Best of Biopharma: Here are the top stories of 2021

As the calendar page turns, we review the top ten biopharma stories of 2021, as presented by BioPharma Dive and edited for quick reading. Here’s to a pandemic-free 2022, sooner or later.

The world has long awaited an Alzheimer’s drug, but the FDA approval of Biogen’s Aduhelm in June may have raised as many questions as it answered. It appeared to be a lucrative and much-needed treatment, but a high price and controversy over whether the drug was effective enough cast concerns on the drug's launch. 

The FDA based its approval on a large study that found patients with early Alzheimer's who were given a high dose of the drug for enough time seemed to fare better on a cognitive test than those who were given a placebo. However, the those results have been questioned, because another study found the opposite to be true, with the placebo outperforming the drug. The drug ended up getting conditional approval, with more tests to be performed. Biogen priced the drug at about $56,000 a year, but has recently reduced the price. 

In October the FDA cleared Pfizer's COVID-19 vaccine for children, moving the US to the next stage of the vaccine rollout. It brought about access to COVID-19 vaccines for 28 million children between 5 and 11 years old. 

According to FDA's acting commissioner, Janet Woodcock "Vaccinating younger children against COVID-19 will bring us closer to returning to a sense of normalcy."

Still, broad use among children was a heavily debated subject. In spite of a near-unanimous vote in favor of authorization, some panelists were unsure whether healthy young children need to be vaccinated.  

The FDA, aiming to curb another COVID-19 surge, cleared the Pfizer and Moderna boosters for all adults in November. The agency cleared a third shot of Pfizer and BioNTech's or Moderna's vaccines for adults who had completed a two-dose regimen of either at least six months ago. Advisers to the CDC followed with a similar recommendation. The FDA's review took only ten days.

State public health officials urged the panel to recommend boosters widely, arguing that the current risk- and age-based guidelines caused confusion. The eligibility criteria are an "obstacle to uptake of boosters in pursuit of precision," said Nirav Shah, president of the Association of State and Territorial Health Officials at the CDC meeting. By then about 30 million Americans had already received booster doses.

COVID-19 pills from Pfizer and Merck were authorized by the FDA in major pandemic milestone. Paxlovid and molnupiravir, the first oral treatments for COVID-19, could be especially valuable new tools as the fast-spreading omicron variant fuels a sharp surge in cases. 

Paxlovid will be available for adults and children over 12 with mild or moderate cases of COVID-19 but who are at high risk of more severe symptoms. Molnupiravir's clearance is limited to adults over the age of 18 and comes with the important caveat that it should only be used to treat people for whom other options, like Paxlovid, aren't available or appropriate.

The Biden administration's call to waive patent rights for coronavirus vaccines triggered great opposition from drug companies and pharma industry groups, which said the move will discourage future drug research and do little to increase supply. Activists argued that a waiver was needed to begin removing hurdles standing in the way of manufacturing vaccines in Asia, Africa and South America.

A patent waiver may not do much to speed the availability of vaccines. Manufacturing vaccines that use messenger RNA is a relatively new, complex and resource-intensive process. There may not be sufficient production capacity for others to boost supply.

President Joe Biden nominated Robert Califf to return as the head of the FDA. Califf, a cardiologist and  researcher, served as FDA commissioner at the end of the Obama administration. After leaving government, he advised Google and its health spinoff Verily Life Sciences, taking a job leading health policy there in November 2019.

If confirmed, Califf would take over an agency heavily stressed by the pandemic and the resulting workload of reviewing new tests, vaccines and drugs for COVID-19. FDA officials have regularly cited the pandemic's burden on agency staff and a number of veteran reviewers in several departments have recently announced plans to retire or depart.

In April the FDA faced a tough choice after a panel backed speedy cancer drug approvals. A group of advisers to the FDA recommended maintaining four of six recent cancer immunotherapy approvals despite a lack of evidence the medicines help patients live longer. The meeting showcased the tradeoffs of clearing a drug before it is fully proven, as well as the scrutiny regulators face when those decisions appear to backfire. The advisory committee, made up of oncologists and patient representatives, struggled at times, not wanting to take treatment options away from cancer patients who have few. 

Each of the approvals debated at the meeting were cleared under an accelerated approval program that was enacted in 1992 to get drugs for diseases with few, if any, treatment options onto market faster. The approvals are made based on surrogate markers that are likely, but not guaranteed, to predict a benefit.

The Congressional Democrats' modified drug pricing plan may not lower spending on pharmaceuticals by nearly as much as their most ambitious proposal would have, but it could have a substantial effect on some of the industry's top-selling products. The new bill would grant the federal government power to negotiate drug prices but represents a significant compromise as its scales back the number of products eligible for negotiation to 20 older drugs. 

Under the new proposal, these older drugs, defined as those no longer protected by regulatory exclusivity and lacking generic competitors, would be subject to price negotiation with the Centers for Medicare and Medicaid Services. The legislation would limit eligible products to small molecule drugs that have been on the market for at least nine years and biologic drugs that have been available for at least 12 years. Small molecule drugs are typically given as pills, while biologics are infused or injected.

Johnson&Johnson, the world's largest drugmaker, plans to split in two. It will separate its consumer health division, which sells well-known brands like Tylenol, Listerine and Band-Aids, into a new publicly traded company. J&J will retain its prescription drug and medical device units and keep in place its current leadership plan. 

CEO Alex Gorsky will step down at the end of the year and become executive chairman. Joaquin Duato, who heads the company's executive committee, will succeed Gorsky and lead J&J after the split. J&J expects to complete the separation in the next 18 to 24 months.