Bionest

Time to Embrace Decentralized Clinical Trials

A clearer roadmap for decentralized clinical trials will enable companies to better plan and more quickly advance their adoption of these strategies, which have the potential of benefiting patients and drug developers alike.

Bionest

New Strategies Against a Hard-to-Treat Killer — Pancreatic Cancer

Pancreatic cancer was the third leading cause of cancer-related death in the United States in 2018. This year, experts estimate that 56,770 new cases will be diagnosed and 45,750 deaths will be recorded. Pancreatic cancer is especially lethal because in its early stages, when the disease is easiest to treat, patients are usually asymptomatic. The disease...

Bionest

Zolgensma Launch “On Track,” but Significant Challenges Remain

On July 18, Novartis told analysts its Zolgensma launch was “on track.” Despite the gene therapy’s $2.1 million price tag, the company states the launch is “one of the most successful launches from an access standpoint in rare diseases.” Novartis said it had reached coverage deals with more than 20 commercial insurance plans, covering approximately 40%...

Bionest

New Excitement About Antibody-Drug Conjugates in Cancer

The ability to target malignant cells without damage to surrounding healthy ones has been a long-sought goal in cancer drug development. Antibody-drug conjugates (ADCs) have been investigated as one such solution aimed at delivering more targeted therapies. ADCs — an approach first envisioned by Paul Ehrlich in the early 20th century — combine the targeting...

Bionest

Real World Evidence Replacing Some Clinical Trials?

Pharmaceutical companies have long used real-world data on drug performance to help justify their pricing decisions to payers. But now such real-world evidence (RWE), gathered from electronic medical records (EMRs), national patient registries and other patient databases, is increasingly being looked at as a way to make the case for new supplemental approvals and label...

Bionest

Highlights from AAN: Part 1

The Bionest team attended the American Academy of Neurology (AAN) in early May, where we heard exciting data presented in a number of neurological conditions. Among the highlights of this year’s conference:   Transformative Success in Spinal Muscular Atrophy   Spinal muscular atrophy (SMA) was a hot topic at AAN, with presentations of positive clinical...

Bionest

Breaking Out of the Bubble

X-linked severe combined immunodeficiency (X-SCID, or SCID-X1), is an orphan monogenic disorder popularly known as ‘bubble boy disease’ that affects 1 in 50,000-100,000 live births. The most serious of all primary immunodeficiencies, X-SCID causes severe failure of the immune system, including a lack of T cells, natural killer (NK) cells, or functional B cells. For...