Category: Health

Big Pharma Is Mellow about FDA Obstacles to Innovation

It sometimes appears that big pharma is comfortable with the hugely expensive FDA drug approval process. Perhaps big pharma firms have learned how to navigate the process and have the resources to do so. And perhaps the process discourages disruptive innovations from small medical startups that have not learned how to navigate the process, and do not have the resources to do so. If so, then the puzzling indifference of big pharma indicated in the passages quoted below, becomes easier to understand.
(There’s a wonderful recent TV ad from big pharma supporting innovation by quoting the Dylan Thomas poem saying we should “rage, rage against the dying of the light.” If only they really meant it.)

(p. A13) In recent years, the arrival of breakthrough drugs for everything from cancer to rare diseases has led to a surge in the number of patients wanting early access to treatments. The pleas — sometimes driven by viral social media campaigns — have proved vexing for companies that have invested millions to get a drug to market and are wary of doing anything to jeopardize their chances.

Today, companies’ policies on granting early access to drugs are a confusing patchwork that tends to favor affluent and well-connected patients at leading medical centers, who have the resources and know-how to navigate the system.
“You have to be pretty sophisticated,” said Dr. Arthur L. Caplan, a bioethicist at New York University who has been working with companies, including Johnson & Johnson, to develop better early-access programs. But the bill passed this week, he said, “does somewhere between nothing and absolutely nothing to help you.”
The bill’s passage represented a victory for proponents of “right to try,” a campaign championed by Vice President Mike Pence and initiated by the Goldwater Institute, a libertarian think tank that favors limiting the scope of the F.D.A. At least 38 states have passed local versions of right-to-try laws, which allow patients to sidestep F.D.A. approval once they have received permission from a company.
The right-to-try measures are opposed by a broad coalition of groups, which contend the bill will not help patients and will undermine the authority of the primary regulatory agency, the F.D.A. Four former F.D.A. commissioners, including two each from Democratic and Republican administrations, oppose the bills, as do dozens of patient groups, including the American Cancer Society Cancer Action Network and the American Lung Association.
The pharmaceutical industry, while not taking a position on the issue, has been circumspect. A spokesman for its main lobbying group, the Pharmaceutical Research and Manufacturers of America, said on Friday, “We believe any legislation must truly benefit and protect patients and not disrupt the future of clinical trials, U.S. Food and Drug Administration oversight and the research and approval of new medicines.”
. . .
The F.D.A. already approves 99 percent of such applications, and the agency has streamlined the approval process. Drug companies also have many other reasons to bar access — often, companies do not have enough extra product to give to patients, or they worry that the logistical work of granting access could slow efforts to get the drug approved, when it would become available to any patient who needed it.
There is also the possibility that the drug does not work — many experimental products fail in late-stage trials.
. . .
“In our view, the F.D.A. plays a really important role,” Dr. Joanne Waldstreicher, the chief medical officer of Johnson & Johnson, said in an interview Thursday. Johnson & Johnson initiated a program in 2015 that delegates decisions about early access to a program set up by Dr. Caplan. The F.D.A., Dr. Waldstreicher said, has “information that we don’t have necessarily; they see safety and efficacy information on products that may be similar.”

For the full story, see:
KATIE THOMAS. “For Terminally Ill People, a Convoluted Procedure Just to Give Drugs a Try.” The New York Times (Saturday, March 24, 2018): A13.
(Note: ellipses added.)
(Note: the online version of the story has the date March 23, 2018, and has the title “Why Can’t Dying Patients Get the Drugs They Want?”)

On Strokes, Doctors Decide for Patients, Even When Patient’s Family Knows More

(p. D1) It was one of those findings that would change medicine, Dr. Christopher Lewandowski thought.
For years, doctors had tried — and failed — to find a treatment that would preserve the brains of stroke patients. The task was beginning to seem hopeless: Once a clot blocked a blood vessel supplying the brain, its cells quickly began to die. Patients and their families could only pray that the damage would not be too extensive.
But then a large federal clinical trial proved that a so-called clot-buster drug, tissue plasminogen activator (T.P.A.), could prevent brain injury after a stroke by opening up the blocked vessel. Dr. Lewandowski, an emergency medicine physician at Henry Ford Health System in Detroit and the trial’s principal investigator, was ecstatic.
“We felt the data was so strong we didn’t have to explain it” in the published report, he said.
He was wrong. That groundbreaking clinical trial concluded 22 years ago, yet Dr. Lewandowski and others are still trying to explain the data to a powerful contingent of doubters.
The skeptics teach medical students that T.P.A.is dangerous, causing brain hemorrhages, and that the studies that found a benefit were deeply flawed. Better to just let a stroke run its course, they say.
It’s a perspective with real-world consequences. Close to 700,000 patients have strokes caused by blood clots each year and could be helped by T.P.A. Yet up to 30 percent of stroke victims who arrive at hospitals on time and are perfect candidates for the clot-buster do not receive it.
The result: paralysis and muscle weakness; impaired cognition, speech or vision; emotional and behavioral dysfunction; and many other permanent neurological injuries.
Stroke treatment guidelines issued by the American Heart Association and the American Stroke Association strongly endorse T.P.A. for patients after they’ve been properly evaluated. But treatment must start within three hours (in some cases, four(p. D4)-and-a-half hours) of the stroke’s onset, and the sooner, the better.
A number of medical societies also endorse the treatment as highly effective in reducing disability. The drug can cause or exacerbate cerebral hemorrhage, or bleeding in the brain — a real risk. But in most stroke patients it prevents brain injury, and in any event, rates of cerebral hemorrhage have declined as doctors have gained experience over the years.
. . .
About a decade ago, Dr. Lewandowski was at work when he got a call that his father had had a stroke — his right side was paralyzed. But his father had gotten to the hospital within 45 minutes, well inside the window to receive T.P.A.
Dr. Lewandowski told his mother to make the family’s wishes very clear. They wanted the emergency room doctor to give the clot-buster to his dad. The doctor refused.
“He told my mom that he doesn’t believe in the drug and he is not giving it. He doesn’t care who I am,” Dr. Lewandowski said.
“I got in my car and drove 400 miles to the hospital,” he recalled. But by the time he got there, it was too late. The treatment window had closed.
His father had a facial droop and slurred speech. His right arm and right leg flopped about uselessly. His stroke scale was 7, moderately disabling, but he survived for a few more years.
“It was very difficult for me personally,” Dr. Lewandowski recalled. “I had spent so much of my professional life working on this treatment. It actually worked.”
“I felt like I had let my dad down.”

For the full story, see:

GINA KOLATA. “A Stroke Treatment Mired in Controversy.” The New York Times (Tuesday, March 27, 2018): D1 & D4.
(Note: ellipsis added.)
(Note: the online version of the story has the date MARCH 26, 2018, and has the title “For Many Strokes, There’s an Effective Treatment. Why Aren’t Some Doctors Offering It?”)

Double-Blind Study Shows Little Heart Benefit from Stents

(p. B3) A new study raised questions about the benefits of a relatively common procedure for heart patients–implanting tiny devices that prop open clogged arteries to relieve chest pain.
The 200-patient study conducted by U.K. researchers found that patients with stable chest pain, or angina, who received stent devices experienced no significant improvement in exercise time on a treadmill, compared with similar patients who received no stents during sham procedures.
All patients had received intensive treatment with heart drugs for six weeks before the real or fake procedures.
“Symptoms didn’t improve as much as expected” in the patients who received stents, Rasha Al-Lamee, an interventional cardiologist at Imperial College London and one of the study’s lead investigators, said in an interview. She presented results of the study at the Transcatheter Cardiovascular Therapeutics medical conference in Denver; results were simultaneously published online Thursday [November 2, 2017] by The Lancet.

For the full story, see:
Peter Loftus. “Study Questions Some Stent Use.” The Wall Street Journal (Friday, Nov. 3, 2017): B3.
(Note: bracketed date added.)
(Note: the online version of the story has the date Nov. 2, 2017, and has the title “Study Raises Questions About Stents in Some Heart Patients.”)

The Lancet article, summarized above, is:
Al-Lamee, Rasha, David Thompson, Hakim-Moulay Dehbi, Sayan Sen, Kare Tang, John Davies, Thomas Keeble, Michael Mielewczik, Raffi Kaprielian, Iqbal S. Malik, Sukhjinder S. Nijjer, Ricardo Petraco, Christopher Cook, Yousif Ahmad, James Howard, Christopher Baker, Andrew Sharp, Robert Gerber, Suneel Talwar, Ravi Assomull, Jamil Mayet, Roland Wensel, David Collier, Matthew Shun-Shin, Simon A. Thom, Justin E. Davies, Darrel P. Francis, Rasha Al-Lamee, David Thompson, Sayan Sen, Kare Tang, John Davies, Thomas Keeble, Raffi Kaprielian, Iqbal S. Malik, Sukhjinder S. Nijjer, Ricardo Petraco, Christopher Cook, Yousif Ahmad, James Howard, Matthew Shun-Shin, Amarjit Sethi, Christopher Baker, Andrew Sharp, Punit Ramrakha, Robert Gerber, Suneel Talwar, Ravi Assomull, Rodney Foale, Jamil Mayet, Roland Wensel, Simon A. Thom, Justin E. Davies, Darrel P. Francis, Ramzi Khamis, Nearchos Hadjiloizou, Masood Khan, Jaspal Kooner, Michael Bellamy, Ghada Mikhail, Piers Clifford, Peter O’Kane, Terry Levy, and Rosie Swallow. “Percutaneous Coronary Intervention in Stable Angina (ORBITA): A Double-Blind, Randomised Controlled Trial.” The Lancet 391, no. 10115 (Jan. 6, 2018): 31-40.

Patients Lower Blood Pressure Best When It Is Self-Monitored

(p. D4) The most effective way to monitor blood pressure may be to do it yourself.
British researchers randomly assigned 1,003 patients with hypertension to one of three groups.
. . .
The study was published in Lancet.
“People who monitor their own blood pressure and share the readings with their physician get better control,” said the lead author, Dr. Richard J. McManus, a professor of primary care at the University of Oxford. “Seventy-five million Americans have hypertension. If a good proportion of those self-monitored, it would lead to a big reduction in stroke.”

For the full story, see:
NICHOLAS BAKALAR. “The Best Way to Monitor Your Blood Pressure? Do It Yourself.” The New York Times (Tuesday, MARCH 13, 2018): D4.
(Note: ellipsis added.)
(Note: the online version of the story has the date MARCH 6 [sic], 2018, and has the title “The Best Way to Monitor Your Blood Pressure? Do It Yourself.”)

The Lancet study summarized above, is:
McManus, Richard J., Jonathan Mant, Marloes Franssen, Alecia Nickless, Claire Schwartz, James Hodgkinson, Peter Bradburn, Andrew Farmer, Sabrina Grant, Sheila M. Greenfield, Carl Heneghan, Susan Jowett, Una Martin, Siobhan Milner, Mark Monahan, Sam Mort, Emma Ogburn, Rafael Perera-Salazar, Syed Ahmar Shah, Ly-Mee Yu, Lionel Tarassenko, F. D. Richard Hobbs, Brendan Bradley, Chris Lovekin, David Judge, Luis Castello, Maureen Dawson, Rebecca Brice, Bethany Dunbabin, Sophie Maslen, Heather Rutter, Mary Norris, Lauren French, Michael Loynd, Pippa Whitbread, Luisa Saldana Ortaga, Irene Noel, Karen Madronal, Julie Timmins, Peter Bradburn, Lucy Hughes, Beth Hinks, Sheila Bailey, Sue Read, Andrea Weston, Somi Spannuth, Sue Maiden, Makiko Chermahini, Ann McDonald, Shelina Rajan, Sue Allen, Brenda Deboys, Kim Fell, Jenny Johnson, Helen Jung, Rachel Lister, Ruth Osborne, Amy Secker, Irene Qasim, Kirsty William, Abi Harris, Susan Zhao, Elaine Butcher, Pauline Darbyshire, Sarah Joshi, Jon Davies, Claire Talbot, Eleanor Hoverd, Linda Field, Tracey Adcock, Julia Rooney, Nina Cooter, Aaron Butler, Naomi Allen, Maria Abdul-Wahab, Kathryn McNicholas, Lara Peniket, Kate Dodd, Julie Mugurza, Richard Baskerville, Rakshan Syed, Clare Bailey, Jill Adams, Paul Uglow, Neil Townsend, Alison Macleod, Charlotte Hawkins, Suparna Behura, Jonathan Crawshaw, Robin Fox, Waleed Doski, Martin Aylward, Christine A’Court, David Rapley, Jo Walsh, Paul Batra, Ana Seoane, Sluti Mukherjee, Jonathan Dixon, Peter Arthur, Karen Sutcliffe, Costas Paschallides, Richard Woof, Peter Winfrey, Matthew Clark, Roya Kamali, Paul Thomas, David Ebbs, Liz Mather, Andre Beattie, Karim Ladha, Larisa Smondulak, Surinder Jemahl, Peter Hickson, Liam Stevens, Tony Crockett, David Shukla, Ian Binnian, Paul Vinson, Nigel DeKare-Silver, Ramila Patel, Ivor Singh, Louise Lumley, Glennis Williams, Mark Webb, Jack Bambrough, Neetul Shah, Hergeven Dosanjh, Frank Spannuth, Carolyn Paul, Jude Ganesegaram, Laurie Pike, Vijaysundari Maheswaran, Farah Paruk, Stephen Ford, Vineeta Verma, Kate Milne, Farhana Lockhat, Jennifer Ferguson, Anne-Marie Quirk, Hugo Wilson, David Copping, Sam Bajallan, Simria Tanvir, Faheem Khan, Tom Alderson, Amar Ali, Richard Young, Umesh Chauhan, Lindsey Crockett, Louise McGovern, Claire Cubitt, Simon Weatherill, Abdul Tabassum, Philip Saunders, Naresh Chauhan, Samantha Johnson, Jo Walsh, Inderjit Marok, Rajiv Sharma, William Lumb, John Tweedale, Ian Smith, Lawrence Miller, Tanveer Ahmed, Mark Sanderson, Claire Jones, Peter Stokell, Matthew J. Edwards, Andrew Askey, Jason Spencer, Kathryn Morgan, Kyle Knox, Robert Baker, Crispin Fisher, Rachel Halstead, Neil Modha, David Buckley, Catherine Stokell, John Gerald McCabe, Jennifer Taylor, Helen Nutbeam, Richard Smith, Christopher MacGregor, Sam Davies, Mark Lindsey, Simon Cartwright, Jonathan Whittle, Julie Colclough, Alison Crumbie, Nicholas Thomas, Vattakkatt Premchand, Rafia Hamid, Zishan Ali, John Ward, Philip Pinney, Stephen Thurston, and Tina Banerjee. “Efficacy of Self-Monitored Blood Pressure, with or without Telemonitoring, for Titration of Antihypertensive Medication (TASMINH4): An Unmasked Randomised Controlled Trial.” The Lancet 391, no. 10124 (March 10, 2018): 949-59.

Pursuit of Slow Hunch Pays Off with Flu Drug

(p. B3) As Americans suffer through the worst influenza outbreak in almost a decade, a Japanese drugmaker says it has developed a pill that can kill the virus within a day.
. . .
“The data that we’ve seen looks very promising,” said Martin Howell Friede, who leads the World Health Organization’s advisory on vaccines, including for influenza. “This could be a breakthrough in the way that we treat influenza.”
. . .
Shionogi scientists began researching a novel flu drug more than a decade ago, shelving almost 2,500 compounds in the process. Then, the 140-year-old Osaka company, which has created blockbuster drugs used to treat HIV and high cholesterol, had a breakthrough.
Shionogi scientists knew from their research that an anti-HIV drug the company had developed with a joint venture of Pfizer Inc. and GlaxoSmithKline Co. worked by blocking a metallic enzyme that HIV uses as a weapon to hijack human cells. They found the flu virus was also exploiting a metallic enzyme.
“So we said, ‘why don’t we build on our HIV knowledge to find a way to treat the flu?’ And we did,” said Takeki Uehara, who led the compound’s development.

For the full story, see:
Preetika Rana. “Drugmaker: Pill Kills Flu in a Day.” The Wall Street Journal (Monday, Feb. 12, 2018): B3.
(Note: ellipses added.)
(Note: the online version of the article has the date Feb. 10, 2018, and has the title “Experimental Drug Promises to Kill the Flu Virus in a Day.”)

FDA Regulations Stop Vape Shop Innovations

(p. A19) After Kimberly Manor lost her husband to lung cancer, she was inspired to make a dramatic career change. Kimberly now owns and operates Moose Jooce in Lake, Mich., a “vape shop” that sells various electronic nicotine devices. These products use battery-powered coils to vaporize liquids, with differing levels of nicotine or none at all. Thus, vapers may inhale nicotine without the tar or other harmful chemicals in tobacco smoke, since there is no tobacco and no combustion. Scientific evidence suggests this is a much safer alternative to smoking.
Ms. Manor estimates that her business has helped more than 500 people quit smoking, most of them longtime smokers in their 50s or older. Yet the Food and Drug Administration is discouraging more such enterprises. In a regulation issued in 2016 known as the “deeming rule,” the agency ordered that vaping products would be subject to the same regulations developed for the cigarette industry under the Tobacco Control Act of 2009.
The deeming rule has been devastating to businesses like Ms. Manor’s. To give just one example, vape shop owners frequently experiment by mixing new flavors for the liquid “juice.” Now, each separate creation requires its own prohibitively expensive application for FDA approval, which means that vape shops have been forced to stop innovating.

For the full commentary, see:
Todd Gaziano and Tommy Berry, “Career Civil Servants Illegitimately Rule America; Leslie Kux has never been elected or confirmed by the Senate. She’s issued nearly 200 regulations.” The Wall Street Journal (Thursday, March 1, 2018): A19.
(Note: the online version of the commentary has the date Feb. 28, 2018.)

Proof of Concept for Regenerating Limbs and Internal Organs

(p. D3) Scientists have decoded the genome of the axolotl, the Mexican amphibian with a Mona Lisa smile. It has 32 billion base pairs, which makes it ten times the size of the human genome, and the largest genome ever sequenced.
The axolotl, endangered in the wild, has been bred in laboratories and studied for more than 150 years. It has the remarkable capacity to regrow amputated limbs complete with bones, muscles and nerves; to heal wounds without producing scar tissue; and even to regenerate damaged internal organs.
This salamander can heal a crushed spinal cord and have it function just like it did before it was damaged. This ability, which exists to such an extent in no other animal, makes its genes of considerable interest.
. . .
The researchers have identified some of the genes involved in regeneration, and some genes that exist only in the axolotl, but there is much work still to be done.

For the full story, see:
NICHOLAS BAKALAR. “TAKE A NUMBER; 32 Billion.” The New York Times (Tuesday, February 6, 2018): D3.
(Note: ellipsis added.)
(Note: the online version of the story has the date FEB. 1, 2018, and has the title “TAKE A NUMBER; The Smiling Axolotl Hides a Secret: A Giant Genome.”)

Environment Can Affect Which Genes Are Activated

(p. D5) In September 1944, trains in the Netherlands ground to a halt. Dutch railway workers were hoping that a strike could stop the transport of Nazi troops, helping the advancing Allied forces.
But the Allied campaign failed, and the Nazis punished the Netherlands by blocking food supplies, plunging much of the country into famine. By the time the Netherlands was liberated in May 1945, more than 20,000 people had died of starvation.
The Dutch Hunger Winter has proved unique in unexpected ways. Because it started and ended so abruptly, it has served as an unplanned experiment in human health. Pregnant women, it turns out, were uniquely vulnerable, and the children they gave birth to have been influenced by famine throughout their lives.
When they became adults, they ended up a few pounds heavier than average. In middle age, they had higher levels of triglycerides and LDL cholesterol. They also experienced higher rates of such conditions as obesity, diabetes and schizophrenia.
. . .
“How on earth can your body remember the environment it was exposed to in the womb — and remember that decades later?” wondered Bas Heijmans, a geneticist at Leiden University Medical Center in the Netherlands.
Dr. Heijmans, Dr. Lumey and their colleagues published a possible answer, or part of one, on Wednesday in the journal Science Advances. Their study suggests that the Dutch Hunger Winter silenced certain genes in unborn children — and that they’ve stayed quiet ever since.
While all cells in a person’s body share the same genes, different ones are active or silent in different cells. That program largely is locked in place before birth.
But scientists have learned that later experiences — say, exposure to a virus — can cause cells to quiet a gene or boost its activity, sometimes permanently.
The study of this long-term gene control is called epigenetics. Researchers have identified molecules that cells use to program DNA, but how those tools work isn’t entirely clear. One of the best studied is a molecular cap called a methyl group.
At millions of spots across our DNA, genes may carry a methyl group. They seem to silence genes — at least, researchers have found that silenced genes often have a collection of methyl groups lurking nearby.

For the full story, see:
Zimmer, Carl. “Dutch Genes Still Bear Scars of a Famine.” The New York Times (Tuesday, February 6, 2018): D5.
(Note: ellipsis added.)
(Note: the online version of the story has the date JAN. 31, 2018, and has the title “MATTER; The Famine Ended 70 Years Ago, but Dutch Genes Still Bear Scars.”)

FDR’s Coast Guard Denied Entry to Future Medical Visionary

(p. B12) Dr. Arno G. Motulsky, a former refugee from Nazi Germany who became a founder of medical genetics, recognizing the connection between genes and health long before mainstream medicine did, died on Jan. 17 [2018] at his home in Seattle.
. . .
“It was his vision to study how heredity could be involved in practically everything,” Dr. Francis Collins, a geneticist and the director of the National Institutes of Health, said in an interview. “The relationship between heredity and the response to drug therapy — nobody was thinking about that until he started, 60 years ago. He anticipated it decades before science made it possible to get the answers that he dreamed of.”
As technologies emerged to decode DNA, the fields that Dr. Motulsky helped originate came to the forefront of medicine, leading to improved diagnosis and treatments for a host of diseases.
. . .
Dr. Motulsky’s path to prominence began in harrowing fashion. He had been one of more than 900 Jewish refugees aboard the German liner St. Louis, which reached the Miami coast in 1939 but was turned away by the United States and sent back to Europe.
. . .
His parents tried to leave Germany with him and his younger siblings, Leah and Lothar, in 1939, before war broke out in Europe. In an account he gave to the Annual Review of Genomics and Human Genetics in 2016, Dr. Motulsky said his family had hoped to join his father’s brother in Chicago but headed for Cuba instead after hearing that a United States quota system was causing long delays in granting visas.
His father left first. His mother followed soon afterward, taking young Arno and his brother and sister with her aboard the St. Louis in Hamburg on May 13, 1939, bound for Havana. But Cuba refused to accept the refugees, as did other Caribbean countries.
“We asked to land in America, but were denied,” Dr. Motulsky said. “When we sailed close to Miami, U.S. Coast Guard cutters and planes shooed us off.”
Its passengers filled with dread, the ship headed back to Europe on June 6.
“Miraculously, a few days before we would have arrived back in Germany, four other countries — England, France, Holland and Belgium — each agreed to take one-fourth of the passengers,” Dr. Motulsky said.

For the full obituary, see:
DENISE GRADY. “Arno Motulsky, a Founder of Medical Genetics 60 Years Ago, Dies at 94.” The New York Times (Tuesday, January 30, 2018): B12.
(Note: ellipsis, and bracketed year, added.)
(Note: the online version of the obituary has the date JAN. 29, 2018, and has the title “Arno Motulsky, a Founder of Medical Genetics, Dies at 94.”)

Blobel Pursued a Slow Hunch for Over 30 Years

(p. B19) Günter Blobel, a molecular biologist who was awarded the 1999 Nobel Prize in Medicine for discovering that proteins in any living cell have virtual ZIP codes that guide them to where they can help regulate body tissues, organs and chemistry, died on Sunday [February 18, 2018] in Manhattan. He was 81.
. . .
The cause was cancer.
. . .
He spent nearly all his working life at Rockefeller University, what he regarded as the Valhalla of research.
Like many scientific advances, Dr. Blobel’s had no moment of “Eureka!” It unfolded over 30 years of painstaking, often frustrating, but occasionally thrilling investigation: a process of building on others’ work, intuitive thinking to form new hypotheses, and testing, using the results to modify his theories, and then testing and modifying again and again.
Driven to find underlying causes of diseases that were being treated for symptoms, and funded by the National Institutes of Health and the Howard Hughes Medical Institute, he successively developed five models of his original “beautiful idea.” Along the way he won many prestigious awards, some for essentially the same insights recognized later by the Nobel committee.

For the full obituary, see:
ROBERT D. McFADDEN. “Günter Blobel, Nobel Laureate Who Found Cell ‘ZIP Codes,’ Dies at 81.” The New York Times (Saturday, Feb. 20, 2018): B19.
(Note: ellipses, and bracketed date, added.)
(Note: the online version of the obituary has a date of Feb. 19, 2018.)

Regulations Threaten Precision Medicine Innovations Against Cancer

(p. A15) The federal government is threatening to limit treatment options for doctors fighting cancer.
. . .
At issue is whether reimbursements will be available to most physicians, hospitals and patients for a diagnostic technology known as next-generation sequencing. A cornerstone of the emerging field of precision medicine, NGS tests analyze molecular changes that occur in cancerous tumors and show up in biopsies.
. . .
Under the proposed policy, only one of hundreds of laboratories that currently offer NGS testing would meet all the new reimbursement requirements. The policy would in effect force clinicians and institutions to send all NGS testing to a single vendor, Foundation Medicine .
This is unfair to cancer patients. The proposal would result in a monopoly, allowing price manipulations, decreasing quality, and potentially contributing to market failure. It would turn the entire genomic-testing industry upside-down. The FDA is already unable to keep up with advances in precision medicine. Restricting access to cutting-edge molecular testing would stifle growth in precision medicine at approved testing sites nationwide. The limits could prevent desperately needed innovation, setting back progress in genomic testing and oncology by at least a decade.

For the full commentary, see:
Olivier Elemento. ”A New Regulatory Threat to Cancer Patients; Washington may impose needless limits on genetic testing.” The Wall Street Journal (Mon., Feb. 26, 2018): A15.
(Note: ellipses added.)
(Note: the online version of the commentary has the date Feb. 25, 2018.)