Laying the Ground Rules: Crafting Guidelines for Rare Disease Patient Organizations

Patient organizations and drug companies have a symbiotic relationship. Both want to see new therapies reach the market and by collaborating, they can better meet the needs of patients and accelerate the process of drug discovery and development. But as patient organizations have grown more sophisticated about both the science and business of drug development, they are increasingly recognizing the need for improving the transparency of their relationships with industry, setting the ground rules upfront as how they will interact, and what they will and won’t do.

A 2017 study in the New England Journal of Medicine raised concerns about potential conflicts of interest at patient organizations found that of 104 of the largest U.S.-based patient-advocacy organizations, at least 83 percent received financial support from drug, device, and biotechnology companies. Some 39 percent of those organization also have a current or former industry executive on the governing board. And as regulators push for patient involvement in the drug development process, the potential for conflicts grow greater.

The International Fibrodysplasia Ossificans Progressiva Association, or IFOPA, took the unusual step to craft a set of guidelines for the organization regarding how it engages with the pharmaceutical industry. It made these guidelines public and continues to refine them. Last year Betsy Bogard, chair of the IFOPA research committee, discussed on a RARECast podcast why the organization created the guidelines and how they have affected interactions between the organization and industry.

Now Bogard and eight other authors from both industry and patient groups (including Global Genes, publisher of Rare Daily), offer a set of guidelines for rare disease patient organizations in Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases in the Orphanet Journal of Rare Diseases. The authors, serving as an expert panel, provide guidelines in four areas: Identification and Engagement with Companies, Patient Engagement and Patient Privacy, Financial Contributions, and Clinical Trial Communication and Support.

The article notes that while documents exist to guide the interaction between patient groups and industry, by and large they have been created for the biopharmaceutical industry or are specific to the European healthcare system. Others, they note, are too general to provide a roadmap for day-to-day decision-making.

The panel, after reviewing existing documents, began with the IFOPA’s guidelines and revised them as guidelines that could be adopted by other patient advocacy organizations. An appendix to the article contains the full guidelines, which were finalized in October 2017.

“Mutual respect is essential, which requires honesty and authenticity,” the authors write. “Transparency and commitment from both parties should begin on day one.”

There’s nothing radical in the guidelines. If anything, they seem a bit tame at times using soft language and gentle phrasing. Rather than set firm rules, they suggest best approaches. For instance, in the section Patient Engagement and Patient Privacy, rather than establishing firm rules, it says “Direct interactions between specific patients and biopharmaceutical companies are best arranged with the involvement or general awareness of the patient advocacy organization.”

Similarly, it says in the financial, rather than saying groups should only accept unrestricted funds or funds to sponsor a specific activity initiated by the patient advocacy organization, the guidelines said doing so would be “ideal.”

What is radical, though, is the document as a whole. It’s a statement to drug companies that patient advocacy organizations want to be treated as equals and that they will be the ones to decide how they will engage with industry.

It’s remarkable to think that most patient advocacy groups don’t have such guidelines in place. Rather than having clear principals how they will engage with industry, what they will require from their partners, and under what circumstances they will accept money, most organizations have taken an ad hoc approach, only to later run into problems when something doesn’t go as they would like, or they realize they are engaging in work that is not in line with their mission because money was available.

These guidelines offer a starting point for patient organization seeking to put their own in place. It will help them think through ethical issues before a dilemma arises. Having such guidelines will help professionalize their work, maintain transparency, and keep the interests of the patients they serve top of mind. All of that should make an organization a better partner, something industry should welcome.

January 24, 2018
Photo: Betsy Bogard, chair of the International Fibrodysplasia Ossificans Progressiva Association research committee