Opinion: Patient advocacy groups and innovators must partner to advance new treatments

People living with debilitating conditions and life-threatening diseases are often put in the terrible position of having to wait — even though they lack the luxury of time — for scientific advances that take years to reach the market, preventing them from accessing their much-needed benefits.

A remedy is coming from an unexpected and long-underutilized source: partnerships between patient advocacy groups and pharmaceutical and medical device companies.

Patient advocacy groups have long helped researchers find participants for clinical trials and consulted with regulators on pressing needs for their constituents. But they must now play a more active, integral, and permanent role in accelerating — and funding — tomorrow’s innovations.

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Novel partnerships between advocacy groups and developers are emerging as the driving forces behind this kind of innovation. These relationships can more effectively propel solutions toward the market. Their success lies in creating collaborative frameworks designed to overcome traditional hurdles and accelerate the pace of progress.

Such partnerships create face-to-face understanding that speeds and better directs drug and therapy development by allowing health-sector companies the opportunity to draw on the deep knowledge and personal connections of patient advocacy groups. They know what’s important to patients and what will provide the biggest impact in their lives. Through collaboration, advocates and innovators — whether in academia or the corporate world — can ensure that those with lived experience of disease or disability inform the creation of products that are fit for purpose by contributing input all along the way, from research and development to market rollout.

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Although collaborations between advocacy groups and health-sector companies that serve the needs of their patients are already moving forward, they must keep pace with unmet need. Last year, The Michael J. Fox Foundation saw the FDA approve Kynmobi, a treatment for Parkinson’s disease funded by the foundation that was developed by Sunovion Pharmaceuticals. Two years earlier, Inbrija, another Parkinson’s treatment developed with foundation funding, received FDA approval. In October 2020, the organization partnered with Acadia Pharmaceuticals, which makes a treatment for the hallucinations and delusions associated with Parkinson’s disease psychosis, to provide educational resources to people with Parkinson’s and their care partners on navigating the cognitive changes wrought by the disease. All are examples of benefits to patients brought about through collaboration and investment of expertise and resources.

In a similar fashion, the Christopher & Dana Reeve Foundation, which I help lead, is investing in science and technology companies, a critical effort in achieving our mission of curing spinal cord injury by advancing innovative research. It’s imperative to speed the pace of advancement for those living with spinal cord injuries because they have not benefited from major advancement in decades.

Many believe that for people living with spinal cord injuries, “recovery” is defined first and foremost as regaining the ability to walk. But the repercussions of spinal cord injury go beyond that and recovery has different degrees. Many of those living with spinal cord injuries hope to normalize their blood pressure, or regain bowel, bladder and other affected bodily functions, for a more self-reliant, healthier life. For most, the top priority is recovery of use of a hand and arm, which translates into meaningful, quality-of-life improvements: being able to independently eat, dress, work, and perform other daily activities.

To help speed the development of new therapies for people living with spinal cord injuries, my organization has partnered with Onward, a Netherlands-based company that has developed breakthrough technology currently in global clinical trials that delivers targeted, programmed stimulation of the spinal cord to restore movement and other functions. That partnership includes financial support to help bring these technologies to market faster to help our patients.

Deep partnerships between patient advocacy groups and drug and device developers represent a truly novel approach to funding discovery and innovation. While it won’t supplant alliances with venture capital investors — who remain essential for providing funding, energy, and business acumen — these partnerships provide more productive guidance to investing and more palpable energy to discovery. They bring specificity of knowledge and experience that sharpen the focus of innovators so their discoveries and creations are more useful, targeted, and effective.

For companies that aim to address unmet needs, collaboration with patient advocacy groups shakes up traditional innovation models by putting them in direct contact with patients and advocacy group resources. Partnerships also provide new ways for nonprofits to work more closely with drug developers to advance tomorrow’s treatments sooner. The commercial and logistical expertise of partner companies can fast-track bringing treatments and devices to market.

These expanded partnerships are a win-win-win for patient advocacy groups and companies and, most of all, for the people waiting for scientific advancements to improve their lives.

Margaret Goldberg is the president and chief operating officer of the Christopher & Dana Reeve Foundation.

Source: STAT