On April 17, more than 30,000 people ran, walked, or wheeled the famed 26.2-mile route from Hopkinton, Massachusetts to Boston. Among them were people who used the event to raise money for charities focused on finding new and more effective treatments for diseases such as cancer and Multiple Sclerosis.
Like a marathon, the road to winning approval for ground-breaking pharmaceuticals and innovative medical devices can be arduous. But the 21st Century Cures Act (Cures), which became law last December, has the potential to change that by modernizing development, evaluation, and approval processes for drugs and devices. The new administration’s focus on reducing regulatory burden could spur agencies toward a more collaborative and adaptive approach when approving and regulating therapies, and could prompt regulatory flexibility that also supports patient access and public safety.
As discussed in Deloitte’s forthcoming report, 21st Century Cures: The future of product innovation and approval, while the law has potential to become a monumental mile marker, there could be some early roadblocks with funding and shifting priorities. The law, which had strong support from both Republican and Democratic lawmakers, allocates $6.3 billion in funding for the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA) over the next 10 years, beginning in fiscal year (FY) 2017. The bulk of the funding ($4.8 billion) is directed to the NIH for cancer research, brain research, precision medicine, and regenerative medicine. That organization’s budget had experienced years of belt-tightening, but the new funding could change that. The law also allocates $500 million to the FDA to help the agency modernize systems that lead to faster drug and device approvals. Other provisions target health IT and certain public health priorities.
Congress appropriated the funding through Cures, but there are no guarantees. The budget blueprint released last month by the White House calls for the NIH budget to be cut by $5.8 billion for FY2018. Further, the current FDA user-fee program agreements — five-year pacts with drug, biologic, and device makers that provide about half of the FDA’s annual budget — expire this September. Without the user-fee funding, the FDA would likely have to let hundreds of employees go, leaving the agency short-staffed to approve new therapies.1 Without proper funding for FDA and NIH, Cures could lose momentum.
The new administration, however, is focused on shearing red-tape and reducing regulatory burden, which could encourage increased collaboration between FDA and other agencies involved in approving therapies. Congress is reviewing separate user-fee agreements for branded drug makers, generic drugs, biosimilars, and medical devices, and eventually plans to package them into a single bill for reauthorization.
During an April 5 confirmation hearing before the Senate Health Education, Labor and Pensions Committee, Scott Gottlieb, MD – the White House’s pick to head FDA – said one of his top priorities would be to accelerate the approval of new drugs, and use authorities granted by the Cures Act to “develop a template to lean forward” in using new technologies to treat disease.2
Cures gives patients a louder voice
Who is likely to know more about the effectiveness (or ineffectiveness) of treatments than the patients who live with the illness? Reliance on patient experience and real-world evidence (RWE) are integral themes woven throughout Cures. Through “patient-focused drug development,” the patient will likely have a louder voice in the drug approval process. The concept, which is not new to the life sciences industry, requires the FDA to issue guidance on how manufacturers and the agency can include patient experience in assessments of benefit and risk for drugs and devices. Allowing greater flexibility in the types of evidence used will likely allow these companies to take greater advantage of recent advances in data analytics.
Our conversations with life sciences stakeholders corroborate a growing reliance on the integration of RWE and patient experience data in drug development and approval. In many ways, the drug and device development and approval process of yesterday is over. Life sciences companies may risk being out-of-date if they fail to take advantage of the newer breakthrough, priority, or accelerated pathways of the future.
Strategies for life sciences companies
Some industry stakeholders are encouraged by provisions that clarify breakthrough device pathways, which builds upon the Expedited Access Program (EAP) that the FDA established in 2014 and published guidance on in April 2015. The EAP aims to reduce the time to develop a device and expedite access for patients with serious conditions whose medical needs are unmet by current technology.
Many issues will be top of mind for biopharma and medical device companies as they seek to capitalize on the evolving regulatory landscape. Leaders at these companies should likely consider:
- Engaging in early discussions with the FDA to design clinical trials that incorporate surrogate endpoints and other tools to shorten drug development timelines
- Expanding capabilities to access, collect, and analyze RWE and patient experience data
- Continuing to work with the FDA, patient advocacy groups, and provider organizations to delineate pathways for patient and caregiver involvement
- Expanding the dialogue on economic evidence between biopharma and medical device companies and health care stakeholders, including payers
- Taking advantage of additional regulatory clarity by investing in breakthrough devices, point of care diagnostics, drug-device combination products, and regenerative medicine
- Advancing the conversation on the regulation of medical software in collaboration with the FDA and other industry stakeholders
If treating diseases were a marathon, we’re still lacing up our shoes on many fronts. Of the thousands of illnesses that affect us — some estimates put the number at 10,000 or more — we have developed treatments for just 500 of them. The shift toward prioritizing new kinds of data, adopting a more patient-centered focus, and incorporating the value paradigm of patients, providers, and payers, will likely continue to shape the strategic choices life sciences companies make. As the industry strives to meet the evolving needs of stakeholders – patients, providers, and health plans – additional regulatory flexibility could help drive both approval and market access.
1 Derrick Gingery and Ferdous Al-Faruque, “A burning FDA hiring freeze question: What about user fee-supported staff?” Pink Sheet, January 24, 2017