Massachusetts Paves the Way for Rare Disease and Orphan Drug Development

A recent article in the Boston Business Journal has revealed the full extent of the Bay State’s booming rare disease research sector. Massachusetts is home to more of the biggest drugs in rare diseases — whether they have been approved or are in late-stage development — than any other state in the United States. The analysis, which was based on data from life science market intelligence firm, EvaluatePharma, demonstrates Massachusetts’ commitment to novel drug development and further solidifies its place as one of the top hubs for life science research.

Why the Investment in Rare Diseases?

The Orphan Drug Act, which went into effect in 1983, was designed to push rare disease drug development forward in the form of financial incentives from the government and regulatory assistance from the Food & Drug Administration (FDA). Moreover, orphan drugs can stay on the market longer before generic competition is allowed — a particularly important edge as patent cliff woes threatened to erode the sales of blockbuster drugs. With all this considered, it’s no wonder orphan drugs will account for 20.2 percent of worldwide prescription sales by 2020, according to EvalutePharma’s Orphan Drug Report 2015.

Source: EvaluatePharma

Not only is this great news for the small — and medium-sized Boston-based companies with limited sales and marketing budgets that have staked their pipelines around the rare disease industry, but with 30 million rare disease patients, the increased investment in this community brings great hope for the many families and health professionals searching for life-saving treatments.

“We’re entering the golden age of orphan drugs,” says Chris Clark, investment manager at RS Investments. “In the next three to five years the industry will usher in many successful drugs that will eradicate diseases. Our children won’t know the diseases we knew growing up.”

Overcoming Communication Challenges

While the rare disease market continues to thrive, there remain several communication challenges that small-to-mid-size companies, in particular, still need to overcome. How companies reach this influential community by building connections not only among patients but also among health professionals — such as nurses and physicians, patient advocates, scientists and payers — is key.

Successful communication requires you to become a part of the community your specific drug treats before you can begin marketing your products. Social media is a great tool for communicating with this active community, particularly when it comes to patient enrollment in clinical trials, as we discussed in this earlier Chemunity post. The Internet and social media channels give companies an opportunity to become a part of the conversation and build trusting relationships with your key influencers. By partnering with patient support groups and organizations, you can gain a better understanding of your audience’s needs in order to effectively educate them about your services, products, and resources.

The life science community has experienced tremendous growth over the past few years and nowhere is that more apparent than in the rare disease space. As Boston continues its domination over this market, finding meaningful ways to communicate these gains to the patient community will remain paramount to continued success.