RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > How can FDA be Improved? Patient Groups Offer Ideas

How can FDA be Improved? Patient Groups Offer Ideas

Posted 10 December 2014 | By Alexander Gaffney, RAC

How can FDA be Improved? Patient Groups Offer Ideas

More than a dozen patient groups and trade associations are offering their advice to the US Food and Drug Administration (FDA) on ways the agency can improve its regulatory and administrative processes to take into account the views and expertise of patients and their advocates.


Since 2012, FDA has launched several initiatives meant to better take into account the views of patients. Some, like its patient-focused drug development initiative, have involved patients in more than a dozen meetings and solicited extensive feedback. Others, such as its medical device-focused Patient Preference Initiative (PPI) are still in the early stages of development.

Then, in November 2014, FDA issued an announcement in the Federal Register asking for feedback—and especially feedback from patient groups—about its patient-centric activities, as well as patients' views about the medical product development process.

For more on FDA's announcement, please see our story, "Is FDA Listening Enough to Patients? Agency Wants Feedback."

Patients and Patient Groups Weigh In on Regulatory Process

Now patients and patient advocacy groups alike are responding to FDA's solicitation, offering the agency advice.

Some selected advice follows:

National Multiple Sclerosis (MS) Society"The Society encourages FDA to offer more detailed guidance on the specific types of patient information that would be useful to the agency in its product reviews. Increasingly, patients are faced with the challenge of ensuring that they are providing the type of information that will be useful to researchers, product developers, and regulators. Furthermore, in open forums with limited time, such as PFDD meetings, it is critical to be able to deliver this information in an efficient and effective way."
National Multiple Sclerosis (MS) Society"FDA should also increase transparency regarding how patient information is being reviewed internally and incorporated into the agency’s decision-making process." … "Additionally, there is a lack of clarity as to how these reports will be updated in the future as new information about the diseases becomes available or if treatment regimens change."
The Leukemia & Lymphoma Society"LLS recommends that the FDA establish a transparent process to work with industry and the patient community to develop template frameworks for [Patient-Reported Outcomes] PROs to streamline the inclusion of substantive, useful PRO data within clinical trials intended to support an approval or new labeling claim. These templates should include best practices for survey content and systematic data collection methods which are tailored for specific disease areas and other variations."
National Kidney Foundation"Develop public guidance on opportunities for patient engagement, tools and processes that will be used for engaging patients, topic areas where patient perspective will be collected, and specifics on how patient perspectives are incorporated into FDA decisions."
FasterCures"Establish an advisory body to provide ongoing counsel about, input to, and monitoring of patient participation in regulatory processes and policy. This body, which would include substantial representation from the patient community, would provide guidance outside individual product decisions on the scope and implementation of programs and activities to foster greater inclusion of patient perspectives in regulatory decision-making for product review, post-market requirements, direct-to-consumer promotion, risk communication, and safety surveillance."
BIO"BIO recommends the establishment of public-private partnerships (PPP) to provide ongoing forums for dialog between patients, industry, clinicians, the scientific community and FDA. The PPP would also develop methodologies and study protocols for surveying patients for their views of their conditions and benefit-risk assessment and would serve as a shared infrastructure for conducting these studies on a voluntary basis."
Parent Project Muscular Dystrophy"We believe the FDA should establish a rare disease patient representative program whereby the agency solicits and maintains a standing list of qualified and vetted patients by disease/condition able to participate in advisory committee, product development or other meetings and exchanges. Undertaking such a process will help guarantee that a patient or primary caregiver directly impacted by the therapeutic area before each advisory committee can play a meaningful role in this most important process."
Pfizer"A potential barrier to these communications [with patient groups] is the current lack of clarity regarding what types of communications are understood by the Agency to be part of the medicine development process vs. what might be viewed as pre-approval promotion. While Pfizer believes that truthful and non-misleading communications about an investigational drug that make no efficacy or safety claims are permissible, we urge the Agency to provide greater clarity in this area."
Arthritis Foundation"We would encourage the creation of a patient portal on the FDA website where patients can get trusted clinical information and can provide informative comments on these treatments and engage directly with the FDA."
The National Health Council"FDA should also increase transparency regarding how patient information is being reviewed internally and incorporated into the agency’s decision-making process. Current outputs, such as the ‘Voice of the Patient’ reports from PFDD meetings, robustly capture the discussion and information collected from the engagements; however, how and to what extent this information is being used by FDA is unclear."
Alpha-1 Foundation"It is concerning that on most FDA panels that have patient representative SGEs, they are non-voting members and thus their perspective carries little weight in actual decision-making that may impact patient lives."
Cancer Leadership Council"We urge a discussion about the current standards for determining conflicts of interest and granting waivers. If the current process is depriving the agency of valuable expertise, the rules may need to be revisited."
Juvenile Diabetes Research Foundation"We suggest the Agency consider establishing disease-specific committees consisting of multiple patients at various stages of the disease (and/or appropriate caregivers). These committees and their members, individually or collectively, could be utilized Agency-wide to gain insight into their unmet needs and perspectives on benefit-risk assessments."
National Psoriasis Foundation"Ensure guidelines on patient and patient organization participants do not preclude engagement by those with knowledge and expertise on the issues under consideration. Just as the agency is encouraged to regularly invite multiple patient perspectives to the table, we encourage the agency to be mindful of establishing conflict of interest guidelines that will not disqualify those with deep expertise in the field."
Pharmaceutical Research and Manufacturers of America (PhRMA)"PhRMA encourages FDA, in consultation with stakeholders, to establish of a framework that encourages the development and predictable use of a set of tools and processes for understanding and incorporating the patient perspective into drug development and regulatory decision-making."
Pharmaceutical Research and Manufacturers of America (PhRMA)"PhRMA believes that use of technology and other novel approaches to patient outreach should be employed to allow for patients to engage in patient participation activities without requiring unnecessary time, effort or burden on the patient. Similarly, there should be an effort to develop models for patient participation that are scalable and feasible for use in any disease area or condition such that all patients can benefit from patient-focused drug development efforts."
Cystic Fibrosis Foundation"The Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision authorizing consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments (FDASIA Section 903). …  We urge the agency to take concrete action to implement the expert consultation program anticipated by FDASIA."
AdvaMed" Incorporation of patient perspective information into the product development and regulatory decision-making process will require patient education in the device development process and TPLC and increased patient understanding of regulatory requirements and processes. It is essential that all stakeholders understand and appreciate that the device development and testing process is in many ways different and in some respects far more complex, than the drug development/testing process."


All comments may be found here.


© 2022 Regulatory Affairs Professionals Society.

Regulatory Focus newsletters

All the biggest regulatory news and happenings.