We need to improve the pace of medical innovation. Not incremental change but real, game-changing progress.
Some disease-specific philanthropies, such as the Cystic Fibrosis Foundation, have taken matters into their own hands and stepped up to raise funds to support critical research for drugs to treat life-threatening diseases. But what about medical devices? Device makers face the same challenges as drug companies - tremendous costs, high risks for early research and increased demands from regulators and payers. It simply takes too long. And in the meantime, patients and families are Neal Kassell (Photo courtesy of suffering. Focused Ultrasound Foundation)
Innovative medical technologies move slowly from concept to widespread adoption as a standard of care. The process is complicated and inefficient and can consume decades of time before a single patient benefits. There are many steps, many stakeholders and far too many obstacles. Consider the gamma knife, a non-invasive treatment for brain disorders. Invented in 1951, the technology was commercially introduced in the United States 36 years later, in 1987.
Without intervention, a similar timeline might be the fate of focused ultrasound, a non-invasive therapeutic technology that is a cost-effective alternative or adjunct to surgery, radiation and drug delivery. (See a BBC video on how this technology works). This technology has the potential to transform treatment of many serious medical disorders, including tumors, Parkinson's disease, epilepsy, back pain and hypertension. With dozens of applications, focused ultrasound could one day improve and extend the lives of millions. It should be sooner rather than later.
Given the potential of focused ultrasound, we cannot just resign ourselves to the fact that revolutionary medical technologies face numerous scientific, regulatory, cultural and financial barriers that delay access and lead to unnecessary death, disability and suffering for patients around the world. For patients with unmet medical needs, the clock is ticking, and the stakes are high.
Can a philanthropic organization accelerate medical technology innovation and progress? The answer is yes.
We established the Focused Ultrasound Foundation in 2006 as the catalyst to accelerate development and global adoption of focused ultrasound. The Foundation embraces the principles of high-performance venture philanthropy in which goals are clearly defined and outcomes are measureable.
While the field is still in the early stages, the results have been impressive. The Foundation has raised more than $40 million to fund and advance the field. The technology is currently approved in the U.S. to treat uterine fibroids and relieve the pain of cancer in the bone. Outside the U.S., focused ultrasounds are used to treat more than 15 conditions, including breast cancer, pancreatic cancer and Parkinsonian tremor. Results from a clinical trial funded by the Foundation to treat essential tremor were recently published in the New England Journal of Medicine and paved the way for a larger study, an important step toward Food and Drug Administration (FDA) approval of focused ultrasound for treating this debilitating condition.
So what role can a foundation play in device development? Our Foundation uses capital to seed the highest-risk, early-stage research as proof of principle, encouraging follow-on funding from other sources that will sustain forward progress. Initial funding of 21 research projects at a cost of $2.2 million has led to follow-on funding of $19.2 million from a range of other philanthropies and the National Institutes of Health. That is a return on investment (ROI) that any investor would be proud of.
A New Model
The model established by the Foundation can serve as a blueprint for other philanthropic organizations seeking to accelerate medical innovation. A critical first step in setting the strategic direction for the field is mapping the entire development process for key applications and prioritizing those applications with the greatest clinical benefit. Once the development process is defined, barriers and obstacles that are likely to hinder progress must be identified. Reimbursement pitfalls plague the medical technology adoption pathway and must be acknowledged and addressed.
The mapping process must also encompass the diverse set of stakeholders and organizations that need to be aligned to ensure forward movement at an acceptable pace. Historically, the pathway from concept to adoption of medical technologies has been slowed by misaligned agendas and timelines of researchers, clinicians, government agencies and industry.
When patients are waiting, an iconoclastic model is needed. The barriers and silos that have traditionally defined academic research and commercial models must be removed. Collaboration must be favored over individual glory. A patient-centric ethos must trump investigator-centric attitudes. The drive to develop products that have potential for commercial success must take precedence over generating publications, grants and promotions. The collective culture of the global community must reflect an entrepreneurial, can-do attitude where there is a shared enthusiasm and energy to precipitate change.
Once barriers are removed, silos dismantled, and stakeholders mobilized, meaningful exchange of ideas and insights will begin to take root. By facilitating this collaboration across stakeholders, innovation is sparked, and a philanthropic organization can become a true "force multiplier" for intellectual capital, dramatically increasing the effectiveness of the group. True collaboration also fosters a shared sense of urgency and helps minimize wasteful duplication of effort.
The need for private philanthropy to accelerate medical innovation has never been greater and addresses a critical gap that industry and government cannot fulfill. Government funding levels are stagnant, and the slow, bureaucratic, risk-averse approach is not compatible with getting game-changing treatments to patients in need in the shortest time possible. On the industry side, funding for research and development is shrinking, and investment in disruptive technologies might not be immediately profitable.
Today's philanthropists can make a difference, but certainly have more expectations than ever on their ROI. Many recognize that unmet medical needs persist, but they must see results. Philanthropists will be attracted to milestone-driven, results-oriented foundations that offer a well-defined strategic roadmap. Regular updates, progress reports and a shared belief of what is possible will engage the kind of donors who can help turn a vision into reality.
While the "to do list" for a philanthropic organization seeking to advance a medical technology might seem intimidating, the effort and commitment are essential for patients in need.
Neal F. Kassell, MD, is founder and chairman of the Focused Ultrasound Foundation based in Charlottesville, VA.