Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act FAQs

The FDA already consults with external experts on the review of treatments. Why is this bill necessary?

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.

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Does this bill require FDA reviewers to reach out to experts outside of the FDA?

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.

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EXPERRT allows outside experts to request consultation with FDA review divisions. Are reviewers required to consult with those who request a meeting?

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. 

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How do the FDA’s conflict of interest rules affect the consultation encouraged by the EXPERRT Act?

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.

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Does this legislation only affect the review of treatments for rare diseases?

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.

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How much will this new initiative cost?

The EXPERRT Act has not yet been scored, although we do not anticipate that it will increase direct spending enough to merit a score.  

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Who has this bill been endorsed by?

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.

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What is the FDA’s position on the EXPERRT Act?

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.

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Do biotech and pharmaceutical companies support 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.

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Is there a specific example of when this type of consultation was needed and not utilized?

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.
With more extensive outreach to external experts in cystic fibrosis who could have provided vital context and advice about CF trials, the process might have moved forward more quickly. Had there been an expert in cystic fibrosis providing input during the drug’s evaluation, we believe this badly needed drug could have been available to patients more quickly.

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Is there a specific example of when this type of consultation was utilized successfully?

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. 
Throughout Kalydeco’s review, the Cystic Fibrosis Foundation and renowned CF experts worked closely with Vertex and the FDA, providing valuable insight on specific issues related to CF, clinical research on CF treatments, and other issues related to the product and its review. We believe this contributed to a more efficient evaluation, a testament to what can be achieved when stakeholders collaborate across sectors to ensure a swift review of critical drugs for patients.

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How does EXPERRT differ from other FDA reform legislation like the FAST Act and the TREAT Act?

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. 

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Will this bill be considered on its own or will it be included in the Prescription Drug User Fee Act (PDUFA) reauthorization?

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.

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Why is the EXPERRT Act different from the new programs and initiatives outlined in the PDUFA Performance Goals negotiated agreement?

There are many important distinctions between the FDA’s negotiated PDUFA agreement and the EXPERRT Act. 

  • 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, while the PDUFA negotiated agreement focuses mainly on outreach to patient representatives. While input from patients on issues such as the risks and benefits of medications is critical, it is also important to have input from rare disease scientific experts on issues like genetically targeted treatments, clinical trial design, and unmet medical needs.

  • EXPERRT not only emphasizes outreach to scientific experts, it specifies the critically important topics on which these reviewers could seek input: disease severity, unmet medical needs, clinical trial challenges and issues, and demographics and the clinical description of patient populations.

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Doesn’t the PDUFA agreement also specifically increase expert collaboration with FDA reviewers?

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.

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There are many references to increasing collaboration with patient organizations and representatives in the negotiated PDUFA agreement.  Isn’t this the same thing as the EXPERRT Act?

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.

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Doesn’t the agreement already reference conducting public meetings on disease severity and unmet medical needs, a topic the EXPERRT Act specifically references?

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.

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Updated 4/10/2012