An obscure but important piece of legislation has been moving quickly though Congress in recent months and it offers important promise for helping to accelerate access to new and innovative treatments for serious mental illness. The legislation, known as the Food and Drug Administration Safety and Innovation Act (S 3187), cleared the House June 20, and is expected to soon pass the Senate and move onto the White House where President Obama has pledged to sign it into law.
What is surprising is that the bill has enjoyed strong bipartisan support – uncommon in the current hyper-partisan environment, especially over an issue involving health care. Nonetheless, Democrats and Republicans came to consensus in recent months over a range of difficult issues including speeding patient access to breakthrough therapies and addressing shortages of prescription drugs (including medications to treat ADHD).
The bill includes reauthorization of a range of user fee programs that allow brand name and generic drug makers and medical device manufacturers to accelerate reviews of new products. The largest of these is the PDUFA V agreement, which will provide the FDA with $700 million in funding for FY 2013 and even more over the following four years of the agreement. These user fees are an important part of ensuring that the Food and Drug Administration (FDA) has the resources needed not only to conduct reviews of new products, but also contact post-marketing reviews of products currently on the market.
Throughout the deliberations on S 3187, NAMI joined other patient and disease advocacy groups to press for inclusion of provisions to speed access to breakthrough therapies, address drug shortages, improve an existing program to conduct trials of medications in children, ensure the integrity of the drug supply chain and improve the performance of FDA advisory panels. In addition, the law requires FDA to more effectively engage patient and disease advocacy groups in agency deliberations – an opportunity NAMI intends to fulfill.
Included below is a summary of major provisions in the Food and Drug Administration Safety and Innovation Act of concern to NAMI.
As noted above, S 3187 includes the PDUFA V agreement. NAMI was pleased to take part in the Patient and Consumer Stakeholder Group that has worked with the FDA throughout this PDUFA V process. Among the key improvement in PDUFA V are:
1. Performance Goals -- NAMI strongly supports the new performance goals that will increase drug review efficiency and predictability and result in more consistent and transparent drug reviews. This Agreement will also more clearly discern how efficacy and safety parameters are balanced as part of the FDA’s risk-benefit analysis.
2. Enhanced Benefit Risk Assessment -- The Agreement should bring greater transparency to the benefit-risk assessments FDA makes that have been opaque and difficult to understand, especially when decisions have been made across multiple divisions and offices. These improvements should also bring more transparency to this process.
3. Electronic Submissions and Standardization of Application Data -- This new requirement for electronic submission and standardization of application is long overdue and is central to making the overall review process more timely, efficient and less costly.
4. Independent Review of PDUFA Performance Goals -- In order to ensure that the PDUFA V goals are met, there must be an independent third party assessment, with full opportunity to all stakeholders to offer comments on the assessment.
5. Regulatory Science Improvements -- NAMI supports the new investments in the Agreement aimed at improvements on pharmacogenomics and biomarkers. The application of qualified biomarkers has enormous potential to accelerate drug development by helping to identify and predict which patient will respond to a particular medication. Biomarkers offer particular promise with respect to treatment for schizophrenia. FDA needs to be able to access new resources to augment its clinical pharmacology and statistical capacity to better address submissions that propose to utilize biomarkers or pharmacogenomics.
6. Patient Reported Outcomes (PROs) -- PROs can be critical endpoints for measuring the benefit-risk profile of a new treatment from the patient’s perspective. NAMI is pleased that the Agreement includes advancement and validation of PROs. This should help improve the agency’s clinical and statistical capacity to address submissions involving PROs and other end point assessment tools.
7. Patient Safety Improvements, REMS & Sentinel -- NAMI supports the improvements in PDUFA V to enhance FDA’s existing safety system. The improvement to REMS and Sentinel are vital to this ongoing effort to ensure patient safety. These improvements should allow FDA to test the feasibility of using Sentinel for evaluating drug safety issues that may require some form of regulatory action such as a label change.
NAMI is pleased that this legislation includes permanent extension of both the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. BPCA and PREA have been remarkably successful in ensuring that medications used in children are tested and labeled appropriately for pediatric use. As a result of BPCA and PREA, the incentives that allow for appropriate drug development studies to be conducted in children have been successful, with more studies conducted over the past 10 years than were conducted over the previous 5 decades. As a result, 426 drug labels have been revised with new pediatric information and an estimated 50% of drugs used in children are now studied as compared to just 20% before the enactment of these two laws. NAMI supports permanent extension of both BPCA and PREA.
Title IX contains a number of provisions that NAMI strongly supports to put in place new processes for expediting the development and review of medications intended to treat serious and life threatening diseases. Many of the important provisions in Title IX have been included in previous bipartisan bills including the MODDERN Cures Solution Act and the Advancing Breakthrough Therapies for Patients Act.
1. Expedited Approval for Serious or Life Threatening Diseases or Conditions – NAMI would urge the HELP Committee to consider additional clarity with regard to the definition of surrogate endpoints for complex conditions such as schizophrenia where research has demonstrated irreversible morbidity related to acute episodes of psychosis.
2. Breakthrough Therapies – NAMI supports this provision and recommends that the Committee consider further clarifying the scope of language defining breakthrough therapies and requiring a demonstration of “substantial improvement” over existing therapies on clinically significant endpoints, such as substantial treatment effects observed early in clinical development. In moving forward, it will be important for this definition to integrate improvement over existing therapies not only with respect to clinical symptoms, but also serious side effects associated with existing therapies.
NAMI is pleased that Title X of the bill includes a range of important reforms to ensure greater accountability and transparency with respect to shortages of prescription drugs. NAMI has always place a priority on ensuring that people with mental illness are able to access the treatment they need. Acute shortages of medications can operate as a substantial barrier – particularly with psychotropic medications that are not clinically interchangeable. Provisions in the bill instituting new advance notification requirements for manufacturers before supply disruptions actually occur will help patients and prescribers better cope with shortages. In addition, new authority in the bill for the Secretary to work with the FDA to evaluate the risks of future shortages and take immediate actions to avoid them in the future will also help prevent future shortages.
Title X also includes a number of provisions designed to address shortages of medications to treat Attention Deficit Hyperactivity Disorder (ADHD). Ensuring adequate availability of product is a particular challenge for these medications as the Drug Enforcement Administration (DEA) plays a critical role in regulating manufacturing capacity of both the immediate release and extended release versions. This is required as amphetamine is regulated as a Schedule 2 controlled substance. In particular, this legislation includes new provisions to integrate DEA into the new federal oversight structure that will work to monitor and prevent shortages and ensure greater accountability on the part of the DEA with respect to consideration of allocation of increased manufacturing quoa.
Section 1142 of the bill contains improvements to streamline the process for appointments to FDA advisory panels. Reforms such as eliminating the cap on the number of conflict of interest waivers the Secretary can grant are long overdue. In addition, simplifying the process will enhance recruitment and ensure greater transparency when waivers are granted. These reforms will help ensure greater balance in FDA advisory panels and help the agency attract needed clinical expertise to ensure that it is making decisions based on the best scientific expertise available.
June 21, 2012
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