Monday, July 20, 2015
Stanley Bergen Building, Rutgers University, Newark, NJ
The NIH innovation fund would raise NIH funding to $31.8 billion in fiscal 2016 and to $34.85 billion in fiscal 2018. Congress appropriated $30.3 billion for the NIH in fiscal 2015, and President Barack Obama requested (PDF) $31.3 billion for fiscal 2016.
The bill would also create an NIH Innovation Fund of roughly $2 billion per year between fiscal years 2016 and 2020. The fund would support precision medicine, young emerging scientists and other unidentified sources of medical innovation.
Research!America, said it was “thrilled” by the new version of the bill but warned that the final legislation must allow the NIH to decide how to allocate the dollars.
From FDA Law Blog by James E. Valentine:
Precision Medicine: Congress finally provided some detail about precision medicine in the second version of the 21st Century Cures Act (Sec. 2041). While the second version of the bill provided a definition of precision medicine, the latest version would have FDA define the term. The revised provision would require FDA to issue a guidance document to assist sponsors in the development of such drugs.
This provision also allows for applications of a precision medicine that has been designated “for a rare disease for a serious condition” to rely upon data or information previously developed by the sponsor for a prior approved drug or indication in order to expedite clinical development of a precision medicine or indication that is using the same or similar approach as that of the prior approved drug. In addition, the provision specifies that applications for precision medicines should be considered as to whether they are eligible for accelerated approval.
From Alexander Gaffney in a RAPS article:
Title II of the 21st Century Cures legislation seeks to build on FDA’s recent launch of a Patient-Focused Drug Development (PFDD) program. That program, launched in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), is meant to incorporate patient preferences into FDA’s regulatory decision-making. The idea behind the proposal is that different patients—even those suffering from the same disease—have varying levels of risk they are willing to accept.
As legislators explained in a white paper accompanying the last draft of the bill, “No one understands a particular condition or disease better than patients living with it.” Accordingly, the bill would require FDA to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.
That measure is likely to win support at FDA, which has sought to develop similar approaches in the past and has been receptive to patient feedback. Under the approach, FDA would have to, within two years, develop and implement a process to allow companies to submit “patient experience data,” which could be used to develop a “structured risk-benefit assessment framework.” A draft guidance document would also be due from FDA within two years.
From The EveryLife Foundation for Rare Diseases:
We are urging advocates to thank Congress for their hard work in assembling this historic legislative package.
This action alert will send an email to thank Congress for including rare disease priorities into the 21st Century Cures legislation.
This Sign-On Letter from PhRMA comments on patent reform legislation being considered in both the House and the Senate, and asks that Congress maintain a strong and secure patent system that address abuses that have emerged at the PTO. With Congress moving quickly to advance comprehensive patent reform, the deadline to sign is Monday, May 18, 2015. If this is also a priority for your organization, please contact Lauren Neff to sign-on.
Research America invites you to Sign-on to this Letter in support of the $10 Billion NIH Innovation Fund, which is included in the draft of 21st Century Cures legislation. Because the bill is scheduled to be marked up on Thursday, May 14th, the deadline for signing on is tight. Please let Ellie Dehoney by noon Tuesday, May 12th. If you are interested in signing on, please provide you Organization’s name as you would like it to be listed.
We hope that your organization will consider signing the attached letter to the House and Senate Judiciary Committees expressing support for balanced patent reform that strengthens the current system.
Support for the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments) on Capitol Hill is growing. The bill provides incentives for companies to repurpose or “rarepurpose” existing treatments for rare disease indications. The legislation was included in the draft 21st Century Cures discussion bill released by the Energy & Commerce Committee. This is a major positive development as Chairman Upton (R-MI) has expressed interest in having the legislation voted on by Memorial Day.
In addition, Reps. Bilirakis (R-FL), Butterfield (D-NC), and McCaul (R-TX) have co-sponsored the stand-alone version of the OPEN ACT, which was officially introduced on Friday, February 13th as HR 971. The bipartisan bill introduction was in concert with strong support from the patient community, with 135 organizations signing-on.
The EveryLife Foundation is still looking for supporting patient organizations to support. Please contact Max Bronstein – email@example.com to sign-on today.