Partnership Strategies With Orphan Disease Advocacy Groups

“Orphan” is a very accurate descriptor of the abandonment and despair that haunts many people living with rare diseases. But many of these extraordinary individuals find the strength to build their own support networks that help manage their disease and improve their overall quality of life. That’s why patient advocacy groups are necessary and important partners with significant implications and advantages for life science companies.

Patient advocacy organizations (PAOs) belong to the orphan disease space like daylight does to the sun, and have become a critical business partner in the drug development industry. This collaboration—which has evolved over the past decade from that of a silent partner to one that takes a seat at the senior management table—offers biopharma companies an opportunity to view all elements of their business along the development continuum through the eyes of the patient.

The math is simple: If the prevalence of a disease is low, there are fewer candidates for clinical studies, market research, and awareness targeting. Creating an integrated relationship with advocacy groups enables industry partners to identify clusters of patients who can influence the direction of treating rare diseases through their first-hand knowledge of a specific illness. Membership in NORD’s well-known Corporate Council counts as one way of fostering relationships.

The Patient as the Ultimate Compass

The first step for biopharma companies wanting to work with PAOs is to seek alignment with potential partners. Within the advocacy community, there can be some questions about how best to work with commercial enterprises, and it is up to industry partners to demonstrate a willingness to support PAO priorities. Importantly, the mindset should be one of discovering shared goals rather than of projecting one’s own pre-existing goals externally. Conflicting goals will poison any relationship. In our experience, aligned goals written down explicitly are a wonderful starting point for successful cooperation.

This alone isn’t enough, though. Another important factor: Avoid competing activities. Rather than offering overlapping and redundant services, it would be more strategic and a better use of valuable resources when each side plays to their strengths. For example, in clinical trial support and development, PAOs are better positioned to bring trials to the patients and patients to the trials, while companies can consult with the group and engaged individuals on how to enhance the patient journey—while keeping the science rigorous and stringent.

When industry and advocacy enter into a relationship, it can only benefit the two if their activities complement each other, and if together they can achieve things that wouldn’t be possible without the cooperation. Sometimes, a partnership is bigger than the sum of its parts, and that’s the sweet spot we should be looking for, so both sides are better off. Or rather: So all sides are better off. The ultimate litmus test for industry-advocacy cooperation is: Does this benefit the patients we serve? If not, go back to the negotiating table.

Long-Term Commitment and Credibility

Biopharma can support orphan disease patients and their advocacy organizations in a big way by providing missing resources. This can be as simple as providing current information and staying in regular contact with PAOs, or offering financial support in the form of sponsorships or unrestricted grants. Whatever strategic direction the company decides to take, the impact can be very tangible. In order to ensure that such efforts are in line with an industry partner’s business goals, questions that need to be asked include:

  • Are the goals of this PAO aligned with ours?
  • Can both of us together achieve something that each of us individually cannot?
  • Does the PAO have the capabilities and the reputation needed to put our assistance to good use?
  • Is our corporate culture compatible with this engagement? Are we sincere enough? Are we trusted enough?

A company that sustains honest relationships continuously will have greater success in establishing and maintaining a strategic advocacy engagement than one that repeatedly starts new, short-lived initiatives without follow-ups and without delivering on promises. Historically this seemingly random “stop-and-go” behavior was quite common in our industry, and did little to dispel unfair prejudice against drug companies.

But there are also great examples of sustained and successful engagements that benefit the entire trifecta—industry, advocacy, and patients. One that instantly springs to mind is MS LifeLines, an initiative that has successfully kept patients motivated, empowered, and adherent for well over a decade. At the end of the day, it’s not even the cooperation per se that’s important, but the achieved result: That patients can rely on each cogwheel to help drive the healthcare machine.

Navigating an Ocean of Non-profits

According to the Urban Institute, well over 85,000 tax-exempt, health-focused non-profit organizations are active in the United States. This sizeable industry has reported over $405 billion in revenue over two years. These numbers alone are already impressive, but they only count the portion of non-profits that are registered with the IRS. PAOs address needs that would otherwise not be met: Providing or advocating for financial assistance, public policy, access to medical care, legal assistance, information, and emotional support. That’s why they form such an important part of the healthcare fabric.

In common chronic or terminal diseases such as cancer, heart disease, and diabetes, there are established PAOs—organizations equipped with the partnerships, funding and professionalism needed to adequately fulfill their roles (which is not to say there aren’t any unmet needs in those disease areas). For people with orphan diseases and their families however, we see a host of passionate individuals who run advocacy. They often lack the funds, the connections, the experience, and the manpower to really be effective. They may move more quickly, because they have less bureaucratic overhead than the big organizations, and sometimes they are more focused than larger umbrella groups.

But they also often struggle daily to maintain their organization due to a lack of adequate resources. As a result, patients seeking support sometimes end up with outdated doctor lists, incomprehensible disease information, or resources that are otherwise inadequate to help them manage their own or their loved-one’s disease. Also, as in the for-profit sector, PAOs of all statures often compete with one another and have an agenda beyond simply helping the patients—this increases the difficulty of navigating the ocean of non-profits, and the need for a reliable advocacy compass.

The reason policymakers have given orphan diseases special status is to help advance the development and accessibility of effective and safe therapies for patients. These government-sanctioned inducements have encouraged many biotech operations to search for the right molecule for the right rare disease, shouldering economic risks with the prospect of maybe being able to help people who have little else to pin their hopes on. It would be a major missed opportunity for life sciences, advocacy, and the rare disease patient community if a lack of cooperation between biopharma and PAOs prevented such therapies from getting to the people who need them.

  • Kathy Gram

    Kathy Gram is a VP at Snow Companies. Kathy’s entire career has been focused on healthcare, with roles in sales, product launches, biotech distribution, medical market research, and advocacy relations. For the past eight years she served in a senior management patient advocacy capacity with Millennium Pharmaceuticals and Takeda Oncology.

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