Rare diseases

Sharing the mission to conquer rare diseases

Closer collaboration between pharmaceutical companies and patient organizations will accelerate drug development

At an ever-quickening pace, biopharmaceutical companies and patient advocacy groups are banding together to hasten development of new drugs for rare diseases. Advocacy groups rely on their partners for financial and scientific support while providing access to repositories of patient data – the key to understanding rare diseases – as well as assistance with clinical trial enrollment. These relationships bring tremendous value to both sides but also present challenges. Increasingly, patient groups expect to be treated as equal partners. They want companies to consult with them earlier in the design of clinical trials, give them greater access to data generated in the studies, and make longer-term commitments to assist their patient communities. In this report, inVentiv Health looks at the dynamics reshaping collaboration in the rare disease space.

INTRODUCTION

When Congress passed the Orphan Drug Act (ODA) in 1983, no one could guarantee that financial incentives in the law would spur development of life-saving drugs. But, the framework has proven highly effective. It provided tax credits, relief from regulatory fees, and other incentives for companies that develop treatments for some 7,000 rare conditions – those affecting fewer than 200,000 people.
In the three-plus decades since the law was signed, the FDA has approved more than 500 drugs for rare diseases and granted more than 3,000 orphan drug “designations.” Europe and Japan followed the US in drafting orphan drug laws, adding to a worldwide boom in drug development aimed at rare diseases. These medications have delivered immense health benefits to patients and opened up business opportunities at a time when pharma companies are grappling with patent cliffs and other hurdles. Global sales of orphan drugs have been growing nearly 12% per year and could reach $178 billion in 2020, equal to one-fifth of annual prescription drug sales, excluding generics, according to consultancy EvaluatePharma.

Advocacy groups, working on behalf of patients and their families, contributed mightily to these medical and commercial success stories. Many drug companies have partnered with these organizations to better understand the natural course of diseases, identify patients who are eligible to participate in clinical trials, and present a compelling case to regulators and payers. Companies supported these organizations and, as a result, earned the privilege to get closer to patients, hear their voices first-hand, and then leverage what they learn to make better products. Up to a point, both sides are satisfied when partnerships contribute to the successful launch of life-altering medications. In the eyes of many advocacy groups, however, these relationships are still works in progress.

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We will be discussing rare diseases at the BioData World Congress on 26-27 October at Wellcome Genome Campus, Cambridge.

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