The New, Patient-Centric FDA: A Double-edged Sword | Pharma Industry Regulation |

A report published by PricewaterhouseCoopers’s Health Research Institute reveals that the FDA, which developed the Patient-Focused Drug Development program in 2012 to better engage with patients, has held 21 disease-specific meetings to gain insight from them.


So far the meetings have focused both on well-known diseases such as breast cancer and HIV, and not so well-understood maladies such as female sexual dysfunction and Chagas disease. But what questions is FDA asking of patients and how often do they recur in the 21 meetings held? (See chart here).


Here are some FDA actions that should warm the cockles of the heart of patient engagement advocates in the future, according to the report. These are based on FDA’s PDUFA (Prescription Drug User Fee Act) reauthorization proposal:


  • Between 2017 and 2021, the agency expects to hire additional staff focused on engaging with patients and facilitating the development as well as use of patient-focused drug development methods.
  • In 2018, FDA is expected to provide draft guidance describing approaches to collecting patient and caregiver input.
  • In 2019, the agency plans to issue draft guidance describing how companies can collect information from patients, and how that information can be used in the drug development and regulatory decision-making process.
  • Another draft guidance describing how meaningful patient perspectives and information can be collected in clinical trials is expected in 2020.
  • And finally, a draft guidance on patient-reported outcome measures to replace the one released in 2009 is set to be available come 2021.


Draft guidance aside, FDA is also home to a treasure trove of data. And now the agency is opening up access so that third parties can turn that information into insight. For instance, through the openFDA database, companies like Iodine and Advera Health Analytics are turning adverse event information on drugs into a searchable format, the report found.


While FDA is dabbling with patient engagement strategies, on the flip side, patient advocacy groups are also getting involved in the regulatory space (read, for example, “How a #pharma Funded ‘Grassroots’ Patient Advocacy Campaign Changed FDA's Approval Process”; The report mentions one — Parent Project Muscular Dystrophy — that developed a guidance document so that companies could use it to develop drugs for Duchenne Muscular Dystrophy. That guidance document ended up being modified and adopted by the FDA for use by companies developing drugs for the disease.


“While patient input is unlikely to improve the approval chances of a drug lacking solid efficacy and safety data, regulators may be more willing to work with companies that are developing a product in close concert with engaged or especially ill patient populations,” it advised.