The Food and Drug Administration (FDA) set a new record in 2018 with the highest number of new drug approvals in the last two decades. The FDA also set a new record in orphan drug approvals in 2018, granting 86 new marketing authorizations for drugs treating rare diseases. In this article I review the delay between orphan drug designation and orphan drug approval, and identify how in many cases orphan drugs wait 10 or more years after reviewing the orphan drug designation and before they get approved.

When you let the scientific community know that your organization is open to fund research around your rare disease you are likely to get many research proposals from academic groups. These will come in different qualities, and will have different relevancefor your disease. This entry adds to Impatient Series I and to the ImpatientRevolution book and discusses in more detail how to determine that a proposed project is the right one and how to monitor its progress.

Once there is a drug that has been developed, it is very clear why talking with patients and collaborating with them is useful for pharmaceutical companies. What is less obvious to both companies and patient organizations is how patients can be active in research before there is any drug in development, during the preclinical phase, and how this might lead to medicines being developed. Some patient organizations might start by funding some academic group, but without a clear vision and a longer-term strategy the return on those efforts will be compromised, and medicines will take longer to come. Here I want to outline the broader strategy that patient organizations can follow to advance research towards new medicines even before companies are working on it.

In the previous entry we discussed the many types of companies that get interested in rare diseases, each of them because of different reasons. If your disease field matches one of those business propositions you will get at the top of the list of interesting diseases for that company, but another part of the decision equation is time, or field maturity. Is this field ready for us to start working on it already The following are the questions that a company will often need to answer to be able to judge if the field is ready for them, too early for them, or maybe even too mature for them to get in.