On January 27, 2015 the House of Representatives Committee on Energy and Commerce released a discussion draft of a bill entitled, “21st Century Cures Act.” The bill includes several proposals that are intended to expand patients’ access to treatments and promote drug and device development, such as modifying processes at the Food and Drug Administration (FDA) and the National Institutes of Health (NIH), and revising several Medicare coverage and reimbursement requirements.
The Senate Committee on Health, Education, Labor and Pensions is engaged in a complementary effort to improve the development of drugs and devices, and on January 29 it released a report entitled, “Innovation for Healthier Americans.” Unlike the House discussion draft, the Senate report does not include specific recommendations. Rather, it contains an assessment of the current process for drug and device development to determine which processes are working well and which processes can be improved.
The committees have solicited feedback from stakeholders. This bicameral initiative presents an opportunity for the public to participate in the development of legislation in this area, and to provide feedback on improving and expediting the processes for developing medical products and bringing them to market.
- FDA Approval Processes: Several initiatives in the bill are aimed at expanding patients’ access to therapies. Examples of key proposals in the House discussion draft are described below:
- Drug Approval Process: FDA would be required to establish a structured risk-benefit assessment framework for the new drug approval process. FDA would be required to establish and implement a process to facilitate the incorporation of patient experience data into the regulatory decision-making process, including a risk-benefit assessment framework for new drugs. In addition, FDA would establish a program under which a sponsor of a drug could submit “real-world” evidence to support the approval of the use of a drug for a new indication and to support or satisfy post-approval study requirements.
- Approval of Breakthrough Drugs and Devices: FDA would be permitted to approve a drug that has been designated as a breakthrough therapy for a serious or life-threatening disease or condition when early stage clinical safety and effectiveness data provides sufficient evidence for the approval of the drug as safe and effective. Similarly, the bill would establish a process at FDA for the designation and expedited review of devices that represent breakthrough technologies with the potential to address unmet medical needs.
- Incentives for Establishing Drugs and Other Treatment Options: The bill would create new incentives to establish drugs intended to be used to address unmet medical needs (i.e., patent term extensions). It would extend exclusivity for two years for significant improvements existing to molecules (i.e., new delivery systems, new drug combinations, and new formulations that lead to less adverse events and increase patient benefits and adherence). In addition, the bill would provide six months of additional market exclusivity for a drug if the manufacturer establishes that the drug treats a rare disease and receives a rare disease indication from the FDA for its label.
- Expanded Access: The bill would establish transparency requirements for certain drug companies that are related to their programs that provide patients with access to drugs before they are approved. It also would create an expanded access task force charged with recommending additional reforms that would improve patients’ access to investigational drugs.
- Regulation of Medical Software: The bill would define “medical software” and “health software,” require the Secretary of the Department of Health and Human Services (HHS) to establish a new regulatory process for “medical software,” and exclude “health software” from the definition of “device” and from regulation under the Act.
- Medicare Coverage: Congress recognizes that Medicare coverage of various treatment options is essential for beneficiaries to have access to the treatments. Examples of proposals in the discussion draft that would revise Medicare coverage requirements are listed below:
- Coverage of Breakthrough Devices: The discussion draft includes a placeholder for a section entitled “CMS Coverage of Breakthrough Devices.” Although there is no draft language under that section, the bill’s summary indicates that the section is intended to provide Medicare and Medicaid transitional coverage benefits for breakthrough medical devices that have the potential to address unmet medical needs that are approved or cleared by FDA.
- Coverage with Evidence Development: The bill would authorize Medicare to cover items and services that are the subject of clinical trials for beneficiaries participating in the trials.
- Medicare Coverage of Disposable Medical Technologies: The bill would provide Medicare coverage and payment for certain “disposable medical technology,” defined as, “medical equipment that – (1) is primarily and customarily used to serve a medical purpose; (2) would otherwise be covered as durable medical equipment under this title but for the fact that such equipment is not durable…, and (3) the Secretary determines substitutes for durable medical equipment.” Reimbursement for the disposable medical technology would be a single payment amount determined in accordance with a calculation set forth in the bill. The competitive bidding program would not apply to such items.
- Coverage for Telehealth Services: The bill would require Medicare to provide for coverage and payment of telehealth services identified by the Secretary of HHS through a rulemaking process.
- Local Coverage Determination (LCD) and National Coverage Determinations (NCD) Reforms: The bill raises questions as to whether there are ways in which the NCD/LCD process can work better for the administration and for those seeking coverage under Medicare.
- Medicare Pharmaceutical and Technology Ombudsman: The Secretary of HHS would be required to provide for a pharmaceutical and technology ombudsman within the Centers of Medicare & Medicaid Services (CMS) to receive and respond to complaints, grievances and requests from manufacturers of pharmaceutical, biotechnology, medical device, or diagnostic products that are covered under Medicare, or for which coverage is being sought, regarding Medicare coverage, coding, or payment for such products.
- Continuing Medical Education Sunshine Exemption: The bill would specifically exempt peer-reviewed journals, journal reprints, journal supplements and medical textbooks from the manufacturer transparency reporting requirements. It would also exempt “a transfer of anything of value to a covered recipient who is a physician if the thing of value is intended solely for purposes of providing continuing medical education to the physician.”
- Medical Product Innovation Advisory Commission: The bill would create the Medical Product Innovation Advisory Commission, which would be responsible for analyzing medical product innovation and recommending policies (including policies of NIH, FDA and CMS) to accelerate the discovery, development and delivery of new medical products.
- 21st Century Chronic Disease Initiative Act: The bill would require the Secretary of HHS to develop a plan to carry out a longitudinal study designed to improve the outcomes of patients with chronic diseases through better understanding of risk, transition from wellness to disease, disease progression, diagnosis, and other factors related to chronic disease, including identifying potential targets for preventive or therapeutic intervention.
- Drug Manufacturing Reforms: The bill includes several proposals related to manufacturing drugs:
- Incentives for Generic Drugs Manufactured in the United States: The bill would extend the exclusivity period for American-manufactured generic drugs and biosimilars. The discussion draft includes a placeholder for the length of the extension.
- Good Manufacturing Practices: Within one year of enactment, FDA would be required to consider modern manufacturing techniques and issue final regulations and guidance updating the good manufacturing practice requirements for drugs.
- Controlled Substance Manufacturing and Exports: The bill would permit controlled substances to be re-exported among members of the European Economic Area.
- Medical Device Reforms: The bill includes several proposals related to medical devices, which are highlighted below:
- Manufacturer Opt-Out Program for Medical Devices: Manufacturers of medical devices would be able to designate certain medical devices as non-covered list, which would preclude insurers from providing for coverage or reimbursement of the devices.
- Third-Party Quality System Assessment: The bill would enable FDA to rely on third party accreditation organizations to certify certain manufacturing changes.
- Valid Scientific Evidence: The bill would define valid scientific evidence, which is one of the standards by which FDA approves Class VII medical devices, to include evidence described in certain well-documented case histories, including registry data, as well as studies published in peer-review journals. It would specify that FDA may consider data collected in countries outside of the United States as valid scientific evidence.
- Training and Oversight in Least Burdensome Means Concept: The bill would require FDA to train its employees involved in the review of premarket submissions under section 515 or section 510(k) of the Federal Food, Drug, and Cosmetic Act on the least burdensome means concept, and to ensure that adequate guidances that describe the concept and its implementation are available.
- Recognition of Standards: The bill would revise the process of government recognition of appropriate performance standards.
- Notification of Marketing Certain Class I Devices: The bill would also modify the process for marketing Class I medical devices.
- General and Specific Uses: The bill would modify the 510(k) process for medical devices.
- Supply Chain Security for Devices: The bill would establish a national framework for licensing medical device wholesalers and third –party logistics providers.
The 21st Century Cures Act is available here.
The Senate report is available here.