Personalized Medicine Runs Into Bureaucratic Barriers

Published May 31, 2016

A new report by the Brookings Institution identifies policy barriers to personalized medicine, the individualized treatment approach which many in the pharmaceutical industry believe is the next major step for health care technology.

“Enabling Personalized Medicine through Health Information Technology: Advancing the Integration of Information,” was written by David M. West, a Brookings vice president and director of governance studies and founding director of the Center for Technology Innovation.  While proponents claim personalized medicine can reduce health care costs and allow for better-targeted therapies, West maintains these benefits are “limited by an outmoded policy regime.”

“Federal regulations were developed years before recent advances in gene sequencing, electronic health records, and information technology. With scientific innovation running far ahead of public policy, physicians, researchers, and patients are not receiving the full advantage of latest developments,” writes West. “Current policies should leverage new advances in genomics and personalized medicine in order to individualize diagnosis and treatment.”

Designed to Identify Specific Targets

Alison Ayers, executive vice president of commercial development for Pfizer, says the personalized medicine process starts with basic scientific research to identify a relevant target that is linked to, for example, tumor development and growth. When the target is identified, chemistry is initiated to create molecules that interact specifically with that target and achieve the required effect of blocking its tumor-promoting activity. When a suitable chemical molecule is found, the process begins for safety testing and formulating the molecule for appropriate administration to humans.

“We then begin clinical trials. The process to discover and develop a molecule to enter into clinical testing can take five or more years,” says Ayers. “In terms of whether or not the personalized medicine process will reduce the overall time from oncology development to approval, it should be noted that a diagnostic still has to be developed. Creating this diagnostic takes time, and is not always possible to accomplish in parallel with drug development.”

Even though researchers hope to achieve regulatory approval of treatments based on a smaller number of patients, the Food and Drug Administration is still going to require substantial patient experience.

“We will still have to submit significant safety data with the initial registration and with an ongoing post-marketing commitment to follow safety guidelines,” says Ayers.

Have to Convince Regulators

One of the challenges for the industry is convincing regulators this more innovative approach to care will deliver positive results for patients.

“There is no evidence that risks are greater with personalized medicine than for any other treatment. We hope the risks may be less, since personalized medicines will target the tumor abnormality, which potentially could reduce other adverse effects,” says Ayers.

‘Policy Regime Restrains Innovation’

One example which could prove to be a model for future treatments is the Pfizer drug Crizotinib, designed to apply to roughly 3-5 percent of lung cancer victims who have a specific mutation. The EML 4 ALK mutation was discovered in 2007, and phase 3 trials with Crizotinib started in 2009. Researchers were able to move into development very quickly, only two years from the identification of target, for a drug targeted to help roughly 10,000 cancer patients. Currently, however, there is no set regulatory path for gaining approvals based on a gene mutation, Ayers says.

“We don’t know what the costs and timeframe will be, as we have not brought a personalized medicine to market yet,” Ayers said. “But we have not spared any developmental costs. It should also be noted that although the trials may be smaller, the cost of processing the tissue to identify the biomarkers and the cost of developing the companion diagnostic can be significant.”

West’s study recommends eight steps policymakers should consider to advance reforms that facilitate personalized medicine.

“Currently, there are problems in terms of interoperability, standards, data sharing protocols, privacy, predictive modeling, and rapid learning,” West writes. “Scientific innovation in the areas of genomics, imaging, and computing is far ahead of public policy. The result is a policy regime that constrains innovation and does not help physicians, researchers, or patients receive the full benefits of these advances.”

Kenneth Artz ([email protected]) writes from Dallas, Texas.


Internet Info:

“Enabling Personalized Medicine through Health Information Technology,” The Brookings Institution: