Room for Improvement: US policy enhancements on the way

March 10, 2015 ctheodoropulos

On many levels it seems like things could not be any better for biotech with positive reports on IPOs, follow-on offerings, M&A, licensing, and market valuations. However, there is always room for improvement, reported David Thomas, Director for Industry Research and Policy Analysis at the Biotechnology Industry Organization (BIO) in his opening remarks at BIO-Europe Spring® in Paris.

He shared two recent studies from the BIO Industry Analysis team based in Washington, DC with participants during the opening plenary session.

The first was drawn from a recently published study covering the last 10 years of US venture capital funding of therapeutics companies.

BIO Chart 1

The study is unique in that it separated out investments in drug delivery and reformulations of approved drugs from the investments in companies with new mechanisms of action and new chemical entities.

The study found a big difference in funding by therapeutic area when looking at the change in novel drug R&D funding from the five years before the financial crash of 2008 and the five years after.

Areas such as endocrine, psychiatry and others were down more than 50% when comparing the two periods. Many other areas were down more than 20%, even though they represent indications that affect millions of patients with chronic diseases.

Oncology seems to have held steady, recovering from a 50% drop during the crisis period.

At the very top of the list of increased funding is the metabolic category, where the rare genetic diseases can be found, an area where we have seen considerable investment interest in recent years, noted Thomas.

“Funding of innovation has started to come back over the last five years, but it is not distributed equally across disease areas,” he said.

Drawing on a second study, based on BIO’s work with Biomedtracker, Thomas stated that “looking at R&D productivity across the entire industry, the latest numbers suggest that for both public and private companies, success rates for clinical trials have not increased.”

For a biotech company with a single Phase II drug, he said, there is only a 17% chance of that product making it to the market. Statistically speaking, this would mean a typical emerging company would need to bring five programs through Phase II to ensure that it could bring one drug to market.

According to Thomas, the two studies would suggest that the biotech industry still has high capital requirements due to failed clinical trials, extremely long timelines, and an exodus from certain chronic diseases at the venture level.

“Can changes in policy help the industry reduce the risk involved in drug development?” he asked. “How can we incentivize investment across all diseases?”

These questions are the focus of a major legislative initiative happening back in Washington, called the 21st century cures initiative, and BIO has been working with the US Congress on these proposals.

As industry brings forward the progress of science through its pipelines, BIO wants the policy environment to be as efficient and as up-to-date as possible.

One of the highlights of the draft legislation is that it recognizes that there has been a paradigm shift toward patient-centricity, and that future drug development will require active collaboration between sponsors, regulators and patients.

The new law, if passed, would establish methodologies and data collection methods for patient experience assessments that could be incorporated into the FDA’s benefit/risk framework and regulatory decisions.

Other provisions in the bill would further enhance and incentivize drug development, such as the creation of a qualification process for novel surrogate endpoints, and new data exclusivities.

Another component to highlight is a provision that calls for the development of a longitudinal study that could deepen our understanding of chronic diseases.

Separately, just last month, President Obama announced the precision medicine initiative. The goal is to create a database consisting of data from one million volunteers who agree to have their genetic information shared and linked to diagnostic tests, and diet and lifestyle information. Access to this “Big Data” could help drug developers derive correlations between disease states, biomarkers, and DNA sequences that will help them learn how best to intervene sooner and with greater success in complex diseases.

“These are big initiatives likely to effect future R&D, and they are moving fast in Washington,” said Thomas. “Coming in right behind these are the preliminary discussions around the New User Fee Act, called PDUFA 6. Recall that the last User Fee Act in the US (PDUFA 5), enacted in 2012, brought us the Breakthrough Therapy Designation and other items, which have been highly successful.”

“We want to expand upon what was achieved, but also find out what is important to biotech and pharmaceutical companies in this current environment,” he said.

“For this reason, BIO has created a platform where you can weigh in. This is an opportunity to anonymously tell BIO and the FDA how your IND process went, how your clinical trial communication was, to tell us what is working well, and to say what needs to be improved upon to help inform policy development,” Thomas told the audience of executives at BIO-Europe Spring.

“We hope to hear more from Chief Medical Officers,” he added.

To access the BIO FDA Survey, please follow this link :

“We look forward to the discussions in the coming months on how to make the changes the industry needs to help bring products to patients sooner,” Thomas concluded.


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