Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act FAQs
While FDA review divisions commendably already consult with external experts on the review of some treatments, this type of collaboration should happen more often, especially as innovative new treatments based on the latest genetic discoveries move to the FDA for evaluation. The EXPERRT Act facilitates increased cooperation between the FDA and external experts in order to encourage a more efficient review process.
No. EXPERRT directs the Secretary of Health and Human Services to ensure that opportunities exist for FDA reviewers to collaborate with experts in rare diseases and subtypes of diseases, subsets of diseases of larger incidence, and those that are genetically targeted. It does not require reviewers to reach out to experts, stating “the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research shall, when appropriate, seek the opinion of external experts.” Outreach is left to the discretion of the review divisions.
No. In cases where outside entities request a meeting with FDA reviewers, EXPERRT indicates that they may “request the opportunity to meet with the review division,” leaving open the possibility that the reviewers could decline to meet with them.
Under the EXPERRT Act, external experts who consult with the FDA will be classified as “Special Government Employees,” meaning they are subject to FDA’s conflict of interest regulations. EXPERRT does not change conflict of interest regulations at the FDA.
The EXPERRT Act not only encourages greater consultation on the review of drugs for rare diseases, but also for subsets of diseases with larger patient populations and on the genetic targeting of treatments.
The EXPERRT Act has not yet been scored, although we do not anticipate that it will increase direct spending enough to merit a score.
This legislation has been endorsed by the National Organization for Rare Disorders (NORD) and Faster Cures: The Center for Accelerating Medical Solutions, the Everylife Foundation for Rare Diseases, and 40 additional organizations.
FDA officials have requested revisions to the EXPERRT Act’s language and Representatives Markey and Marino and Senator Whitehouse are continuing talks with the agency about the EXPERRT Act.
The Biotechnology Industry Organization (BIO) shares our goal of increasing opportunities for drug reviewers to access external expertise as they evaluate rare disease drugs and the organization is currently reviewing the legislation. The Pharmaceutical Research and Manufacturers of America (PhRMA) is reviewing the legislation as well, and Representatives Markey and Marino and Senator Whitehouse are maintaining contact with both groups as the EXPERRT bill moves forward.
The review of Cayston, an inhaled antibiotic for use in cystic fibrosis patients approved in March 2010, would have benefited from more open communication between reviewers and experts in cystic fibrosis and design of trials for CF products. The review process took 18 months, a time period during which CF patients were unable to obtain access to Cayston, an important addition to CF treatment. In part, the review was lengthened due to prolonged conversations between the drug sponsor and the FDA about the way the design of the clinical trials testing the drug.
On January 31, the FDA approved Kalydeco™, a groundbreaking new drug developed by Vertex Pharmaceuticals that for the first time addresses the underlying cause of cystic fibrosis in a subset of CF patients. Approved in three months, its review time was one of the fastest in the FDA’s history.
While there are several acts before congress right now that aim to enhance the FDA review process such as TREAT (S 2113) and FAST (HR 4132), EXPERRT is the only act that directs the FDA to facilitate greater access to external experts that specialize in the particular issues that affect rare diseases and subtypes of diseases.
The goal is for this legislation to be attached in some form to the PDUFA reauthorization bill. Representatives Markey and Marino and Senator Whitehouse are in contact with the House Energy and Commerce Committee and the Senate Health, Education, Labor and Pensions (HELP) Committee and are discussing options moving forward.
There are many important distinctions between the FDA’s negotiated PDUFA agreement and the EXPERRT Act.
While two provisions in the negotiated agreement specifically refer to external expertise, more needs to be done on this critical issue.
The “Advancing Development of Drugs for Rare Diseases” section commendably includes a directive to conduct one public meeting on clinical trial challenges for studying drugs for rare diseases, which is very important to the rare disease community. However, it is important that this type of collaboration happen much more often, on a case by case basis, and include discussion of the challenges of personalized medicine and the challenges associated with genetically targeted drugs for small populations, topics not covered under the mandated public meeting.
The “Improving FDA Performance Management” section provides that the studies conducted under the PDUFA agreement are intended to foster, among other things, development of programs to improve access to internal and external expertise, which is exactly what the EXPERRT Act does.
There are directives in this agreement that staff in both the rare disease program and elsewhere in CDER and CBER conduct increased utilization of and outreach to patient representatives. However, it is critical that FDA specifically encourages its reviewers to reach out to rare disease experts who can provide guidance on complex issues like clinical trial design and epidemiology.
Yes, in the “Making Benefit-Risk Assessment in Regulatory Decision Making,” it directs FDA to initiate a public process to nominate a set of disease areas that could benefit from a more systematic and expansive approach to obtaining the patient perspective on disease severity or unmet medical needs, convening 4 meetings per year with each meeting focused on a different disease area.
However, there is no guarantee that one of the disease areas discussed in the meetings will have to do specifically with rare diseases. In addition, the description of these meetings does not specify outreach to external experts like those in academia or other researchers, specifying instead the “relevant patient advocacy community and other interested stakeholders.” Lastly, it is important that consultation on disease severity and unmet medical need happen on a case by case basis, as reviewers encounter questions that may require additional input from scientific experts.