On Thursday, May 19, 2015, the Energy and Commerce Committee of the House of Representatives voted 51 to 0 to approve the 21st Century Cures Act (“the Cures Act”) which would, if passed by the full House and Senate and signed by the President bring about a number of significant changes in NIH research funding, the process of drug development, and the FDA review of New Drug Applications (“NDAs”). Because the current draft is 300 double-spaced pages in length the focus in this post will be on just six of those pages--“Sec. 2001. Development And Use Of Patient Experience Data To Enhance Structured Risk-Benefit Assessment Framework.” While there are likely to be some changes to the current draft, given the overwhelming bipartisan support in the House, it seems that much, if not all, of the current draft will become law.
Section 2001 of the Curs Act consists of two subsections, currently denominated (x) and (y). Section 2001 would appear to require very little change to the current approach of the FDA to drug approval. Section 2001 Subsection x (1) states that “The Secretary shall implement a structured risk-benefit assessment framework in the new drug approval process [A] to facilitate the balanced consideration of benefits and risks; and [B] to develop a consistent and systematic approach to the discussion of…the benefits and risks of new drugs.” The current draft of Sec. 2001 Subsection (x) 2 contains the proviso that “Nothing in Paragraph (1) “shall alter the criteria for evaluating an application for premarket approval of a drug.” The apparent focus of this subsection (x) is to bring greater clarity and consistency to the FDA’s weighing of risks versus benefits. This is an unobjectionable goal, if somewhat difficult to implement. For example, one cancer drug may cause severe skin reactions while increasing overall survival by one month, while another drug for the same form of cancer may cause severe gastric effects while increasing overall survival by 1.2 months. Comparing the risks and benefits of those two drugs is obviously difficult to do. It is that difficulty that may be the objective of the much more interesting Subsection (y).
Subsection (y) is entitled “Development And Use Of Patient Experience Data To Enhance Structured Risk-Benefit Assessment Framework.” Subsection (y) 1 requires the Secretary of HHS, over a period of 2 to 3 years, to a promulgate a number of guidances and establish procedures by which non-NDA sponsor entities may submit patient experience research proposals for feedback from the Secretary as well as patient experience data and data analyses. Patient experience data, or, as it is also known, patient-reported outcomes data has been a field of interest in clinical research for a number of years. Patient-reported outcomes was the subject of an FDA Guidance issued in 2009 (“2009 Guidance”). The 2009 Guidance recognized the value of assessing patient experience endpoints in clinical trials and encouraged patient input in developing the instruments to be used in assessing patient reported outcomes or experience:
Item generation should include input from the target patient population to establish the items that reflect the concept of interest and contribute to its evaluation. The population will help generate item wording, evaluate the completeness of item coverage, and perform initial assessment of clarity and readability.
However, the approach to patient experience data in the Cures Act goes well beyond the 2009 Guidance. Subsection (y) provides the Cures Act definition of patient experience data:
In this subsection, the term ‘patient experience data’ means data collected by patients, parents, caregivers, patient advocacy organizations, disease research foundations, medical researchers, research sponsors or other parties determined appropriate by the Secretary that is intended to facilitate or enhance the Secretary’s risk-benefit assessments, including information about the impact of a disease or a therapy on patients’ lives.
The statutory inclusion of data collected by “patients, parents, caregivers, patient advocacy organizations, [and] disease research foundations” is radically different from the drug-sponsor driven approach that was addressed in the 2009 Guidance. Bringing data collected by persons or entities other than the drug sponsor into the New Drug Approval process (or the sNDA process of approving additional indications or major label changes) would be a fundamental change in the concept of data to be used by the FDA in new drug review and would significantly increase the role of patients and patient organizations in the drug approval process itself. If so, the 21st Century Cures Act’s effect on drug development might be as dramatic as the title of the Cures Act suggests.