Studying such real-world evidence offers manufacturers a powerful
tool to prove the value of their drugs - something Roche <ROG.S>
aims to leverage, for example, with last month's $2 billion purchase
of Flatiron Health.
Real-world evidence involves collecting data outside traditional
randomized clinical trials, the current gold standard for judging
medicines, and interest in the field is ballooning.
Half of the world's 1,800 clinical studies involving real-world or
real-life data since 2006 have been started in the last three years,
with a record 300 last year, according to a Reuters analysis of the
U.S. National Institutes of Health's clinicaltrials.gov website.
Hot areas for such studies include cancer, heart disease and
respiratory disorders.
Historically, it has been hard to get a handle on how drugs work in
routine clinical practice but the rise of electronic medical
records, databases of insurance claims, fitness wearables and even
social media now offers a wealth of new data.
The ability to capture the experience of real-world patients, who
represent a wider sample of society than the relatively narrow
selection enrolled into traditional trials, is increasingly useful
as medicine becomes more personalized.
However it also opens a new front in the debate about corporate
access to personal data at a time when tech giants Apple <AAPL.O>,
Amazon <AMZN.O> and Google's parent Alphabet <GOOGL.O> are seeking
to carve out a healthcare niche.
Some campaigners and academics worry such data will be used
primarily as a commercial tool by drugmakers and may intrude upon
patients' privacy.
DRUGMAKERS DELVE
Learning from the experience of millions of patients provides
granularity and is especially important in a disease like cancer,
where doctors want to know if there is a greater benefit from using
a certain drug in patients with highly specific tumor
characteristics.
In the case of the Flatiron deal, Roche is acquiring a firm working
with 265 U.S. community cancer clinics and six major academic
research centers, making it a leading curator of oncology evidence.
Roche, which already owns 12.6 percent of Flatiron, will pay $1.9
billion for the rest.
But interest in such real-world data goes far beyond cancer.
All the world's major drug companies now have departments focused on
the use of real-world data across multiple diseases and several have
completed scientific studies using the information to delve into key
areas addressed by their drugs.
They include diabetes studies by AstraZeneca <AZN.L> and Sanofi <SASY.PA>,
joint research by Pfizer <PFE.N> and Bristol-Myers Squibb <BMY.N>
into stroke prevention, and a Takeda Pharmaceutical <4502.T> project
in bowel disease.
"It's getting more expensive to do traditional clinical trial
research, so industry is looking at ways it can achieve similar
goals using routinely collected data," said Paul Taylor, a health
informatics expert at University College London.
"The thing that has made all this possible is the increasing
digitization of health records."
Significantly, the world's regulators are taking notice.
U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb
- the gatekeeper to the world's biggest pharmaceutical market -
believes more widespread use of real-world evidence (RWE) could cut
drug development costs and help doctors make better medical choices.
Under the 21st Century Cures Act, the FDA has been directed to
evaluate the expanded use of RWE. "As the breadth and reliability of
RWE increases, so do opportunities for FDA to also make use of this
information," Gottlieb said in a speech last September.
[to top of second column] |
The European Medicines Agency, too, is studying ways to use RWE in
its decision making.
(GRAPHIC: Drug research gets real - http://tmsnrt.rs/2otRk8g)
WHOSE DATA IS IT ANYWAY?
But the growth of real-world evidence also raises questions about
data access and patient privacy, as Britain's National Health
Service (NHS) - a uniquely comprehensive source of healthcare data -
has found to its cost.
An ambitious scheme to pool anonymized NHS patient data for both
academic and commercial use had to be scrapped in 2016 after
protests from both patients and doctors.
And last year a British hospital trust was rapped by the Information
Commissioner's Office for misusing data, after it passed on personal
information of around 1.6 million patients to
artificial-intelligence firm Google DeepMind.
Sam Smith, a campaigner for medical data privacy at Britain's
MedConfidential, is concerned drugmakers' RWE studies are just a
cover for marketing. "How much of this is really for scientific
discovery and how much is it about boosting profits by getting one
product used instead of another?"
Some academics also worry RWE studies could be susceptible to "data
dredging", where multiple analyses are conducted until one gives the
hoped-for result.
AstraZeneca's head of innovative medicines Mene Pangalos, whose
company has struck several deals with tech start-ups and patient
groups to gather real-world data, acknowledges ensuring privacy and
scientific rigor is a challenge.
"It's a real problem but I don't think it's insurmountable," he told
Reuters.
"As people get more comfortable with real-world evidence studies I
think it will be much more widely used. I would like to see a world
where real-world data can be used to help change drug labels and be
used much more aggressively than it is today."
NEXT FRONTIER
Roche Chief Executive Severin Schwan believes data is the next
frontier for drugmakers and he is betting that the Swiss group's
leadership in both cancer medicine and diagnostics will put it in
pole position.
"There's an opportunity for us to have a strategic advantage by
bringing together diagnostics and pharma with data management. This
triangle is almost impossible for anybody else to copy," he said in
a December interview.
Still, even Roche cannot work alone in this new world.
"You can have a big debate about whose data it is - the patient's,
the government's, the insurer's - but one thing for sure is the
pharmaceutical company does not own it. So there's no choice but to
do partnerships," Schwan said.
With Apple's latest iPhone update including a new feature allowing
users to view their medical records, Amazon teaming with Berkshire
Hathaway <BRKa.N> and JPMorgan Chase <JPM.N> on a new healthcare
company, and numerous start-ups flooding in, the partnering
opportunities are plentiful.
"You are going to see more deals," said Susan Garfield, a partner in
EY's life sciences advisory practice. "Data already has tremendous
value and it is going to have increasing value in future. The
question is who is going to own and capture it."
(Reporting by Ben Hirschler; Editing by Pravin Char)
[© 2018 Thomson Reuters. All rights
reserved.] Copyright 2018 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed.
Thompson Reuters is solely responsible for this content. |