The US Food and Drug Administration (FDA) must be made an independent agency with a clear mandate to encourage the development of innovative products, industry leaders have urged.
And a progressive approval pathway needs to be created at the agency, so that patients can have earlier access to promising new treatments before they reach final approval, according to Jim Greenwood, chief executive of the US Biotechnology Industry Organisation (BIO), speaking at the industry group's annual conference in Washington.
These proposals are part of a plan drawn up by BIO which includes 11 regulatory provisions aimed at creating a "21st century FDA." The initiative is the result of the industry group's year-long "big thinking project," through which they have asked industry leaders, investors and patients across the US for their "biggest and smartest ideas" for creating the ideal environment that would enable policy to keep up with biotechnology science, said Mr Greenwood.
"We have spent the last year collecting ideas, thinking them through and hammering them into legislative and regulatory reforms that we will hand-deliver to the Congress and to the President," he told the meeting.
Respondents to the initiative told BIO the industry needs an FDA "that is more interested in promoting new drugs than postponing them," he said. "We need tax policy that encourages private investment in biotechnology companies," and "we need Congress to get real about risk and benefit on behalf of patients who are running out of time."
Currently, the FDA has neither the resources nor the authority to do its job, Mr Greenwood told delegates. The regulatory process has become too unpredictable, time-consuming and expensive, regulatory science has not kept up with the frontiers of innovation and the agency has been given too many conflicting missions without sufficient resources to perform them, he added.
The industry group's proposed programme of reform of the FDA would - as well making the agency independent and requiring it to support innovation - provide it with greater continuity of leadership by giving the FDA Commissioner a fixed term of office, rather than changing with every new presidential administration. It would also appoint a chief innovation officer at the agency to ensure that forward-thinking, cutting-edge science is integrated into its operations at all levels, and provide it with new ways to bring in "top-notch" external scientific and medical advice and more input from patients.
Turning to the US deficit, Mr Greenwood pointed out that Medicare - the federal health insurance programme for the over-65s and some disabled people - is facing an unfunded liability of almost $25 trillion over the next 20 years, and that the number of Medicare beneficiaries is set to rise from 50 million at present to 65 million by the end of this decade and more than 80 million by the end of the next.
The cost of caring for these beneficiaries’ cardiovascular disease, cancer, diabetes and Alzheimer's is “unaffordable," but BIO has been telling Members of Congress: “if you give us the chance, we can help you solve that problem."
"Because you can't do it with price controls. And if you try, you will kill innovation," he warned.
"Instead of Congress fixating on the cost of innovative medicines, they should consider this - a new drug that could delay the onset of Alzheimer's by even five years would save Medicare $50 billion a year," he said, adding: "that's what biotechnology can do."
But for the industry to deliver on that promise, the economic model that drives biotech innovation has to be re-engineered, Mr Greenwood went on. The tax code currently doesn't recognise that biotech companies are different – that "they have to survive on investor capital for long and rocky years before they make a dime," and that they need tax laws which include incentives to encourage, not punish, more investment in innovation, he told the conference.