Digitized Health Opens RWE Floodgates. Can Artificial Intelligence Harness the Power?

Artificial intelligence is creating another new frontier in real-world evidence gathering and analysis. AI's algorithms can approximate-and maybe even surpass-human cognition and judgment in the analysis of complex medical data. Sure, humans will sign off, but AI software will do the heavy lifting.

To Be Continued:The Search for a Cure for HIV.

Effective treatment has helped curb the epidemic, but practical cures have been elusive. Now being tested: a "sterilizing cure" that eradicates HIV from the body, and a "functional cure" that effectively reduces the viral load so it cannot be transmitted or progress to AIDS.

Why Biosimilars Can Never Be Identical To Originators-and Why They Don't Need To Be.

The FDA's approval criteria require a biosimilar to be highly similar to its original biologic and show "no clinically meaningful differences." But mere similarity is not close enough for many clinicians and some patients; it makes biosimilars seem riskier than the original biologics.

Show Us (the U.S.) the Savings.

Companies with originator biologics on the market have used a variety of regulatory and legal tricks to fend off biosimilars and keep the revenue generated by their products pouring in. The most common approach has been for the "originators" to take legal action to assert or defend the patents attached to their products.

Rumble Over Drug Rebates As Pharma, Payers Duke It Out.

Pharma says rebates are a primary driver of high drug prices because drug companies raise prices in response to payers' demands for rebates in exchange for preferred placement on their formularies. PBMs and payers object. They say the rebates they get from drugmakers are largely passed on to health plans and patients and save consumers billions of dollars in the form of lower premiums and copays.

Dupixent, a New Entrant In the Asthma Lists.

Sanofi and Regeneron's Dupixent (dupilumab)-which is already approved for atopic dermatitis-has an FDA action date of October 20 for its asthma indication. It will join Nucala, (mepolizumab), Cinqair (reslizumab), and Fasenra (benralizumab) as a monoclonal antibody approved as a treatment for the type 2 inflammation phenotype in severe asthma.

In Cardiac Imaging Things Are Looking Up for CTA.

But the adoption of new technology is rarely smooth; learning curves create jagged edges. The gee-whiz factor of technology can easily get ahead of true utility and effectiveness. And, of course, there's the expense. Hospitals and other providers need to be ready to plow millions in new machines.

Aimovig for Migraine Prevention: The New Kid May Have Trouble Fitting in.

Aimovig (erenumab-aooe), codeveloped by Amgen and Novartis, has been recently approved for the prevention of migraines. Its mechanism of action is different than other migraine medications. But perhaps more interestingly, it is the first drug recently developed specifically for migraine prevention.

A Reality Check for Checkpoint Inhibitors.

They take the brakes off the immune system so it attacks cancer cells. But many people don't respond to checkpoint inhibitors, so researchers are looking for ways to defeat the resistance.

Take a Bow, Pharma, for the Hepatitis C Drugs.

The direct-acting virals revolutionized the treatment of hepatitis C. They also ushered turbocharged pricing. At least patients-and society-got a major health benefit in return.

How RWE Is Becoming the Real Deal.

The randomized controlled trial reigns supreme, but the FDA is working on ways to incorporate real-world evidence into its approval processes.

Real-World Evidence Not Quite Believable Enough.

In theory, this approach could help untangle some knotty cost and quality concerns about medications as they move from clinical trials and into clinical use. But there's a credibility issue: The perception that much of the research is biased and that the studies are designed so the results serve the interests of the sponsors.

Is CAR-T Really Putting Us On Road to Gene Therapy?

Some say gene editing platforms like CRISPR are a truer version of gene therapy because they are designed to home in on a particular genomic location.

Why marijuana is headed for the mainstream.

The credibility of cannabis as a source of a legitimate pharmaceutical ingredient in prescription medications took a major step forward in 2018 when the FDA approved Epidiolex (cannabidiol) for two types of severe seizures. Epidiolex was a stellar candidate for approval. It reduced convulsive seizures by about 40% and has a good safety profile.

Opioids, Part 2: Novel Therapies Could Help Wean Americans Off Opioids.

Opioids remain the go-to products because they target the mu opioid receptor, which has been shown to be the most effective pathway to reduce pain. The holy grail for drug developers is an agent that stifles pain without producing the euphoria and addiction of opioids.

Biosimilars Shake Off Doubters, Go on a Roll.

Seven biosimilars have crossed the FDA-approval finish line. The FDA has largely held to the abbreviated pathway it laid out originally. The big change is that the rest of the world is starting to understand it. Approvals are based upon "the totality of evidence," "analytic similarity," and clinical performance with much less emphasis placed on phase 3 trials.

Otezla, Warts and All, Racks Up Sales and Eyes Blockbuster Status.

Otezla-the generic name is apremilast-also exploited a new mechanism of action as the first inhibitor of phosphodiesterase 4 (PDE4) that results in increased expression of both anti-inflammatory proteins and reduced expression of their pro-inflammatory counterparts.

Regimen Change: Gilead's TAF Drugs Toppling TDFs in HIV Treatment.

Truvada is getting a new lease on life as a preventive agent. It is the only drug approved to prevent HIV infections, and Truvada is the key pharmaceutical component of pre-exposure prophylaxis, which is aimed at preventing, rather than treating, HIV infection and transmission.

After a Big Entrance, It's Just So-So for Entresto.

Some industry observers wonder why the wunderkind hasn't done more wonderfully on the market. As a brand name drug, Entresto costs substantially more than the generic ACE inhibitors and ARBs. GoodRx reports the average cash price for uninsured patients for Entresto is $504 per month, compared with $44 for enalapril.

Drugs for Diabetes: It's No Longer Just About Controlling Blood Sugar.

The link between diabetes and cardiovascular disease has been clear for decades, but until 2015 no clinical trial had convincingly demonstrated a link between medications that lower blood glucose and cardiovascular risks. Enter SGKT2 inhibitors and GLP-1 agonists.