Drugs for orphan indications are a fascinating area of the pharma industry. Our recent report on the topic looked at the successes, challenges and future for the companies operating in the area and questioned whether we are finally going to see growth tailing off. In our webinar, report authors Melanie Senior and Andreas Hadjivasiliou talked through the key data points. They were joined by Jeremy Levin, CEO of rare disease specialist Ovid Therapeutics to discuss the impact of the looming Inflation Reduction Act (IRA) on the orphan arena. Here’s what we learned.
- One line can have a big impact
In over 700 pages of detailed legislation lurks a single sentence that could upend 40 years of activity. The full ramifications are still unclear but the change means that in future only drugs that treat a single indication will qualify for the benefits afforded under the Orphan Drug Act. Currently, many R&D processes start small – sometimes for an orphan indication – and broaden out to wider, potentially more lucrative indications. This is a fundamental of science and it makes no sense scientifically or commercially to do it the other way around, i.e. start big and focus in. The likely goal was to curb blockbuster orphans but the unintended consequence may be that orphan indications lose out.
- Orphan Drug legislation is a victim of its own success
How did we get here? Years ago, there was very little interest in rare diseases. The original Orphan Drug Act from the 1980s changed that, stimulating a flurry of activity that led to over 500 drugs for orphan conditions being approved in the past 40 years. However, since then the science has galloped forward, leaving us with legislation that is from a different era and an erosion of the original provision. The incentives that sparked all that enthusiasm, when combined with the scientific progress, have led to increasingly small subsets of cancers in particular achieving orphan status for drugs that have become blockbusters.
- Leaving the current legislation alone “is not an option”
The combination of the IRA, the elderly ODA and the huge challenges around reimbursement is no longer sustainable. In the webinar, Jeremy stated that “leaving it alone is not an option” but that any changes around incentives for orphan drugs must be done in consultation with industry. Otherwise, as with the IRA, there will be unintended consequences that will lead to patients with rare diseases having fewer options. Notably, most rare disease drugs are not created by big pharma but are bought or licenced in. Currently the policy makers don't discriminate between the small innovators and the big companies who take them on.
Those were my key takeaways from the session but is so much more in the webinar, particularly around the nuances of the IRA and the challenges raised by reimbursement. To get all the views and data from Melanie, Andreas and Jeremy, grab your headphones and check out the on-demand webinar now. Or dig further into the data in some of the other pieces we have available here.