The p-value.

Statisticians use it. Non-statisticians have heard of it (likely in the context of the standard threshold of p = 0.05). While it’s not the only component of a clinical trial design, the p-value helps determine the maximum acceptable level of uncertainty associated with clinical evidence and, in a way, the fate of patient access to a new medical device.

But, is the traditional 0.05 threshold too restrictive for the patient populations willing to accept more uncertainty, depriving them of treatment options? Is it too permissive for other patient populations? Can we optimize clinical trial design by considering patients’ urgency for new therapeutic options, as well as their willingness to accept uncertainty?

Basic biomedical research may be in its most fruitful period in history: each year, with new technologies helping scientists advance our understandings of the underlying basis of human disease. At the same time, it’s increasingly difficult to undertake the process of translating promising biomedical discoveries into new drugs or diagnostics, especially those that will help relatively small numbers of people. “The problem rests not in the science,” says Whitehead Institute Founding Member Harvey Lodish, “but with the lack of funding for early-stage development.”

Andrew Lo, a professor of finance at the Massachusetts Institute of Technology, dialled in from Boston to talk about regulation, markets and the future of machine learning.

Lo pioneered the adaptive markets hypothesis, which describes markets as a complex evolutionary ecosystem, populated by different stakeholders that adapt to changes on the basis of certain behavioural traits and biases.

There's big money in biotech in the Greater Boston area. Last year, venture capital firms invested more than $3 billion in the state's biotech and pharmaceutical companies, according to the Massachusetts Biotechnology Council. The amount has risen every year since 2012. Local companies have already produced revolutionary treatments for rare diseases, cancer and more. And there's promise and hope for cures and treatments still to come. Because of that, there's no shortage of capital.Biotech companies often go public even if they haven't yet put a product on the market, let alone turned a profit. MIT finance professor Andrew Lo says there's good reason for so much exuberance.

Opinion article by Andrew W. Lo and Kenneth Kosik on the potential role of California in the space of Alzheimer's Disease research.

“Nobody’s talking about this story,” Max Tokarsky said. Not exactly true, as the founder and CEO of InvestAcure is telling anyone who will listen. A former non-profit executive-turned-evangelist for the Impact Investment and Public Benefit Corporation model, Tokarsky is slated to present the keynote address at the Alzheimer's-2018 International Conference in Rome, Italy May 8. The Rome conference brings together researchers from around the world focused on groundbreaking research to slow, reverse or prevent dementia and Alzheimer’s disease. A life-long social entrepreneur and former non-profit executive, Tokarsky will present "The Enigma of Eroom’s Law and The Wall Street Math Stifling Alzheimer’s Drug Discovery" in Rome. He wants to talk about funding a cure for Alzheimer’s disease. But he’s not talking about just asking major corporations or the government or even Big Pharma for help. Tokarsky wants everyone to pitch in. And he’s figured out a way we can.

Companies are going public without revenues and the stock market is jittery. It feels like we’ve been here before, but there are some important differences.

Derek Lowe's commentary on drug discovery and the pharma industry. An editorially independent blog from the publishers of Science Translational Medicine. All content is Derek’s own, and he does not in any way speak for his employer.

How do you drive investors to spend money on cutting-edge cancer treatments? One idea, according to economist Andrew Lo, is to sell securities in a megafund of research projects. Economics correspondent Paul Solman explores how financial engineering could be the starting point for curing cancer.

A biotech focused on developing drugs to treat genetic diseases just raised $135 million. BridgeBio Pharma, which was founded in 2015, builds out subsidiary companies around different inherited genetic diseases. The series C round was co-led by investment firms Viking Global Investors and KKR, which were joined by Perceptive Advisors, AIG, Aisling Capital, Cormorant Capital, and Janus Funds.

Andrew Lo, the MIT professor who recently proposed creating a megafund to finance a cure for cancer, has another way for the financial services community to help eradicate seemingly intractable diseases. Lo was the keynote speaker at a data science conference put on by the consulting group Mass Insight last Friday. The purpose of the conference was to explore how Massachusetts could become a leader in Big Data and machine learning. It brought together everyone from bank executives to venture capitalists to government officials at the Federal Reserve Bank of Boston.

Professor Andrew Lo of the Massachusetts Institute of Technology (MIT) says he wants to be like Harvey Lodish and help save the life of a relative one day. Lodish, a molecular and cell biologist at MIT, helped develop a drug to treat a rare genetic disorder known as Gaucher disease. Little did Lodish know at the time that later his grandson would be diagnosed with this disorder and Lodish’s pioneering work would save his grandson’s life. Lo’s goal may seem lofty considering he is a financial economist, not a doctor or a scientist. Like many of us, he has watched helpless as family members and friends are diagnosed with cancer. But sitting in the waiting room of a doctor’s office one day, Lo decided to follow in Harvey Lodish’s footsteps and take the first step towards saving a life.

Patients with a rare, chronic disease can find themselves in a conundrum when seeking treatment: Researchers can’t find enough patients to enroll in a statistically rigorous clinical trial and there is uncertainty in the regulatory path. Frustrated by the lack of treatment options on the market, patients may be willing to accept a certain level of risk that device manufacturers normally shy away from.

"Not that long ago, it looked like it was game over for the field of study that produced the mortgage backed security, the credit default swap and all manner of other exotic derivatives that exploded so spectacularly in the financial crisis of 2008.

"Now, financial engineering theory is being touted by several academics as the key to curing cancer faster, reducing the impact of climate change and imposing better risk discipline on the same banks that were once almost felled by the discipline’s creations..."

On this episode of the HBO documentary series, Andrew Lo discusses an idea he and colleagues developed with regard to the FDA approval process. They've developed a model that incorporates patient preferences into the design of clinical trials in order to set appropriate thresholds for FDA approval.