Out with the broad and in with the rare

Could a growing interest in rare disease on the part of venture capitalists be to the detriment of novel discoveries by up-and-coming pharmas and biotechs with regard to diseases that affect large populations of patients?

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Ah, serendipity. As we bring out DDNews’ managing editor, Lloyd Dunlap, for his first editorial in these pages, I decided to run a piece on a new report, “Transforming Rare Disease Patients’ Lives Through Innovation” (that report, which ran in our March 2015 issue along with this editorial, appears below) to fill the remaining space, since reports from industry organizations are, in my mind, certainly a form of commentary themselves.
As I pondered what to write for my own editorial this issue, I didn’t expect to be addressing a counterpoint to what one would think would be only good news—after all, who doesn’t want to see rare diseases get attention? And, besides, such treatments can be good business for pharmas and biotechs because while the patient populations might be small, the price points for rare disease therapeutics can be quite attractive.
But a sort of counterpoint I did get, when by chance I stumbled upon the very recent “Venture Funding of Therapeutic Innovation” report from the Biotechnology Industry Organization (BIO) that talks about venture funding and where it is—and isn’t—going these days. Apparently, rare diseases are getting the shine right now, and diseases that affect large populations are not.
In what BIO says is a first-of-its-kind study on venture financing broken down by disease area and novelty of research over a 10-year period from 2004 to 2013, encompassing $38 billion of venture capital into more than 1,200 U.S. drug companies that received more than 2,000 rounds of funding, “The aim was to identify funding trends for emerging drug developers within specific therapeutic areas and across varying levels of innovation,” said Dr. Cartier Esham, BIO’s executive vice president of emerging companies.
Two findings that struck me were that disease areas affecting large populations—diabetes, psychiatry, gastrointestinal, respiratory and cardiovascular—all have experienced a decline in novel drug R&D venture funding, while rare disease funding has seen a large increase over the past decade in terms of both dollars raised and number of companies funded.
In an article about the report, San Francisco Business Times reporter Ron Leuty quoted OncoMed Pharmaceuticals Inc. President and CEO Paul Hastings as saying venture capitalists are more likely to invest in companies developing orphan disease treatments or cancer treatments because those disease areas have well-tested pathways and are relative easy targets compared to more general ailments that require very large clinical trials and longitudinal studies.
It would be easy to look at Hastings’ statement and say, “Of course; makes sense.” But he’s not simply talking about a longstanding venture capital preference. Because what BIO has tracked is a trend toward the niche and away from the broad.
This is certainly not alarming to me by any means. While venture capital is important in any market, and perhaps pharma and biotech more than many, there are plenty of established companies working on plenty of treatments and vaccines for common diseases. And even with flat funding and cuts over the years in government support and financial challenges for foundations, academic institutions get many grants for innovative life-sciences research.
But at the same time, one of the other things we’ve seen over recent years is a tightening of the belts even in Big Pharma, and a resulting constriction of R&D spending. So, increasingly, some of the most interesting innovation we see in big companies comes by way of smaller companies they’ve partnered with or acquired—companies that might not have existed at all without venture funding.
No, I’m not worried about a significant decline in therapeutics for large-population disease areas. But I do feel a twinge of regret that for the “attractive” needs of the few, the more “banal” needs of the many may miss out on some innovative treatment solutions in the coming years.

A decade of notable progress against rare diseases
WASHINGTON, D.C.—The Pharmaceutical Research and Manufacturers of America (PhRMA) released on Feb. 25 a new report, “A Decade of Innovation in Rare Diseases,” to document the significant progress made in the last 10 years in understanding a broad range of rare diseases and translating this knowledge into groundbreaking therapies for a variety of patient populations.
The report illustrates that more than 230 new medicines to treat rare, or orphan, diseases were approved by the U.S. Food and Drug Administration in the last decade, and there are currently more than 450 orphan drugs in development.
It also explores significant treatment advances seen over the past decade in five rare diseases which have led to improvements in patient survival and quality of life: chronic myelogenous leukemia (CML), chronic lymphocytic leukemia (CLL), pulmonary arterial hypertension (PAH), hereditary angioedema (HAE) and cystic fibrosis (CF). Furthermore, the report spotlights additional rare conditions where major milestones transformed treatment—including several in which first-ever treatment options have become available to patients.
Key findings include:
  • Targeted therapies can now effectively treat many of the recently identified mutated forms of CML and allow for treatment plans tailored to each patient’s particular genetic profile.
  • Novel targeted therapies treat the root cause of CLL, resulting in lasting remissions and new treatment options for even the sickest patients.
  • New treatments go beyond symptom management to treat the underlying cause of PAH, allowing patients to maintain active lifestyles with reduced risk of serious heart events.
  • New discoveries in the underlying cause of HAE have led to breakthroughs in both preventative and acute treatment options for patients, including those targeting the root cause of the disease.
  • CF patients now have new treatment options allowing for better management of symptoms, as well as a new option which allows many to target the underlying cause of their disease. If mortality rates continue to decline, patients may now hope to live into their 50s.
“Rare diseases are one of the most scientifically complex health challenges we face,” said PhRMA President and CEO John J. Castellani. “Over the last 10 years, biopharmaceutical researchers have leveraged cutting-edge technologies and dramatically expanded our understanding of rare diseases to develop new therapies for chronic myelogenous leukemia, cystic fibrosis and other diseases which often impact very small patient populations. We continue to work toward options for rare disease patients, where there may currently be few or no treatments available.”
The new report was released ahead of Rare Disease Day, which was celebrated Feb. 28, 2015. The day is an opportunity for the international rare disease community—including academia, patient advocacy groups, pharmaceutical and biotechnology companies, research and regulatory government agencies—to collectively educate and raise awareness about rare diseases and their impact on the lives of patients and their families.
Thirty million Americans, about one in 10, and an estimated 350 million people worldwide are currently living with a rare disease, which is defined as a condition that affects fewer than 200,000 people. There are currently 7,000 known rare diseases, and approximately 80 percent are caused by genetic abnormalities. However, despite incredible progress made against rare disease in recent years, only 5 percent have available treatment options.
While there remains a significant need to develop new medicines for patients, this is a challenging endeavor, PhRMA notes. Rare diseases are often complex, and the underlying biological mechanisms that cause them are not always well understood. Nevertheless, researchers continue to build on recent breakthroughs and are dedicated to bringing new medicines to patients. With more than 450 medicines in development to treat rare disease, the promise has never been greater for patients.

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