John Maraganore, CEO of Cambridge-based Alnylam Pharmaceuticals, begins his chairmanship of BIO this morning when outgoing Chairman Ron Cohen passes him the gavel after two successful years at the helm.
Maraganore is a beloved figure in the Cambridge biotech community. He has worked in the same half-mile radius around Kendall Square for 29 years. Before being tapped to lead Alnylam in 2002, he served in senior executive roles at Millennium Pharmaceuticals and Biogen.
During Maraganore’s tenure, he will oversee BIO’s work to help ensure the implementation of more patient-centered drug development as well as efforts to move to a more competitive, value-based system on drug pricing.
During negotiations over the 21st Century Cures Act and PDUFA VI, BIO helped to craft new policies to bring the patient’s voice into the drug development and approval process. This work is personal for Maraganore: his mother passed away nine years ago from her chronic lymphocytic leukemia, a condition she would have survived with today’s medicines. .
We asked the incoming chairman about his priorities for BIO and the industry as a whole as he assumes the chairmanship of the world’s largest biotechnology trade association.
You are taking the reins of BIO at a time of unprecedented progress and possibility for biotechnology, and some might say, unprecedented peril for innovation policy. How do you see the state of our industry?
We must focus on progress every chance we get. Biotechnology is on the cusp of transforming modern medicine from chronic disease management to cures. The drugs in the pipeline right now are a quantum leap forward. The new millennium has brought rapid, unprecedented advances in our understanding of human biology and genetics. The science is unstoppable, but turning these gains into actual medicines requires investment, and too many of our investors have been spooked by the litany of political and social media attacks against innovators. That’s why BIO’s Value Campaign continues to matter. We have to keep educating the public and our elected officials about our complex ecosystem, why it’s the best in the world, why U.S. companies innovate 57 percent of the world’s new medicines, and why patients would be harmed by bad policy that undermines investment.
It’s not just health where biotechnology is making a difference. Our members generate biofuels and cleaner bio-based products that are made from renewable plant feedstocks, rather than oil. Our members create seeds that can withstand drought or insect damage. Our members innovate countermeasures so we can swiftly respond in the event of a pandemic outbreak.
What’s your top priority as BIO Chair?
The two most important guiding principles for me will be how we structure our advocacy to support innovation and help patients. So our first job is get PDUFA VI over the finish line. After two years of negotiations, we’re almost there. But once enacted, the law will only be as good as its implementation. So we have to continue working closely with the FDA to make sure the agency make additional hires with specific expertise in the new tools of 21st century medicine.
In the new user fee agreement and the 21st Century Cures Act, we’re codifying a commitment to give patients a real voice in how the FDA makes its decisions. We’ve secured commitments from the FDA over the next five years to work with our industry to develop new ways to transmit patient feedback, including perceptions of their disease and how they view treatment risks vs. benefits. The FDA has also agreed to look at clinical trials from a more patient-centered perspective. That means organizing trials in a way that helps more patients participate and stay enrolled. In this era, a patient can measure their blood pressure on an Apple watch or an iPhone. We should be able to utilize this new technology in modern clinical trials.
Because of BIO’s advocacy, the FDA has agreed to look at ways to collect and evaluate data from the perspective of the person who is sick. Survivability isn’t the only important metric. When the FDA examines risks and benefits, they do look at things like quality of life, level of pain, and severity of side effects; however, they should be evaluating these risks and benefits with systematic input from patients. Ultimately, we want data on patient perspectives and outcomes to be scientifically valid, so it can go on a drug’s label.
We need to start using evidence that is already cataloged by key players across the health care system. A lot of information has been collected on how patients respond to different medicines. A record is created every time a patient encounters the health care system. Electronic medical records show what medicines patients have taken; how they responded; and whether they got better, sicker, or ended up in surgery. Medicare, insurers, hospitals and doctors all have databases that allow them to analyze what’s working. Right now, the FDA doesn’t use this evidence to approve new drugs. PDUFA VI will improve the process by which companies can access these databases. Real-world evidence can supplement clinical data so new indications for drugs can be approved by the FDA and reach more patients in need.
One of the criticisms of our industry on pricing has been that we focus too much on what we’re against and not enough on what we’re for to bring prices down. Is that fair?
BIO just joined a broad new, industry-wide coalition to address these criticisms and work toward a holistic solution on price. The coalition includes the country’s two largest PBMs and two of the four largest health insurers. We’re working together on ways to bring more generics and branded drugs to market faster. We’re working together on breaking down regulatory barriers that prevent us from moving to value-based pricing. And we’re working together to provide information to consumers so they understand what their insurance company is charging them out of pocket for their different medicines. These are concrete solutions that will promote competition and transparency and make medicines more accessible. These approaches actually address the underlying market and regulatory failures that gave rise to the abuses that have gotten so much attention. Longer term, the BIO Board has established a committee to reimagine the drug pricing system. We’re promoting new ideas on novel payment arrangements and alternative financing that will position us for the coming generation of personalized medicine and curative therapies.
BIO has worked hard to change the conversation on price to a conversation on value. Why is this so important?
What we have not always done well as an industry is to communicate the overall value of the medicines we bring forward. It’s across two dimensions. One is the pharmacoeconomic benefit of reducing the cost of disease burden. It’s real. You can quantify it. There are many examples of drugs that reduce hospitalization, reduce mortality, and reduce other morbidities and the costs associated with that. There’s no question there’s a near-term value proposition that drugs bring to the table.
The second part is the reality that the medicines Americans pay for today will be free or nearly free for our children – and our children’s children. That’s because of our unique social compact. Biotech companies get a limited period of exclusivity – usually, 10 or 12 years – and when that window closes, we lose exclusivity and generics enter the market, commoditizing our innovations for the social good. If we don’t pay for innovation today, we’re ultimately depriving our children of access to new medicines tomorrow.
What’s the price of not having drugs for Alzheimer’s? What’s the price of not addressing hepatitis C? If you buy something, and the consequence of buying it is you don’t have the disease, and your children don’t have the disease, and your children’s children don’t have the disease, that’s incredibly valuable. The price has to be consistent with the value. But the time period in which you measure that value matters. If you measure the value as a payer might, in their annual budget projections, you can become very short-sighted about the value of a given medicine. If you measure value by looking at the long-term impact of that medicine and decreased morbidity over time, like with Sovaldi’s impact on liver cancer and transplants, then it’s incredibly valuable. Interestingly, the United Kingdom’s single-payer system had no trouble reimbursing Sovaldi and Harvoni.
The U.S. innovation ecosystem is delivering. If you take a consolidated look at the industry pipeline, we’re developing medicines that could have scarcely been imagined 10 years ago. New hope, new cures, new breakthroughs: That’s the value biotechnology provides. As chairman, I will fight to preserve and strengthen the ecosystem that has made these gains possible.