In many ways it has been a challenging year for the biopharmaceutical industry and it is certain that company executives will be glad to close the curtains on a period that has made planning difficult. This is because they have had to deal with uncertainties swirling around such issues as health care reform and tax changes, a market sentiment for the sector that blew hot and cold, and the ongoing contentious debate over the price of drugs. Throughout the year, in BioWorld Insight, we published the views of company managers, business executives, government regulators and scientists on those and a variety of other topics, and in our popular annual feature we include a selection of those that helped define 2017.

The Editor's favorite quote from the year:

"We are still in a system where we are delivering Star Wars advancements and technologies into the Flintstones health care system."

–Freda Lewis-Hall, chief medical officer at Pfizer Inc., speaking at the Cleveland Clinic's 15th Medical Innovation Summit

The Trump effect

"To me, you just can't worry about it. I think we need to stop being obsessed with what he tweets about at three o'clock in the morning."

–Morrie Ruffin, Adjuvant Partners, speaking at the BIO-Europe Spring meeting

"I haven't a clue about what to expect from [President-elect Donald] Trump in 2017. He says, 'Oh, we're going to do something on pricing,' and the markets go down and then at the end of the day the markets go back up. As far as I'm concerned, it's business as usual until it changes. I have no other way to operate, because if it's not business as usual the universe of things that could happen is so great that I can't possibly handicap it."

–Jay Lichter, president and CEO of COI Pharmaceuticals Inc. and managing director at Avalon Ventures

Drug pricing: A thorn in the industry's side

Drug pricing issues have been pervasive throughout the year. As the Biotechnology Innovation Organization's CEO, Jim Greenwood, noted at the BIO 2017 international convention, "The future of the industry right now really hinges on how we come out of this pricing thing."

"You folks have done a terrific job over the years, but we have to get prices down for a lot of reasons. We have no choice. For Medicare, Medicaid, we have to get the prices way down."

–President Donald Trump, speaking at a meeting with the Pharmaceutical Research and Manufacturers of America, together with the CEOs of several major U.S. biopharma companies

"End the shenanigans. Our economic model, which rewards highly innovative drugs with the opportunity to hold monopolies for a limited period of time through patents and exclusivities, and to freely price their products to a measure of the value that a transformative drug offers, also depends on the generic approval process working as intended."

–FDA Commissioner Scott Gottlieb, to brand drug manufacturers at the opening of a Federal Trade Commission workshop looking at the obstacles to robust competition in the U.S. prescription drug market

"Spending on prescription medicines in the vast majority of OECD countries has been stable around 10 [to] 15 percent of total health care budgets for a number of years. This is quite remarkable, given the industry in the last 10 [to] 15 years has developed a number of breakthrough treatments."

–Thomas Cueni, director general of the International Federation of Pharmaceutical Manufacturers, speaking at the The World Health Organization Fair Pricing Forum

"If consumers are priced out of the drugs they need, that's a public health concern that FDA should address, within the scope of its mandate and authorities. While FDA doesn't control drug pricing, our policies do affect competition in the market."

–FDA Commissioner Scott Gottlieb

"If a lifetime treatments costs $6 million and I can replace that with one infusion, can I charge $6 million? Probably not."

–Sandy Macrae, president and CEO of Sangamo Biosciences Inc., in a discussion on value-based pricing at BIO CEO & Investor 2017

Regulatory Policy

"All too often, as we rightly focus on evidence-based medicine, we can lose sight of the human experience of these diseases and different therapies. What may seem simple in a lab may be overwhelming or difficult when applied to real patients in real-life situations – all the more so when children are involved. The FDA and all agencies should ensure that they have appropriate processes to seek and incorporate this vital input. It is critical that treatments that do exist for those with rare conditions are accessible and affordable."

–Sen. Amy Klobuchar (D-Minn.), calling on Congress to protect patients from discrimination in insurance coverage and to work to bring down drug costs

"We're announcing three new, significant policy documents to advance the FDA's approach to the development and proper oversight of innovative digital health tools. We know that consumers and health care providers are increasingly embracing digital health technologies to inform everyday decisions. From fitness trackers to mobile applications tracking insulin administration, these digital tools can provide consumers with a wealth of valuable health information. Further, clinical evidence demonstrates that consumers who are better informed about health make better and more efficient decisions, take steps to improve their lifestyles and their health choices, and often experience better outcomes."

–FDA Commissioner Scott Gottlieb

"From cancer drugs to expensive eye drops, many drug companies insist on selling their products in excessively large, one-size-fits-all vials that contain more medicine than the average patient needs. This is a colossal and completely preventable waste of taxpayer dollars, and it means American patients and hard-working families are paying for medication that gets tossed in the trash. Instead of allowing the pharmaceutical industry to profit at our expense, it's time we put an end to this wasteful spending."

–Sen. Dick Durbin (D-Ill.), who is co-sponsoring a proposed Reducing Drug Waste Act

Gene therapy comes of age

The potential of cell-based therapies "has become a thrilling reality."

–Janet Lambert, CEO of the Washington-based Alliance for Regenerative Medicine

The promise has always been there for gene therapies. Unfortunately, the field had seen a number of false starts for the expected explosion of investment and business development around treatments that correct for faulty genes. The breakthrough appeared to have been made following the European approval of Glybera (alipogene tiparvovec, Uniqure BV), for the treatment of the ultra-orphan metabolic disease lipoprotein lipase deficiency (LPL). But, while the medicine was widely heralded as the "first gene therapy" in the Western world, it failed to take off and its developer subsequently abandoned plans to have it approved in the U.S.

Five years on from Glybera's approval we appear to be finally at the beginning of a sustainable growth path for the gene therapy market. Confidence has returned big time, thanks to the FDA's precedent-setting approval of Novartis AG's chimeric antigen receptor T-cell (CAR T) immunotherapy, Kymriah (tisagenlecleucel), to treat patients up to age 25 with B-cell precursor acute lymphoblastic leukemia. Observers and industry analysts now expect that gene therapy will finally make good on its promise to transform 21st century medicine.

The field ended on a high note when Spark Therapeutics Inc. gained the FDA's nod for Luxturna (voretigene neparvovec-rzyl), which became the first gene therapy approved in the U.S. that targets an inherited disease, caused by mutations in a specific gene. The treatment was approved to treat children and adults with confirmed biallelic RPE65 mutation-associated retinal dystrophy, an ultra-rare progressive disease that leads to vision loss and may cause blindness.

"Today's approval marks another first in the field of gene therapy - both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss - and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses. We're at a turning point when it comes to this novel form of therapy and at the FDA, we're focused on establishing the right policy framework to capitalize on this scientific opening. Next year, we'll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters — including new clinical measures — for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted."

–FDA Commissioner Scott Gottlieb

Data are paramount

"Data by itself is amazing, but it is frozen knowledge. It is up to us as entrepreneurs to use our energies to melt this data into meaningful projects."

–Atul Butte, director of the Institute for Computational Health Sciences at the University of California San Francisco, a panelist on the roundtable on Investing in Data: The Future of Health Delivery at the J.P. Morgan Healthcare conference

"Where we are now in our journey in artificial intelligence, is, thanks to the advances that have been made, we're seeing an inflection in which many of us who had been using machine learning are now transitioning to deep learning. Hopefully, soon there will be a lot more time spent on the computational aspect and a lot less time being spent on the wet lab."

–Andrea de Souza, Nvidia, speaking at BioTaiwan's BioBusiness Asia Conference

Real-world evidence

"Despite the approval of several new cancer medicines in recent years, there is a lack of infrastructure that would allow us to gather data and insights in a systematic and timely manner on the use and effectiveness of medicines in a real-world setting. This collaboration allows us to develop a readily scalable and sustainable infrastructure across multiple cancer medicines, indications and geographies, and it will help reduce the administrative burden on health care providers who provide the data."

–Gergana Zlateva, Pfizer Inc.'s vice president of payer insights and access for oncology, on a new collaborative initiative to search out real-world data on the use of cancer treatments across seven top European markets

"We're really interested in understanding the burden of the disease on patients and, equally, we're interested in understanding the burden of current treatments and how well they're alleviating symptoms. We'd like to know what symptoms or problems are most important for patients to have ameliorated, what they would expect from a new therapy and how they think that should be measured. Understanding the daily lives of patients, their experiences and how the burden of disease affects them in various ways will really help us to develop patient-reported outcomes so that we can get a very clear picture of any impact that a new therapy is having – both the good and, possibly, the downside."

–Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, speaking at the Arthritis Foundation's Patient-Focused Drug Development meeting

Looking for breakthroughs in neurodegenerative diseases

"In the 21st century, there is not a single cure for a single neurodegenerative disease. Not one . . . There is no therapeutic. There is no strategy. There is bupkes in the 21st century."

–Roberta Diaz Brinton, director of the University of Arizona Center for Innovation in Brain Science at the University of Arizona Health Sciences, speaking at the 2017 annual meeting of the Society for Neuroscience

"Over the last few years, a lot of pharmaceutical companies have run away from CNS and we've actually run toward it."

–Bob McQuade, executive vice president and chief strategic officer at Otsuka Pharmaceutical Development and Commercialization Inc.

"One of the issues that you've seen so far in the development of Alzheimer's drugs, particularly for amyloid plaque, is you've had some failed trials or maybe you've had failed drugs, and the problem is we don't know which is which."

–Ken Verburg, senior vice president of global development, Pfizer Inc., speaking at the Public Policy and the Coming Epidemic of Neurodegenerative Disease panel during BIO 2017

"We're thinking it's mostly because Alzheimer's is polygenic – many genes are involved. One target, one disease doesn't work in Alzheimer's."

–Claude-Henry Volmar, senior scientist at the University of Miami Medical School's Center for Therapeutic Innovation

Immuno-oncology

"We believe this is the beginning of a new wave of treatment options that offer new hope to patients suffering from many different types of cancer. As the first company to invest in CAR T by forging a first-of-its-kind collaboration with the University of Pennsylvania, we are already working to advance many more therapeutic options in the field of cancer. We remain deeply committed to advancing this field of medicine and to continuing to bring innovative treatment options to patients."

–Bill Hinshaw, general manager, U.S. Oncology, Novartis AG, on the FDA approval of Kymriah, the CAR T immunotherapy

"We're quite interested in things that would augment cellular therapy. We're going to take some time to look at our own portfolio and determine what, if any other things, we should do."

–John Milligan, president and CEO of Gilead Sciences Inc., on its acquisition of Kite Pharma Inc., whose most advanced candidate, the CAR T therapy axicabtagene ciloleucel (axi-cel, previously KTE-C19), received FDA approval

"In thinking about I-O, particularly as a small company, one needs, for the future of the field, to be able to serve the largest number of patients and treat the largest number of tumors by having the ability to engage both arms of the immune system. The way we think about I-O today is a little bit like a boxer only being able to fight with one hand, and that's the adaptive immune system. Freeing up the other hand by combining with the innate immune system will lead to responses that will benefit patients and advance the field more in a revolutionary than evolutionary manner."

–Gary Glick, co-founder and CEO, IFM Therapeutics Inc., whose company has been acquired by Bristol-Myers Squibb Co.

Brexit

"A disorderly Brexit has the potential to have a very negative impact on patient health across Europe. Negotiators from both the EU and the U.K. have a responsibility to do everything they can to avoid this risk."

–Nicola Bedlington, secretary general of the European Patients' Forum

A coin toss

Amsterdam won the race to be the new home for the EMA on the toss of a coin, after it was tied with Milan following three rounds of voting in a secret ballot. Amsterdam was, in fact, the city most favored by the EMA's 890 staff as the new home for the agency. In a survey of its employees, 81 percent said they would be prepared to move to the Netherlands. The agency now has just over 16 months to prepare for the move and take up its operations in Amsterdam on March 30, 2019, at the latest.

"We welcome today's decision on the new location of EMA. Now that we finally know where our journey is taking us, we can take concrete actions for a successful move. Amsterdam ticks many of our boxes. It offers excellent connectivity and a building that can be shaped according to our needs. However, even though a majority of EMA staff would be willing to move to Amsterdam, our activities will be impacted and we need to plan for this now to avoid the creation of gaps in knowledge and expertise."

–EMA Executive Director Guido Rasi

"Losing the EMA is a bitter pill to swallow. The focus must be on ensuring that there is a smooth transition to allow for this to happen so that we do not see a detrimental impact on the supply of medicines to patients in the U.K. and EU."

–Steve Bates, CEO of the U.K. Bioindustry Association

China

"China's medical landscape is changing and improving every day. People like to criticize it and say it's not mature, that you can't find good health care in China, but that isn't true. You just have to know where to find it and that is gradually becoming easier."

–Sigal Atzmon, president at Medix Group, an international medical consultancy

Summing up

"On the surface, 2017 seems to have been a pretty solid year for the biotech sector. The IBB and NBI indices are up 21 percent (YTD as of 12/15/17), just edging out the S&P500 (up 20 percent). Political rhetoric and concerns over drug pricing policy initiatives have cooled off substantially since this time last year, opening the door for broader money flow into the sector. Innovations in both new (CAR T, gene therapy, cystic fibrosis) and old (depression, headache) areas abound. And with Scott Gottlieb at the helm, the FDA has maintained its more permissive, pragmatic and predictable approach."

–RBC Biotech Outlook