So, after years of bemoaning a cumbersome and risk-averse licensing process for drugs, the industry is discovering that the
real challenge is reimbursement. The language of engagement has changed. Innovation defined around the merits of the science
is insufficient; it must offer value to the user as well, relying on metrics of performance against existing therapies, clinical
standards of practice, and patient-payer preferences. The problem is that, outside of Italy and an elaborate set of algorithms
devised by its national drug regulator, AIFA, no one has bothered to define what "value" is; payers by and large want this
definition to rest vaguely, in the opaque eyes of the beholder.
A breakthrough on this front is likely this year in the United Kingdom, where the Department of Health is finalizing an effort
to refigure the 50 year-old Pharmaceutical Price Regulation Scheme (PPRS) with a new system of value-based pricing that depends
heavily on metrics linked to disease states and unmet medical need. While many in industry welcome the idea of greater clarity
and transparency to bind the concept of value, precedent suggests that the prudent advice is to be careful what you wish for.
Bending the data glut
A particular concern is the scope and quality of the evidence base, which requires a consensus on how to make sense of mounting
volumes of raw data. "We are still data rich and information poor," Neil De Crescenzo, SVP of Oracle Health Sciences, said
in an interview with Pharm Exec. However, he contends this great disconnect is gradually being breached, as industry comes to recognize that information,
constructively applied, has amazing untapped potential to change patient and provider behavior, while regulators strive to
make up lost ground in exploiting IT to speed drug approvals and anticipate and resolve problems once a new drug is introduced
to clinical practice. "The desire of regulators to collaborate with industry in applying information to enhance the integrity—and
thus the credibility—of the drug approval process is a trend that has been little noticed, but it is decisively important
in easing the way to integrating medicines with better public health outcomes," he said. "That's the sweet spot, where both
sides should want to be."
Over the longer term, FDA and the EMA have to think carefully about how the registration decision will facilitate—or hinder—ultimate
access to the patient through reimbursement. The gap between the two has already been partially bridged in Europe through
the EMA mechanism of parallel review, which includes not only the regulator and the applicant but national authorities responsible
for establishing reimbursement eligibility through clinical and cost effectiveness studies. The next step is a pilot project
in 2013 on "adaptive licensing" to consider how a system of periodic checks of a product's performance against its label,
throughout the lifecycle, might work.
In the United States, the new Patient Centered Outcomes Research Institute (PCORI) is funding research to create a national
infrastructure for the conduct of outcomes studies as well as their application in real-world clinical settings. While PCORI—for
now—has no mandate to look at cost, it is relevant to point out exactly who is financing the group and its work plan: insurance
companies and the big self-insured employers, who under health reform must pay a special tax as a ratio to the number of people
Tying registration to the broader perspectives of a community that worries about value and cost can be lauded as a time-saving
end to duplicative data dumps, or as a concession to reality in forcing rigor on companies to develop drugs that payers really
want. But there are inherent dangers too, the most important of which is the premature rendering of "no go" judgments about
promising early stage therapies, even turning science into a game show for budget-obsessed politicians. The expert advice
to Big Pharma? Get in and help shape that process, or the process will shape you.