective, according to the Personalized Medicine Coalition.

In addition to incentives like the Orphan Drug Act and the opportunity to benefit from accelerated development pathways, smaller clinical trials, and opportunities to deliver first-in-class/best-in-class therapies, drugmakers have come to see growing benefits from pursuing rare disease indications.

The market success for rare disease therapies is good news. It validates the investments being made by Wall Street and the industry in this area and will drive further investment in developing new treatments for these conditions. That’s because investors and drug developers, like journalists, are trained to follow the money.

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