research reports and media, page-733

Edski, yes it all adds up to assisting us to develop our drug and it's future application to the rare disease of MPS. It is a two way street in that FDA are trying to help the unfortunate patients that have this rare disease AND PAR is also doing their bit in setting up this more holistic approach to the patient's needs by:

1) Tailoring endpoints aimed at the patients, what is the most important aspect to help them?

2) Developing a patient centric focus in recruitment of patients for the final trial.

3) Involving the patients AND the care givers, it's onboarding a different approach to a usual drug development path as it's a rare disease and a number of these patients need full time care.

4) Being cognizant of the fact that this terrible affliction does not just involve joint pain in isolation, there are multiple and often devastating symptoms involved. The ADL (Activities of daily living) come more into play.

5) Working with the National MPS Society within the realms of both recruitment and focus groups to ensure the patients will benefit from our drug while being mindful of their needs.


As I have said this is a two way street with the patient in the middle, they will stand to finally benefit from this approach and the FDA has produced guidelines to this effect¹.


DYOR

References:
1] https://www.fda.gov/media/113653/download
2] https://www.congress.gov/bill/114th-congress/house-bill/34