Rare Disease Lobbying Works, but Is It Worth it?

In Regulation & Government by CameronLeave a Comment

New study shows that lobbying by rare disease patient groups can influence between 3-15% of NIH funding allocated for rare disease research

A report released from the New York University Stern School of Business has analysed the impact of rare disease lobbying by patient groups on funding allocated by the National Institute of Health. The report suggests that through congressional ‘soft earmarks’ rare disease groups are able to influence the allocation of anywhere between 3% to 15% of the NIH’s funding allocation for rare diseases.

The lobbying of government and federal agencies has long been criticised by those who see it as a way for the most powerful patient groups to dictate the interests of the NIH and it’s funding programs. And as the largest funder of medical research in the world, if true, lobbying would certainly be a questionable practice. But this new report suggests that bigger does not equal better when it comes to lobbying for rare disease groups.

The report highlights two additional features that dictate the effectiveness of a groups lobbying: scientific opportunity, and disease burden. This means that effective lobbying is multiplied by the presence of exciting and new scientific opportunities to be explored, as well as the burden of the disease and the potential relief that could be brought to patients in the long term. And with lobbying only affecting between 3% and 15% of the NIH’s spend on rare diseases, it’s good news for anyone concerned that lobbying might be dramatically skewing their funding priorities.

The author’s suggestion is that lobbying could well be acting to inform politicians and legislators about rare diseases, their burden on patients, as well as their public burden. This then translates into ‘soft earmarks’: language in congressional appropriations bills that encourage spending but is not enshrined in law.

“Lobbying may be transmitting information about the public burden [of rare diseases] and scientific opportunities [for research] into the allocation process that might not otherwise use this information. It may have a beneficial effect, but we don’t have a conclusive way of establishing that.”Deepak Hegde, NYU 

The NIH funding that was diverted towards causes that were earmarked tended to be through ‘requests for applications’ and ‘program announcements.’

The authors, Deepak Hegde (NYU) and Bhaveb Sampat (Columbia University), are clearly optimistic about the role of lobbying when it comes to rare disease research. Presumably this is is down the the tiny percentage of funding that is actually affected, along with the other factors that determine the effectiveness of lobbying. But perhaps we should be concerned with competition at the rare disease level. There are numerous ultra-rare diseases with tiny patient populations, can these small groups compete with groups with greater numbers and greater funding? But perhaps the amount of funding up for grabs through lobbying is so small we shouldn’t even worry about smaller rare disease groups not necessarily getting a say.

One question would like to see answered however, is whether it is economical for rare disease groups to lobby politicians if the funding available is so small. Perhaps these groups should dedicate more time to ensuring that viable, exciting, and innovative new scientific opportunities are available for future treatments. This way they might stand a better chance at accessing the remaining 97% of peer-reviewed funding.

You can find the report here.

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